Evaluation of Different Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease
2021年5月3日 更新者：Baxalta now part of Shire
A Phase 2, Exploratory, Placebo-Controlled, Multicenter, Double-Blind Evaluation of the Safety, Pharmacokinetics, Pharmacodynamics, and Clinical Effects of Two Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease
Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of sickle-shaped red blood cells. In the U.S. and the U.K. this occurs primarily in persons of African origin. There is only one drug (hydroxyurea) approved to manage SCD, but it is not fully efficacious and can produce medically significant side effects. Aes-103 is being evaluated as a novel agent for the long term management of SCD. By directly reducing the sickling process, Aes-103 has a different mechanism of action than hydoxyurea. The active ingredient in Aes-103 is 5-hydroxymethyl furfural, a naturally occurring small molecule that is chemically related to glucose.
This study will evaluate the safety and pharmacokinetic profile of two dosing regimens of Aes-103 for up to 28 days in up to 50 adult subjects with stable SCD compared with subjects receiving placebo.
研究概览
详细说明
This study will evaluate evaluate in subjects with stable SCD the safety, pharmacokinetic profile, clinical pharmacology actions and clinical activities of two dosing regimens of Aes-103 (1000 mg four times daily in Cohort A and a higher or lower dose given once daily or up to four times daily in Cohort B) given for up to 28 days in adult subjects with stable SCD compared with subjects receiving placebo.
研究类型
介入性
注册 (实际的)
35
阶段
- 阶段2
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习地点
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London、英国、SE1 1YR
- Quintiles Ltd. - Quintiles Drug Research Unit, 6 Newcomen Street
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
18年 至 60年 (成人)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Male or female, aged 18-60 years old, inclusive
- Diagnosis of SCD (hemoglobin SS) without hospitalization for pain crises or any other reason in the 14 days before enrollment
- Have normal organ function as defined by direct bilirubin <1.1 mg/dL (19 μmol/L), alanine transaminase (serum glutamic pyruvic transaminase) ≤120 IU/L, and Creatinine ≤1.3 mg/dL (115 μmol/L)
- Have at least one of the following baseline values: hemoglobin level of <10 g/dL, numerical pain rating scale (NPRS) score of ≥ 4, or 6-minute walk distance (6MWD) of <500 m
- If female, be nonpregnant and nonbreastfeeding and be surgically sterile or using an acceptable method of contraception throughout the study and for 3 months after the last dose of study medication
- Have completed an outpatient screening visit consisting of medical history, physical examination, 12-lead electrocardiogram (ECG), vital signs, hematology and chemistry tests, urinalysis, urine drug screen, urine or serum pregnancy test (females), hemoglobin electrophoresis, hepatitis B and C screening, and HIV serology
- Be able to understand and have provided written informed consent including signature on an informed consent form approved by an institutional review board or independent ethics committee
- Have provided written authorization for use and disclosure of protected health information
- Agree to abide by the study schedule and to return for the required assessments
Exclusion Criteria:
- Have been hospitalized in the 14 days before enrollment, for any reason
- Have evidence of clinically significant cardiovascular, respiratory, renal, hepatic, pulmonary, gastrointestinal, hematological, neurological, psychiatric, or other disease that may interfere with the objectives of the study or the safety of the subject, or have been hospitalized in the past 6 months as a result of these conditions (for SCD-related morbidity, a minimum of 14 days from the last hospitalization is required)
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：三倍
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的：Cohort A (Drug)
Subjects randomized 3:1 to receive 4 times daily dosing of 1,000 mg of Aes 103 or placebo for 28 days
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The active ingredient in Aes-103 is 5-hydroxymethyl furfural (5-HMF).
Aes-103 and matching placebo are administered in a liquid oral formulation.
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安慰剂比较：Cohort A (Placebo)
Subjects randomized 3:1 to receive 4 times daily dosing of 1,000 mg of Aes 103 or placebo
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实验性的：Cohort B (Drug)
In this adaptive design, the dose frequency and the total amount given per day to Cohort B will be adjusted depending on the tolerability, clinical pharmacology and clinical endpoint results of Cohort A. Study terminated prior to completion of Cohort A due to unblinding between study product and placebo groups for participant, site and Sponsor.
The study was stopped before initiation of Cohort B.
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The active ingredient in Aes-103 is 5-hydroxymethyl furfural (5-HMF).
Aes-103 and matching placebo are administered in a liquid oral formulation.
