HOPE-2 试验的开放标签扩展 (HOPE-2-OLE)
HOPE-2 杜氏肌营养不良症试验的开放标签扩展
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months, over a period of approximately 60 months. Following completion of the initial open-label phase (Month 60), subjects who have completed all Month 60 assessments will be eligible to continue into a long-term open label extension (LT-OLE) period and can continue to receive deramiocel once every 3 months until deramiocel is commercially available or the sponsor terminates the study, or the subject withdraws consent or study participation is terminated by the sponsor.
Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, and all LT-OLE visits. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and at all LT-OLE trial visits, unless otherwise indicated.
All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, with continued dosing in the LT-OLE visits. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.
研究概览
详细说明
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months, over approximately a period of 60 months. Additional analyses of efficacy and safety will be conducted in the subsequent Long Term Open-Label Extension (LT-OLE) phase of the study.
Subjects will undergo a targeted screening during a 30-day screening period to determine eligibility based on protocol inclusion and exclusion criteria.
Eligible subjects will undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel. Administration of deramiocel (Day 1) should occur within a maximum of 30 days following confirmation of eligibility.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12 (± 14 days, each), 15, 18, 21, 24, 27, 30, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, and all LT-OLE visits (± 21 days, each). Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated, and at all LT-OLE visits.
All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57 trial visits. Continued dosing in the LT-OLE phase will begin at LT-OLE Visit 1, which may overlap with Month 60 of the initial OLE phase/LT-OLE Visit 1, in some cases. Prior to each deramiocel administration, medications will be administered to the subject as determined by the Investigator based on the pre-treatment guidelines as outlined in the protocol and/or institutional protocols to minimize the risk of potential severe allergic reactions such as anaphylaxis. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day if medically cleared by the site Investigator. If clinically indicated, an unscheduled in-person visit will be performed at the investigative site with targeted assessments based on presentation of signs and symptoms following any infusion.
研究类型
注册 (实际的)
阶段
- 阶段2
联系人和位置
学习地点
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California
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Sacramento、California、美国、95817
- University of California, Davis
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Colorado
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Aurora、Colorado、美国、80045
- Children's Hospital Colorado
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Missouri
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St Louis、Missouri、美国、63110
- Washington University
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Ohio
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Cincinnati、Ohio、美国、45229
- Cincinnati Children's Hospital Medical Center
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Wisconsin
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Milwaukee、Wisconsin、美国、53226
- Children's Hospital Wisconsin
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12
- Willing and able to provide informed consent to participate in the trial if ≥ 18 years of age, and assent with parental or guardian informed consent if < 18 years of age
- Adequate venous access for intravenous deramiocel (CAP-1002) infusions in the judgement of the Investigator
- Assessed by the Investigator as willing and able to comply with the requirements of the trial
Exclusion Criteria:
- Planned or likely major surgery in the next 12 months after planned first infusion
- Risk of near-term respiratory decompensation in the judgment of the investigator, or the need for initiation of non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L
- History of non DMD-related chronic respiratory disease including, but not limited to, asthma, bronchitis, and tuberculosis
- Acute respiratory illness within 60 days prior to first infusion
- Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products
- Treatment with an investigational product ≤ 6 months prior to first infusion
- History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial
- Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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其他：Deramiocel
Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months |
Peripheral infusion of 150 million allogeneic cardiosphere-derived cells administered every three months
其他名称：
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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 Treatment-Emergent Adverse Events (TEAEs) From Baseline Through Month 12
大体时间：Baseline up to Month 12
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Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs. |
Baseline up to Month 12
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12
大体时间：Baseline up to Month 12
|
Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death. |
Baseline up to Month 12
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Change From Baseline in Functional Capacity as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) Total Score.
大体时间：Baseline, Month 12
|
PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. |
Baseline, Month 12
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) at Month 24, Month 36, Month 48 and Month 60
大体时间：Month 24, Month 36, Month 48 and Month 60
|
Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs. |
Month 24, Month 36, Month 48 and Month 60
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 60
大体时间：Baseline up to Month 60
|
Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death. |
Baseline up to Month 60
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Change From Baseline in Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
|
PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. |
Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
|
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Change From Baseline in Distal-Level (Wrist and Hand) Upper Limb Function as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
|
PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. |
Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
|
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Change From Baseline in Mid-Level (Elbow) as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) at Month 12, Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
|
PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. |
Baseline, Month 12, Month 24, Month 36, Month 48, and Month 60
|
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Change From Baseline in Cardiac Parameter: Left Ventricular Ejection Fraction (LVEF) at Month, 24, 36, 48, and 60
大体时间：Baseline, Month 24, Month 36, Month 48, and Month 60
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LVEF is a measurement of how much blood the left ventricle pumps out with each contraction. Change in LVEF from baseline as measured by Cardiac Magnetic Resonance (cMRI) |
Baseline, Month 24, Month 36, Month 48, and Month 60
|
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Change From Baseline in Cardiac Parameter: Left Ventricular End Systolic Volumes-Indexed (LV-ESVI) at Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 24, Month 36, Month 48, and Month 60
|
Change from baseline in LV-ESVI as assessed by Cardiac Magnetic Resonance (cMRI). |
Baseline, Month 24, Month 36, Month 48, and Month 60
|
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Change From Baseline in Cardiac Parameter: Left Ventricular End Diastolic Volumes-Indexed (LV-EDVI) at Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 24, Month 36, Month 48, and Month 60
|
Change from baseline in LV-EDVI as assessed by Cardiac Magnetic Resonance (cMRI. |
Baseline, Month 24, Month 36, Month 48, and Month 60
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Change From Baseline in Cardiac Parameter: Left Ventricle Mass (LV Mass) at Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 24, Month 36, Month 48, and Month 60
|
Change from baseline in LV mass was assessed by Cardiac Magnetic Resonance (cMRI). |
Baseline, Month 24, Month 36, Month 48, and Month 60
|
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Change From Baseline in Cardiac Parameter: Unindexed Volumes at Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 24, Month 36, Month 48, and Month 60
|
Change from baseline in unindexed volumes as assessed by Cardiac Magnetic Resonance (cMRI) |
Baseline, Month 24, Month 36, Month 48, and Month 60
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Change From Baseline in Cardiac Parameter: Left Ventricle End Diastolic Wall Thickening (LVEDWT) at Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 24, Month 36, Month 48, and Month 60
|
Change from baseline in LVEDWT was assessed by Cardiac Magnetic Resonance (cMRI). |
Baseline, Month 24, Month 36, Month 48, and Month 60
|
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Change From Baseline in Cardiac Parameter: Left Ventricle End Systolic Wall Thickening (LVESWT) at Month 24, Month 36, Month 48, and Month 60
大体时间：Baseline, Month 24, Month 36, Month 48, and Month 60
|
Change from baseline in LVESWT was assessed by Cardiac Magnetic Resonance (cMRI). |
Baseline, Month 24, Month 36, Month 48, and Month 60
|
合作者和调查者
调查人员
- 研究主任：Mark Awadalla、Capricor Inc.
- 首席研究员：Craig McDonald, MD、UC Davis
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- CAP-1002-DMD-02-OLE
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
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