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

NCT00515203 | Completed Phase 1 Results

• 1 other identifier: 20060195
• Study Type: interventional
• Target: 22
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

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

Keywords

Immune (Idiopathic) Thrombocytopenic Purpura; Pediatric Idiopathic Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Occurrence of one or more adverse events in the participant during the 12-week treatment period

  12 weeks

Secondary Outcomes (5)

• Weeks With Platelet Count ≥ 50 x 10^9/L

  12-week treatment period

• Bleeding Events (Grade 2 or Higher)

  12-week treatment period (Weeks 2 - 13)

• Platelet Count ≥ 50 x 10^9/L for Two Consecutive Weeks

  12-week treatment period

• Increase in Platelet Count ≥ 20 x 10^9/L Above Baseline for Two Consecutive Weeks

  12-week treatment period

• Requirement for Rescue Therapy (as Defined Per Protocol)

  12-week treatment period

Study Arms (2)

II.

PLACEBO COMPARATOR

5 thrombocytopenic (as defined per protocol) subjects

Drug: Placebo

I.

EXPERIMENTAL

15 thrombocytopenic (as defined per protocol) subjects

Drug: AMG 531

Interventions

Placebo DRUG

Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.

II.

AMG 531 DRUG

Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.

Also known as: romiplostim

I.

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Amgen: lead

Related Publications (2)

• Bussel JB, Buchanan GR, Nugent DJ, Gnarra DJ, Bomgaars LR, Blanchette VS, Wang YM, Nie K, Jun S. A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia. Blood. 2011 Jul 7;118(1):28-36. doi: 10.1182/blood-2010-10-313908. Epub 2011 Apr 18.

  PMID: 21502541 BACKGROUND

• Klaassen RJ, Mathias SD, Buchanan G, Bussel J, Deuson R, Young NL, Collier A, Bomgaars L, Blanchette V. Pilot study of the effect of romiplostim on child health-related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP). Pediatr Blood Cancer. 2012 Mar;58(3):395-8. doi: 10.1002/pbc.23312. Epub 2011 Sep 9.

  PMID: 21910213 BACKGROUND

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombocytopenic

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Amgen Inc.

866-572-6436

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 9, 2007
• First Posted: August 13, 2007
• Study Start: July 1, 2007
• Primary Completion: March 1, 2009
• Study Completion: August 1, 2009
• Last Updated: July 25, 2014
• Results First Posted: March 1, 2011

Record last verified: 2014-07