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安慰剂比较：Cohort B (Placebo)
The dosing regiment of placebo will match that of the Aes-103 treatment in Cohort B. Study terminated prior to completion of Cohort A due to unblinding between study product and placebo groups for participant, site and Sponsor.
The study was stopped before initiation of Cohort B.
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Number of Participants With Adverse Events, Including Sickle Cell-specific Symptoms, During the Double-blind Treatment Period
大体时间：Double-blind treatment period of 28 days (Day 1 to Day 28)
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Number of participants with adverse events (AEs) reported during the double-blind treatment period.
AEs included clinically-significant changes in vital signs, ECG, clinical laboratory assessments, physical and neurological examinations.
Sickle cell-specific symptoms included the development of new skin ulcers, hospitalization or ambulatory acute care, intravenous analgesics visit for pain episodes (i.e., sickle-cell disease related pain), acute chest syndrome, priapism, and stroke.
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Double-blind treatment period of 28 days (Day 1 to Day 28)
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Number of Participants With Adverse Events, Including Sickle-cell Specific Symptoms, During the Placebo lead-in Period
大体时间：Placebo lead-in period of 14 days (Day -14 to Day -1)
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Number of participants with adverse events (AEs) reported during the placebo lead-in period.
AEs included clinically-significant changes in vital signs, ECG, clinical laboratory assessments, physical and neurological examinations.
Sickle cell-specific symptoms included the development of new skin ulcers, hospitalization or ambulatory acute care, intravenous analgesics visit for pain episodes (i.e., sickle-cell disease related pain), acute chest syndrome, priapism, and stroke.
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Placebo lead-in period of 14 days (Day -14 to Day -1)
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Number of Participants With Adverse Events, Including Sickle-cell Specific Symptoms, During the Post-treatment Observation Period
大体时间：Post-treatment observation period of 21 days (Day 29 to Day 49)
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Number of participants with adverse events (AEs) reported during the post-treatment observation period.
AEs included clinically-significant changes in vital signs, ECG, clinical laboratory assessments, physical and neurological examinations.
Sickle cell-specific symptoms included the development of new skin ulcers, hospitalization or ambulatory acute care, intravenous analgesics visit for pain episodes (i.e., sickle-cell disease related pain), acute chest syndrome, priapism, and stroke.
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Post-treatment observation period of 21 days (Day 29 to Day 49)
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Number of Participants With Sickle-Cell Disease-related Symptoms
大体时间：Placebo lead-in period of 14 days (Day -14 to Day -1), double-blind treatment period of 28 days (Day 1 to Day 28) and post-treatment observation period of 21 days (Day 29 to Day 49)
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Placebo lead-in period of 14 days (Day -14 to Day -1), double-blind treatment period of 28 days (Day 1 to Day 28) and post-treatment observation period of 21 days (Day 29 to Day 49)
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Number of Clinically Significant Observations of Vital Signs, 12-lead ECGs, Clinical Laboratory Assessments, and Physical and Neurological Examinations
大体时间：Throughout the study period (approximately 9 weeks)
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Vital signs, 12-lead ECGs, clinical laboratory assessments, and physical and neurological examinations that were deemed clinically significant by the investigator in agreement with the sponsor study director.
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Throughout the study period (approximately 9 weeks)
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PK: - Plasma AUC, Cmax, Tmax, and T1/2 of Aes-103 and Its Metabolite, HMFA - RBC Hemolysate AUC (0-8h), Cmax, Tmax, and T1/2 of Aes-103 - Percentage of Hemoglobin Bound to Aes-103
大体时间：PK blood samples were to be taken within 10 minutes before dosing and 0.5, 1, 2, 4, and 6 hours after the first dose of study product on Days 1 and 7 and at the same time points on Day 28 (or early termination)
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Pharmacokinetic endpoints in the study protocol were as follows:- - Plasma Area under curve (AUC), Maximum plasma concentration (Cmax), time at which Cmax observed (Tmax), and terminal half-life (T1/2) of Aes-103 and its metabolite, 5-hydroxymethyl-2-furoic acid (HMFA) - red blood cell (RBC) hemolysate Area under curve between 0 and 8 hours (AUC [0-8h]), Cmax, Tmax, and T1/2 of Aes-103 - Percentage of hemoglobin bound to Aes-103
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PK blood samples were to be taken within 10 minutes before dosing and 0.5, 1, 2, 4, and 6 hours after the first dose of study product on Days 1 and 7 and at the same time points on Day 28 (or early termination)
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
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Resting Oxygen Saturation as Measured by Oximetry (SpO2) - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 4, Day 7, Day 14 and Day 28
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A measure of the amount of oxygen in the blood.
