- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT00111475
Evaluating the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
A Dose-finding Study Evaluating the Safety and Efficacy of AMG 531 in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
연구 개요
연구 유형
등록 (실제)
단계
- 2 단계
참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Diagnosis of ITP according to American Society of Hematology (ASH) guidelines at least 3 months before enrollment
- Have completed at least 1 prior treatment for ITP
Two (including day -2) of the 3 platelet counts taken during the screening and pre-treatment periods must have fulfilled the following:
- less than 30 x 10^9/L for those subjects not receiving any ITP therapy,
- less than 50 x 10^9/L for those subjects receiving any ITP therapy
- Eastern Cooperative Oncology Group performance status of 0 to 2
- Serum creatinine concentration ≤ 2 mg/dL (≤ 176.8 µmol/L)
- Adequate liver function, as evidenced by a serum bilirubin ≤ 1.5 times the laboratory normal range
- Hemoglobin greater than 10.0 g/dL
- Written informed consent
Exclusion Criteria:
- Considered a substantial risk for adverse outcomes because of a clinically important trend (as determined by the investigator) detected in the platelet counts during the screening period
- Any known history of bone marrow stem cell disorder
- Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years before randomization
- Documented diagnosis of arterial thrombosis (ie, stroke, transient ischemic attack, or myocardial infarction) in the previous year; history of venous thrombosis (ie, deep vein thrombosis, pulmonary embolism) and receiving anticoagulation therapy
- Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure [New York Heart Association (NYHA) greater than class II], uncontrolled hypertension [diastolic greater than 100 mmHg] or cardiac arrhythmia)
- Have 3 or more of the following predisposing factors for thromboembolic events: diabetes; smoker using oral contraceptives; hypercholesteremia (> 240 mg/dL); treatment for hypertension
- Known positive test for human immunodeficiency virus (HIV) infection or hepatitis C virus
- Received any treatment for ITP (except for a constant dose schedule of corticosteroids) within 4 weeks before the screening visit
- Received intravenous (IV) immunoglobulin (Ig) or WinRho within 2 weeks before the screening visit
- Received hematopoietic growth factors, including interleukin (IL)-11 (Neumega®) within 4 weeks before the screening visit
- Past or present participation in any study evaluating polyethylene glycol recombinant human magakaryopoiesis differentiating factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), or related platelet product
- Received any alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
- Received any monoclonal antibody (eg, rituximab) within 16 weeks before the screening visit or anticipated use during the time of the proposed study
- Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication before the screening period
- Less than 2 months since major surgery (including laparoscopic splenectomy)
- Pregnant or breast feeding
- Subjects of reproductive potential who are not using adequate contraceptive precautions, in the judgment of the investigator
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 순차적 할당
- 마스킹: 더블
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: Part A: Romiplostim 0.2 µg/kg
Participants received 0.2 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
|
Administered by subcutaneous injection
다른 이름들:
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실험적: Part A: Romiplostim 0.5 µg/kg
Participants received 0.5 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
|
Administered by subcutaneous injection
다른 이름들:
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실험적: Part A: Romiplostim 1.0 µg/kg
Participants received 1.0 µg/kg romiplostim subcutaneously on day 1 and on day 15 or 22 depending on platelet counts.
|
Administered by subcutaneous injection
다른 이름들:
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실험적: Part A: Romiplostim 3 µg/kg
Participants received 3.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
|
Administered by subcutaneous injection
다른 이름들:
|
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실험적: Part A: Romiplostim 6 µg/kg
Participants received 6.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
|
Administered by subcutaneous injection
다른 이름들:
|
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실험적: Part A: Romiplostim 10 µg/kg
Participants received 10.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
|
Administered by subcutaneous injection
다른 이름들:
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위약 비교기: Part B: Placebo
Participants received placebo subcutaneously once a week for 6 weeks.
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피하 주사로 투여
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실험적: Part B: Romiplostim 1.0 µg/kg
Participants received 1.0 µg/kg subcutaneously once a week for 6 weeks.
|
Administered by subcutaneous injection
다른 이름들:
|
|
실험적: Part B: Romiplostim 3.0 µg/kg
Participants received 3.0 µg/kg subcutaneously once a week for 6 weeks.
|
Administered by subcutaneous injection
다른 이름들:
|
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실험적: Part B: Romiplostim 6.0 µg/kg
Participants received 6.0 µg/kg subcutaneously once a week for 6 weeks.
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Administered by subcutaneous injection
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Number of Participants With Adverse Events
기간: From first dose of study drug through 8 weeks (Part A) or 6 weeks (Part B) after last dose of study drug; 78 days
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From first dose of study drug through 8 weeks (Part A) or 6 weeks (Part B) after last dose of study drug; 78 days
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Number of Participants With Anti-romiplostim or Anti-endogenous Thrombopoietin Neutralizing Antibodies
기간: Assessed on day 29 (Part A only), day 43 (Part B only), and day 78
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The development of antibodies to romiplostim or to endogenous thrombopoietin (eTPO) was assessed using a neutralizing bioassay.
