Safety and Efficacy Study of Romiplostim (AMG 531) to Treat ITP in Pediatric Subjects

Amgen

Status and phase

Completed

Phase 2

Phase 1

Conditions

Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Idiopathic Thrombocytopenic Purpura

Treatments

Drug: AMG 531

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT00515203

20060195

Details and patient eligibility

About

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.

Enrollment

22 patients

Sex

All

Ages

1 to 17 years old

Volunteers

No Healthy Volunteers

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