Phase 2
N=45
Evaluating the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
Idiopathic Thrombocytopenic Purpura
Bottom Line
View on ClinicalTrials.gov: NCT00111475 ↗Enrolled (actual)
45
Serious AEs
13.3%
Results posted
Jan 2020
Primary outcome: Primary: Number of Participants With Adverse Events — 4; 4; 4; 4 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Romiplostim (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Jun 2004
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events |
4; 4; 4; 4; 4; 4 | — |
| PRIMARY Number of Participants With Anti-romiplostim or Anti-endogenous Thrombopoietin Neutralizing Antibodies |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants Who Achieved Targeted Therapeutic Platelet Level in Part A |
1; 0; 0; 1; 1; 1 | — |
| SECONDARY Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part A |
1; 1; 1; 2; 2; 3 | — |
| SECONDARY Number of Participants With a Peak Platelet Count ≥ 100 x 10⁹ Cells/L in Part A |
1; 0; 0; 1; 2; 3 | — |
| SECONDARY Number of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part A |
0; 0; 0; 0; 1; 2 | — |
| SECONDARY Peak Platelet Count After Each Dose in Part A |
123.0; 163.0; 309.0; 746.3; 65.0; 140.5 | — |
| SECONDARY Change From Baseline in Peak Platelet Count After Each Dose in Part A |
109.5; 150.2; 289.3; 725.5; 51.50; 122.6 | — |
| SECONDARY Time to Peak Platelet Count After Each Dose in Part A |
10.0; 11.0; 9.5; 15.0; 10.0; 11.0 | — |
| SECONDARY Duration Within the Targeted Therapeutic Platelet Range In Part A |
5.0; 8.0; 11.0; 10.0; 3.0; 3.5 | — |
| SECONDARY Percentage of Participants Who Achieved Targeted Therapeutic Platelet Level In Part B |
25; 88; 38; 0 | — |
| SECONDARY Percentage of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part B |
50; 100; 75; 100 | — |
| SECONDARY Percentage of Participants With a Peak Platelet Count of ≥ 100 x 10⁹ Cells/L in Part B |
25; 63; 63; 100 | — |
| SECONDARY Percentage of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part B |
0; 0; 25; 100 | — |
| SECONDARY Percentage of Participants With a Peak Platelet Count of > 500 x 10⁹ Cells/L in Part B |
0; 0; 25; 100 | — |
| SECONDARY Peak Platelet Count in Part B |
80.8; 134.5; 240.9; 520.0 | — |
| SECONDARY Change From Baseline in Peak Platelet Count in Part B |
52.4; 117.6; 226.9; 505.0 | — |
| SECONDARY Time to Peak Platelet Count in Part B |
63; 18; 19; 21 | — |
| SECONDARY Duration Within the Targeted Therapeutic Platelet Range in Part B |
6.0; 3.0; 5.0; 4.0 | — |
Summary
The primary objective of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with ITP.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00111475). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.