Oxygen saturation was determined by pulse oximetry.
A pulse oximeter was placed over a nail polish-free finger nail to determine peripheral oxygen saturation (SpO2).
Baseline was defined as the most recent value obtained prior to the start of dosing on Day 1 of the double-blind treatment period.
A mean change from baseline >0 indicates an increase in oxygen saturation, a mean change <0 indicates a decrease in oxygen saturation.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 4, Day 7, Day 14 and Day 28
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Oxygen Binding p50/p20 Value - Change From Baseline
大体时间：During the double-blind treatment period at baseline, Day 1, Day 4 and Day 7
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A measure of the ability of hemoglobin to bind oxygen.
The p50 is the oxygen level at which 50% of the hemoglobin contains oxygen.
The p20 is the oxygen level at which 20% of the hemoglobin contains oxygen.
Baseline is defined as the most recent value obtained prior to start of dosing on Day 1 of the double-blind treatment period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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During the double-blind treatment period at baseline, Day 1, Day 4 and Day 7
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Plasma Erythropoietin (EPO) Levels - Change From Baseline
大体时间：At baseline and Day 28 during the double-blind treatment period
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Erythropoietin (EPO) is a hormone produced by the kidney that promotes the formation of red blood cells by the bone marrow.
EPO can be detected and measured in the blood.
Baseline defined as the most recent value obtained prior to the start of dosing on Day 1 of the double-blind treatment period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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At baseline and Day 28 during the double-blind treatment period
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Hematocrit Levels - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14 and Day 28
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Baseline defined as the most recent value obtained prior to the start of dosing on Day 1 of the double-blind treatment period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14 and Day 28
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Lactate Dehydrogenase (LDH) Levels - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14 and Day 28
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LDH levels were measured as a biomarker for intravascular hemolysis.
The results are based on the LDH Total measurement.
Baseline defined as the most recent value obtained prior to the start of dosing on Day 1 of the double-blind treatment period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14 and Day 28
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Hemoglobin Levels - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 4, Day 7, Day 14, Day 21 and Day 28
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A clinical laboratory endpoint that reflects the amount of red blood cells present in the blood.
Baseline defined as the most recent value obtained prior to the start of dosing on Day 1 of the double-blind treatment period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 4, Day 7, Day 14, Day 21 and Day 28
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Reticulocyte Percent- Change From Baseline
大体时间：At baseline, Day 1 and Day 7 during the double-blind treatment period
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Category title includes number of participants with available data (n) for participants treated with study product.
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At baseline, Day 1 and Day 7 during the double-blind treatment period
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Direct Bilirubin - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14, Day 21 and Day 28
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Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14, Day 21 and Day 28
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LDH Isoform - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14 and Day 28
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 7, Day 14 and Day 28
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C Reactive Protein Levels - Change From Baseline
大体时间：At baseline, Day 1 and Day 7 during the double-blind treatment period
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Category title includes number of participants with available data (n) for participants treated with study product.
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At baseline, Day 1 and Day 7 during the double-blind treatment period
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Serum Ferritin Levels - Change From Baseline
大体时间：At baseline, Day 1 and Day 7 during the double-blind treatment period
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At baseline, Day 1 and Day 7 during the double-blind treatment period
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N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) Levels- Change From Baseline
大体时间：At baseline, Day 1, Day 7, Day 14 and Day 28 during the double-blind treatment period
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Category title includes number of participants with available data (n) for participants treated with study product.
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At baseline, Day 1, Day 7, Day 14 and Day 28 during the double-blind treatment period
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Body Weight - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 4, Day 7, Day 14, Day 21 and Day 28
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A negative change in body weight denotes a weight decrease, a positive change in body weight denotes a weight increase.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 1, Day 4, Day 7, Day 14, Day 21 and Day 28
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Exercise Tolerance: 6-Minute Walk Distance During the Double-blind Treatment Period - Change From Baseline
大体时间：Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 4, Day 7, Day 14 and Day 28
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Functional exercise capacity was evaluated by using the 6-minute walk test (6MWT) which measures the distance that a subject can quickly walk on a flat, hard surface in a period of 6 minutes.
