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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00515203
• Date of registration: 09/08/2007
• Prospective Registration: No
• Primary sponsor: Amgen
• Public title: Safety and Efficacy Study of Romiplostim (AMG 531) to Treat ITP in Pediatric Subjects
• Scientific 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
• Date of first enrolment: July 2007
• Target sample size: 22
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00515203
• Study type: Interventional
• Phase: Phase 1/Phase 2

Countries of recruitment

Australia, Canada, Spain, United States

Contacts

• Name: MD
• Affiliation: Amgen

Key inclusion & exclusion 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



Age minimum: 1 Year
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

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

Idiopathic Thrombocytopenic Purpura

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

Intervention(s)

Drug: AMG 531

Drug: Placebo

Primary Outcome(s)

Adverse Events [Time Frame: 12 weeks]

Secondary Outcome(s)

Requirement for Rescue Therapy (as Defined Per Protocol) [Time Frame: 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]

Weeks With Platelet Count = 50 x 10^9/L [Time Frame: 12-week treatment period]

Bleeding Events (Grade 2 or Higher) [Time Frame: 12-week treatment period (Weeks 2 - 13)]

Platelet Count = 50 x 10^9/L for Two Consecutive Weeks [Time Frame: 12-week treatment period]

Secondary ID(s)

20060195

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/03/2011
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00515203