Safety and Efficacy Study of Romiplostim to Treat ITP in Pediatric Subjects

This study is currently recruiting participants.
Verified January 2014 by Amgen
Sponsor:
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT01444417
First received: September 29, 2011
Last updated: January 29, 2014
Last verified: January 2014
  Purpose

The purpose of this study is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric subjects with Immune Thrombocytopenia Purpura (ITP) as measured by durable platelet response.


Condition Intervention Phase
Idiopathic Thrombocytopenic Purpura
Thrombocytopenia
Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenic Purpura
Drug: romiplostim
Drug: Placebo
Phase 3

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 Phase 3 Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)

Resource links provided by NLM:


Further study details as provided by Amgen:

Primary Outcome Measures:
  • incidence of durable platelet response defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10^9/L during weeks 18 through 25 of treatment [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • incidence of overall platelet response defined as subjects who achieve a platelet count ≥ 50 x 10^9/L at a minimum of 4 times during weeks 2 to 25 of the treatment period [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • number of weekly platelet counts ≥ 50 x 10^9/L during weeks 2 to 25 of the treatment period [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • incidence of rescue ITP medications used [ Time Frame: 25 weeks ] [ Designated as safety issue: No ]
  • number of composite bleeding episodes defined as clinically significant bleeding events or the use of a rescue medication to prevent a clinically significant bleeding event during weeks 2 to 25 of the treatment period [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
  • incidence of adverse events, including thromboembolic events and hematologic malignancies, clinically significant changes in laboratory values and the incidence of antibody formation [ Time Frame: 25 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 60
Study Start Date: December 2011
Estimated Study Completion Date: April 2015
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AMG 531
AMG 531
Drug: romiplostim
The starting dose of romiplostim is 1 µg/kg administered weekly by subcutaneous injection. Subjects will return to the clinic weekly to provide platelet counts and undergo dose titrations under the supervision of the treating physician. Weekly dose increases will continue in increments of 1 µg/kg up to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. Dose adjustment will be allowed during the treatment period to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L.
Placebo Comparator: Placebo
Placebo Comparator
Drug: Placebo
The starting dose is 1 µg/kg administered weekly by subcutaneous injection. Subjects will return to the clinic weekly to provide platelet counts and undergo dose titrations under the supervision of the treating physician. Weekly dose increases will continue in increments of 1 µg/kg up to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. Dose adjustment will be allowed during the treatment period to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L.

  Eligibility

Ages Eligible for Study:   1 Year to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of primary ITP according to the ASH Guidelines at least 6 months prior to screening, regardless of splenectomy status
  • Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies; prior therapy includes first-line therapies
  • Age ≥ 1 year and < 18 years at the time of providing informed consent
  • The mean of 2 platelet counts taken during the screening period must be ≤ 30 x 10^9/L with neither count > 35 x 10^9/L
  • A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period
  • Adequate liver function; serum bilirubin ≤ 1.5 times the laboratory normal range during the screening period;AST and ALT ≤ 3.0 times the laboratory normal range during the screening period
  • Hemoglobin > 10.0 g/dL during the screening period
  • Subject and/or subject's legally acceptable representative has provided informed consent prior to any study-specific procedure; subject has provided assent, where required

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 active or prior malignancy except adequately treated basal cell carcinoma
  • Known history of congenital thrombocytopenia
  • Known history of hepatitis B, hepatitis C, or HIV
  • Known history of H. pylori by urea breath test or stool antigen test within 6 months of enrollment or successfully treated with no evidence of infection
  • Known history of systemic lupus erythematosus, evans syndrome, or autoimmune neutropenia
  • Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant
  • Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
  • Previous history of venous thromboembolism or thrombotic events
  • Previous use of romiplostim, PEG-rHuMGDF, Eltrombopag, rHuTPO or any platelet producing agent
  • Rituximab (for any indication) or 6-MP within 14 weeks before the screening visit, or anticipated use during the time of the proposed study
  • Splenectomy within 4 weeks of the screening visit
  • All hematopoietic growth factors including IL-11 (oprelvekin) within 4 weeks before the screening visit
  • Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
  • Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
  • Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
  • Other criteria may apply
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01444417

Contacts
Contact: Amgen Call Center 866-572-6436

  Show 33 Study Locations
Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen
  More Information

Additional Information:
No publications provided

Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01444417     History of Changes
Other Study ID Numbers: 20080279, 2010-018426-39
Study First Received: September 29, 2011
Last Updated: January 29, 2014
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Australia: Human Research Ethics Committee
Australia: Therapeutic Goods Administration
Canada: Health Canada
Canada: Institutional Review Board
United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Amgen:
Immune (Idiopathic) Thrombocytopenic Purpura
Pediatric Immune (Idiopathic) Thrombocytopenic Purpura

Additional relevant MeSH terms:
Thrombocytopenia
Purpura
Purpura, Thrombocytopenic
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 April 15, 2014