Guidelines developed by the American Thoracic Society were used for conducting the test and interpreting the results.
Baseline defined as the most recent value obtained prior to the start of dosing on Day 1. Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Prior to, during and after the end of dosing in the double-blind treatment period, i.e., at baseline, Day 4, Day 7, Day 14 and Day 28
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Exercise Tolerance: 6-Minute Walk Distance on Day 49 of the Post-treatment Observation Period - Change From Baseline
大体时间：Prior to dosing at baseline and on Day 49 of the post-treatment observation period
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Functional exercise capacity was evaluated by using the 6-minute walk test (6MWT) which measures the distance that a subject can quickly walk on a flat, hard surface in a period of 6 minutes.
Guidelines developed by the American Thoracic Society were used for conducting the test and interpreting the results.
Baseline defined as the most recent value obtained prior to the start of dosing on Day 1.
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Prior to dosing at baseline and on Day 49 of the post-treatment observation period
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Exercise Tolerance: 6-Minute Walk Distance on Day 49 of the Post-treatment Observation Period - Change From Last Day of Double-blind Treatment Period (Day 28)
大体时间：On last day of double-blind treatment period (Day 28) and on Day 49 of the post-treatment observation period
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Functional exercise capacity was evaluated by using the 6-minute walk test (6MWT) which measures the distance that a subject can quickly walk on a flat, hard surface in a period of 6 minutes.
Guidelines developed by the American Thoracic Society were used for conducting the test and interpreting the results.
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On last day of double-blind treatment period (Day 28) and on Day 49 of the post-treatment observation period
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Exercise Tolerance: Cardiopulmonary Exercise Test [CPET]
大体时间：On last day of double-blind treatment period (Day 28)
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CPET was optional, based on capacity of participant to complete the test.
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On last day of double-blind treatment period (Day 28)
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Patients´ Global Impression of Change (PGIC) During the Double-blind Treatment Period - Change From Baseline
大体时间：At baseline and once weekly on Days 7, 14, 21, and 28 during the double-blind treatment period
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Participants assessed the change in activity limitations, symptoms, emotions, and overall quality of life by using the 1- to 7-point PGIC scale.
The question and scale was as follows: Since beginning treatment at this clinic, how would you describe the change in your sickle cell condition?
Please circle the number that matches your overall judgment.
-3 - much worse -2 - moderately worse -1 - minimally worse 0 - no change +1 - minimally improved +2 - moderately improved +3 - much improved.
Baseline defined as the most recent value obtained prior to the start of dosing on Day 1 of the double-blind treatment period.
No values available for placebo group for Day 28.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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At baseline and once weekly on Days 7, 14, 21, and 28 during the double-blind treatment period
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Patients´ Global Impression of Change (PGIC) During the Post-treatment Observation Period - Change From Baseline
大体时间：At baseline and once weekly on Days 35, 42, and 49 during the post-treatment observation period
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Participants assessed the change in activity limitations, symptoms, emotions, and overall quality of life by using the 1- to 7-point PGIC scale.
The question and scale was as follows: Since beginning treatment at this clinic, how would you describe the change in your sickle cell condition?
Please circle the number that matches your overall judgment.
-3 - much worse -2 - moderately worse -1 - minimally worse 0 - no change +1 - minimally improved +2 - moderately improved +3 - much improved.
Baseline defined as the most recent value obtained prior to the start of dosing on Day 1 of the double-blind treatment period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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At baseline and once weekly on Days 35, 42, and 49 during the post-treatment observation period
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Patients´ Global Impression of Change (PGIC) During the Post-treatment Observation Period - Change From Last Day of Double-blind Treatment Period
大体时间：At baseline and once weekly on Days 35, 42, and 49 during the post-treatment observation period
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Participants assessed the change in activity limitations, symptoms, emotions, and overall quality of life by using the 1- to 7-point PGIC scale.
The question and scale was as follows: Since beginning treatment at this clinic, how would you describe the change in your sickle cell condition?
Please circle the number that matches your overall judgment.
-3 - much worse -2 - moderately worse -1 - minimally worse 0 - no change +1 - minimally improved +2 - moderately improved +3 - much improved.
Baseline was defined as the most recent value obtained on the last day of the double-blind treatment period.
