Safety and Efficacy Study of Romiplostim (AMG 531) to Treat ITP in Pediatric Subjects
This study has been completed.
Sponsor:
Amgen
Information provided by:
Amgen
ClinicalTrials.gov Identifier:
NCT00515203
First received: August 9, 2007
Last updated: August 11, 2011
Last verified: August 2011
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Purpose
The purpose of this study is to evaluate the safety and tolerability of romiplostim (AMG 531) in the treatment of thrombocytopenia in pediatric subjects with chronic ITP. We will also evaluate the efficacy of romiplostim (AMG 531) and characterize the pharmacokinetics of romiplostim (AMG 531). It is anticipated that romiplostim (AMG 531), when given at an effective dose and schedule, will be well tolerated treatment for thrombocytopenia among pediatric subjects with chronic ITP.
| Condition | Intervention | Phase |
|---|---|---|
|
Idiopathic Thrombocytopenic Purpura Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) |
Drug: Placebo Drug: AMG 531 |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | A Randomized, Double-Blind, Placebo-controlled Phase 1/2 Study to Determine the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Pediatric Subjects With Chronic Immune (Idiopathic) Thrombocytopenic Purpura |
Resource links provided by NLM:
Genetics Home Reference related topics:
thrombotic thrombocytopenic purpura
Drug Information available for:
Romiplostim
U.S. FDA Resources
Further study details as provided by Amgen:
Primary Outcome Measures:
- Adverse Events [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]Occurrence of one or more adverse events in the participant during the 12-week treatment period
Secondary Outcome Measures:
- Weeks With Platelet Count ≥ 50 x 10^9/L [ Time Frame: 12-week treatment period ] [ Designated as safety issue: No ]The number of weeks with platelet count ≥ 50 x 10^9/L during the 12 week treatment period.
- Bleeding Events (Grade 2 or Higher) [ Time Frame: 12-week treatment period (Weeks 2 - 13) ] [ Designated as safety issue: No ]Total number of bleeding events (Grade 2 or higher, i.e., mild to life-threatening, as defined in the protocol) for each participant during Weeks 2-13 (end-of-study visit for non-responders)
- Platelet Count ≥ 50 x 10^9/L for Two Consecutive Weeks [ Time Frame: 12-week treatment period ] [ Designated as safety issue: No ]Participant incidence of achieving a platelet count ≥50 x 10^9/L for two consecutive weeks during the 12 week treatment period.
- Increase in Platelet Count ≥ 20 x 10^9/L Above Baseline for Two Consecutive Weeks [ Time Frame: 12-week treatment period ] [ Designated as safety issue: No ]Participant incidence of achieving an increase in platelet count ≥20 x 10^9/L above baseline for two consecutive weeks during the 12 week treatment period.
- Requirement for Rescue Therapy (as Defined Per Protocol) [ Time Frame: 12-week treatment period ] [ Designated as safety issue: No ]Participant required rescue therapy (as defined per protocol) during the 12 week treatment period.
| Enrollment: | 22 |
| Study Start Date: | July 2007 |
| Study Completion Date: | August 2009 |
| Primary Completion Date: | March 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Placebo Comparator: II.
5 thrombocytopenic (as defined per protocol) subjects
|
Drug: Placebo
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.
|
|
Experimental: I.
15 thrombocytopenic (as defined per protocol) subjects
|
Drug: AMG 531
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.
Other Name: romiplostim
|
Eligibility| Ages Eligible for Study: | 12 Months to 17 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Before any study-specific procedure, the appropriate written informed consent must be obtained. In addition to the written informed consent, the assent of the child from those subjects capable of providing assent must also be obtained if requested by the IRB/IEC.
- Diagnosis of ITP according to The American Society of Hematology (ASH) Guidelines at least six months prior to screening
- Age ≥ 12 months and < 18 years at enrollment
- The mean of two platelet counts taken during the screening period must be ≤ 30 x 10^9/L with no single count >35 x 10^9/L
- A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category)
- Adequate liver function; serum bilirubin ≤ 1.5 times the laboratory normal range
- Hemoglobin >10.0 g/dL
Exclusion Criteria:
- Known history of a bone marrow stem cell disorder (any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study)
- Known history of venous or arterial thrombotic or thromboembolic event
- Known history of congenital thrombocytopenia
- Known history of malignancy except basal cell carcinoma
- Known history of hepatitis B, hepatitis C, or HIV
- Known history of systemic lupus erythematosus, Evans Syndrome, or autoimmune neutropenia
- Known positive lupus anticoagulant or history of antiphospholipid antibody syndrome
- Known history of Disseminated Intravascular Coagulation, Hemolytic Uremic Syndrome, or Thrombotic Thrombocytopenic Purpura
- Currently receiving any treatment for ITP except for corticosteroids
- IV Ig or anti-D Ig within two weeks prior to the screening visit
- Rituximab (for any indication) within 14 weeks before the screening visit or anticipated use during the time of the proposed study
- Splenectomy within eight weeks of the screening visit
- Received hematopoietic growth factors including IL-11 (oprelvekin) within four weeks before the screening visit
- Received any alkylating agents within eight weeks before the screening visit or anticipated use during the time of the proposed study
- Subject is currently enrolled in or has not yet completed at least four weeks since ending other investigational device or drug trial(s), or subject is receiving investigational agent(s)
- Past or present participation in any study evaluating PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO), AMG 531, or related platelet product
- Pregnant (i.e. positive urine pregnancy test) or breast feeding
- Subject is not using adequate contraceptive precautions, if applicable.
- Known hypersensitivity to any recombinant E coli-derived product
- Subject has any kind of disorder that compromises the ability to comply with all study procedures
Contacts and Locations More Information
Additional Information:
No publications provided by Amgen
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Global Development Leader, Amgen Inc. |
| ClinicalTrials.gov Identifier: | NCT00515203 History of Changes |
| Other Study ID Numbers: | 20060195 |
| Study First Received: | August 9, 2007 |
| Results First Received: | October 28, 2010 |
| Last Updated: | August 11, 2011 |
| Health Authority: | Australia: Therapeutic Goods Administration Canada: Health Canada France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Spain: Agencia Española de Medicamentos y Productos Sanitarios United States: Food and Drug Administration Australia: Department of Health and Ageing Therapeutic Goods Administration Australia: Human Research Ethics Committee United States: Institutional Review Board |
Keywords provided by Amgen:
|
Immune (Idiopathic) Thrombocytopenic Purpura Pediatric Idiopathic Thrombocytopenic Purpura |
Additional relevant MeSH terms:
|
Purpura Purpura, Thrombocytopenic Thrombocytopenia Purpura, Thrombocytopenic, Idiopathic Blood Coagulation Disorders Hematologic Diseases Hemorrhage Pathologic Processes |
Skin Manifestations Signs and Symptoms Thrombotic Microangiopathies Blood Platelet Disorders Immune System Diseases Hemorrhagic Disorders Autoimmune Diseases |
ClinicalTrials.gov processed this record on May 16, 2013