Participants positive for neutralizing antibodies at any of the assessments during the study are reported.
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Assessed on day 29 (Part A only), day 43 (Part B only), and day 78
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Number of Participants Who Achieved Targeted Therapeutic Platelet Level in Part A
기간: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
|
Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and between 50 to 450 x 10⁹ cells/L. Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. |
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part A
기간: After first dose (day 1 to day 15 or 22), and after second dose (day 15 or 22 to day 78)
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Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
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After first dose (day 1 to day 15 or 22), and after second dose (day 15 or 22 to day 78)
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Number of Participants With a Peak Platelet Count ≥ 100 x 10⁹ Cells/L in Part A
기간: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
|
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
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After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Number of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part A
기간: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
|
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
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After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Peak Platelet Count After Each Dose in Part A
기간: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
|
Platelet count data after the use of rescue medication were not included.
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After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Change From Baseline in Peak Platelet Count After Each Dose in Part A
기간: Baseline and after first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Platelet count data after the use of rescue medication were not included.
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Baseline and after first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
|
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Time to Peak Platelet Count After Each Dose in Part A
기간: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Platelet count data after the use of rescue medication were not included.
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After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Duration Within the Targeted Therapeutic Platelet Range In Part A
기간: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
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Targeted therapeutic platelet level was defined as a platelet count that was double the baseline level and ≥ 50 and ≤ 450 × 10⁹ cells/L. Platelet count data after the use of rescue medication were not included. |
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
|
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Percentage of Participants Who Achieved Targeted Therapeutic Platelet Level In Part B
기간: Day 1 to day 78
|
Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and within the range of greater than or equal to 50 x 10⁹ cells/L and less than or equal to 450 x 10⁹ cells/L. Platelet count data after use of rescue medication were not included in the analysis. |
Day 1 to day 78
|
|
Percentage of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part B
기간: Day 1 to day 78
|
Platelet count data after administration of rescue medication were not included in the analysis.
Participants with no platelet count data were considered non-responders.
|
Day 1 to day 78
|
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Percentage of Participants With a Peak Platelet Count of ≥ 100 x 10⁹ Cells/L in Part B
기간: Day 1 to day 78
|
Platelet count data after administration of rescue medication were not included in the analysis.
Participants with no platelet count data were considered non-responders.
|
Day 1 to day 78
|
|
Percentage of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part B
기간: Day 1 to day 78
|
Platelet count data after administration of rescue medication were not included in the analysis.
Participants with no platelet count data were considered non-responders.
|
Day 1 to day 78
|
|
Percentage of Participants With a Peak Platelet Count of > 500 x 10⁹ Cells/L in Part B
기간: Day 1 to day 78
|
Platelet count data after administration of rescue medication were not included in the analysis.
Participants with no platelet count data were considered non-responders.
|
Day 1 to day 78
|
|
Peak Platelet Count in Part B
기간: Day 1 to day 78
|
Platelet count data after administration of rescue medication were not included in the analysis.
|
Day 1 to day 78
|
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Change From Baseline in Peak Platelet Count in Part B
기간: Baseline and day 1 to day 78
|
Platelet count data after administration of rescue medication were not included in the analysis.
|
Baseline and day 1 to day 78
|
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Time to Peak Platelet Count in Part B
기간: Day 1 to day 78
|
Platelet count data after administration of rescue medication were not included in the analysis.
Time to peak platelet count was analyzed using the Kaplan-Meier method.
|
Day 1 to day 78
|
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Duration Within the Targeted Therapeutic Platelet Range in Part B
기간: Day 1 to day 78
|
Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and within the range of greater than or equal to 50 × 10⁹ cells/L and less than or equal to 450 × 10⁹ cells/L. Platelet count data after administration of rescue medication were not included in the analysis. |
Day 1 to day 78
|
공동 작업자 및 조사자
스폰서
간행물 및 유용한 링크
일반 간행물
- Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, Lyons R, Garcia-Chavez J, Carpenter N, Wang X, Eisen M. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017 Aug;102(8):1342-1351. doi: 10.3324/haematol.2016.161968. Epub 2017 Apr 14.
- Bussel JB, Kuter DJ, George JN, McMillan R, Aledort LM, Conklin GT, Lichtin AE, Lyons RM, Nieva J, Wasser JS, Wiznitzer I, Kelly R, Chen CF, Nichol JL. AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP. N Engl J Med. 2006 Oct 19;355(16):1672-81. doi: 10.1056/NEJMoa054626. Erratum In: N Engl J Med. 2006 Nov 9;355(19):2054.
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (추정)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- 20000137
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