Categories contain Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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At baseline and once weekly on Days 35, 42, and 49 during the post-treatment observation period
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Numerical Pain Rating Scale (NPRS): Worst Pain (Weekly Average) in the Double-blind Treatment Period - Change From Baseline
大体时间：Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 7, Day 14, Day 21, and Day 28 assessments
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Participants assessed their pain levels by using the 0-10 Numeric Rating Scale: 0 = No pain 1-3 = Mild pain (nagging, annoying, interfering little with activities of daily living [ADLs]) 4-6 = Moderate pain (interferes significantly with ADLs) 7-10 = Severe pain (disabling; unable to perform ADLs A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the average of all measures taken from screening through the period prior to start of dosing on Day 1. Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 7, Day 14, Day 21, and Day 28 assessments
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Numerical Pain Rating Scale (NPRS): Worst Pain (Weekly Average) in the Double-blind Treatment Period - AUC
大体时间：Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 7, Day 14, Day 21, and Day 28 assessments
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Participants assessed their pain levels by using the 0-10 Numeric Rating Scale: 0 = No pain 1-3 = Mild pain (nagging, annoying, interfering little with activities of daily living [ADLs]) 4-6 = Moderate pain (interferes significantly with ADLs) 7-10 = Severe pain (disabling; unable to perform ADLs A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the average of all measures taken from screening through the period prior to start of dosing on Day 1. Area under the curve (AUC) was computed using change from baseline in weekly average values at Day 7, Day 14, Day 21. and Day 28.
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Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 7, Day 14, Day 21, and Day 28 assessments
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Numerical Pain Rating Scale (NPRS): Worst Pain (Weekly Average) in the Post-treatment Observation Period - Change From Baseline
大体时间：Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
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Participants assessed their pain levels by using the 0-10 Numeric Rating Scale: 0 = No pain 1-3 = Mild pain (nagging, annoying, interfering little with activities of daily living [ADLs]) 4-6 = Moderate pain (interferes significantly with ADLs) 7-10 = Severe pain (disabling; unable to perform ADLs A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the average of all measures taken from screening through the period prior to start of dosing on Day 1. Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
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Numerical Pain Rating Scale (NPRS): Worst Pain (Weekly Average) in the Post-treatment Observation Period - AUC
大体时间：Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
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Participants assessed their pain levels by using the 0-10 Numeric Rating Scale: 0 = No pain 1-3 = Mild pain (nagging, annoying, interfering little with activities of daily living [ADLs]) 4-6 = Moderate pain (interferes significantly with ADLs) 7-10 = Severe pain (disabling; unable to perform ADLs A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the average of all measures taken from screening through the period prior to start of dosing on Day 1. Area under the curve (AUC) was computed using change from baseline in weekly average values at Day 7, Day 14, Day 21, Day 28, Day 35, Day 42 and Day 49.
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Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
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Numerical Pain Rating Scale (NPRS): Worst Pain (Weekly Average) in the Post-treatment Observation Period - Change From Last Day of Double-blind Treatment Period (Day 28)
大体时间：Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
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Participants assessed their pain levels by using the 0-10 Numeric Rating Scale: 0 = No pain 1-3 = Mild pain (nagging, annoying, interfering little with activities of daily living [ADLs]) 4-6 = Moderate pain (interferes significantly with ADLs) 7-10 = Severe pain (disabling; unable to perform ADLs A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the most recent value obtained on the last day of the double-blind dosing period (Day 28).
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
|
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Numerical Pain Rating Scale (NPRS): Worst Pain (Weekly Average) in the Post-treatment Observation Period - Change From Last Day of Double-blind Treatment Period (Day 28) - AUC
大体时间：Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
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Participants assessed their pain levels by using the 0-10 Numeric Rating Scale: 0 = No pain 1-3 = Mild pain (nagging, annoying, interfering little with activities of daily living [ADLs]) 4-6 = Moderate pain (interferes significantly with ADLs) 7-10 = Severe pain (disabling; unable to perform ADLs A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the most recent value obtained on the last day of the double-blind dosing period (Day 28).
Area under the curve (AUC) was computed using change from baseline (Day 28) in weekly average values at Day 35, Day 42 and Day 49.
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Participants assessed and recorded their pain level daily. Weekly averages were calculated for the Day 35, Day 42 and Day 49 assessments
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Brief Pain Inventory (BPI): Average Pain Level in Last 24 Hours (Double-blind Treatment Period) - Change From Baseline
大体时间：At baseline, Day 7 and Day 28 during the double-blind treatment period
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Participants rated the severity of their pain by using the BPI short form.
Pain was rated on a scale from 0 (no pain) to 10 (pain as bad as you can imagine).
A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the most recent value obtained prior to the start of the dosing on Day 1 of the double-blind dosing period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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At baseline, Day 7 and Day 28 during the double-blind treatment period
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Brief Pain Inventory (BPI): Worst Pain Level in Last 24 Hours (Double-blind Treatment Period) - Change From Baseline
大体时间：At baseline, Day 7 and Day 28 during the double-blind treatment period
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Participants rated the severity of their pain by using the BPI short form.
Pain was rated on a scale from 0 (no pain) to 10 (pain as bad as you can imagine).
A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the most recent value obtained prior to the start of the dosing on Day 1 of the double-blind dosing period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
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At baseline, Day 7 and Day 28 during the double-blind treatment period
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Brief Pain Inventory (BPI): Worst Pain Level in Last 24 Hours (Post-treatment Observation Period) - Change From Baseline
大体时间：At baseline and Day 49 during the post-treatment observation period
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Participants rated the severity of their pain by using the BPI short form.
Pain was rated on a scale from 0 (no pain) to 10 (pain as bad as you can imagine).
A negative mean change denotes a pain decrease.
A positive mean change denotes an increase in pain.
Baseline was defined as the most recent value obtained prior to the start of the dosing on Day 1 of the double-blind dosing period.
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At baseline and Day 49 during the post-treatment observation period
|
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Brief Pain Inventory (BPI): Interference of Pain With Aspects of Life (General Activity) During Double-blind Treatment Period - Change From Baseline
大体时间：At baseline, Day 7 and Day 28 during the double-blind treatment period
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Participants rated the degree to which their pain interfered with various daily functions by using the BPI short form.
Interference with general activity was rated on a scale from 0 (does not interfere) to 10 (completely interferes).
Baseline was defined as the most recent value obtained prior to the start of the dosing on Day 1 of the double-blind dosing period.
Category title includes number of participants with available data (n) for participants treated with study product, followed by participants treated with placebo.
|
At baseline, Day 7 and Day 28 during the double-blind treatment period
|
|
Brief Pain Inventory (BPI): Interference of Pain With Aspects of Life (General Activity) During Post-treatment Observation Period - Change From Baseline
大体时间：At baseline and Day 49 during the post-treatment observation period
|
Participants rated the degree to which their pain interfered with various daily functions by using the BPI short form.
Interference with general activity was rated on a scale from 0 (does not interfere) to 10 (completely interferes).
Baseline was defined as the most recent value obtained prior to the start of the dosing on Day 1 of the double-blind dosing period.
|
At baseline and Day 49 during the post-treatment observation period
|
|
Analgesic Use
大体时间：Throughout the study period (approximately 9 weeks)
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Analgesic use assessed with pain levels by numerical pain rating scale (NPRS) and brief pain inventory (BPI).
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Throughout the study period (approximately 9 weeks)
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Reduction in Sickle Cell-specific Complications
大体时间：Throughout the study period (approximately 9 weeks)
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Throughout the study period (approximately 9 weeks)
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2013年12月3日
初级完成 (实际的)
2015年3月16日
研究完成 (实际的)
2015年3月16日
研究注册日期
首次提交
2013年10月29日
首先提交符合 QC 标准的
2013年11月13日
首次发布 (估计)
2013年11月20日
研究记录更新
最后更新发布 (实际的)
2021年5月25日
上次提交的符合 QC 标准的更新
2021年5月3日
最后验证
2021年5月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.
安慰剂的临床试验
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City of Hope Medical CenterNational Cancer Institute (NCI)主动，不招人造血和淋巴细胞肿瘤 | 骨髓纤维化 | 慢性淋巴细胞白血病 | 缓解期成人急性髓性白血病 | 骨髓增生异常综合症 | 缓解期成人急性淋巴细胞白血病 | 骨髓增殖性肿瘤 | 慢性期慢性粒细胞白血病，BCR-ABL1 阳性 | 成人淋巴母细胞淋巴瘤 | 加速期慢性粒细胞白血病，BCR-ABL1 阳性 | HLA-A*0201 阳性细胞存在 | 巨细胞病毒感染 | 成人霍奇金淋巴瘤 | 成人非霍奇金淋巴瘤美国
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Mila (bMotion Technologies)完全的
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Universidad Autonoma de MadridCentro Universitario La Salle完全的