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Safety and Efficacy Study of AMG 531 to Treat ITP in Pediatric Subjects
This study is ongoing, but not recruiting participants.
Sponsored by: Amgen
Information provided by: Amgen
ClinicalTrials.gov Identifier: NCT00515203
  Purpose

The purpose of this study is to evaluate the safety and tolerability of AMG 531 in the treatment of thrombocytopenia in pediatric subjects with chronic ITP. We will also evaluate the efficacy of AMG 531 and characterize the pharmacokinetics of AMG 531. It is anticipated that 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: AMG 531
Drug: Placebo
Phase I
Phase II

Genetics Home Reference related topics: hemophilia thrombotic thrombocytopenic purpura
Drug Information available for: AMG 531
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study
Official Title: A Randomized, Double-Blind, Placebo-Controlled Phase 1/2 Study to Determine the Safety and Efficacy of AMG 531 in Thrombocytopenic Pediatric Subjects With Chronic Immune (Idiopathic) Thrombocytopenic Purpura

Further study details as provided by Amgen:

Primary Outcome Measures:
  • The incidence of adverse events, including anti-AMG 531 antibody formation and cross-reacting anti-TPO antibody formation, by treatment group during the 12 week treatment period of the study. [ Time Frame: 12 week treatment period ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetic exposure of AMG 531 as measured by drug concentrations taken at specified times during the study. [ Time Frame: At Week 12 for all subjects and Week 13-16 for responders as defined per protocol. ] [ Designated as safety issue: Yes ]
  • The number of weeks with platelet count ≥50 x 10^9/L during the 12 week treatment period. [ Time Frame: 12 week treatment period ] [ Designated as safety issue: No ]
  • The total number of bleeding events (Grade 2 or higher as per protocol) for each subject during Weeks 2-13 (end-of-study visit for non-responders). [ Time Frame: 12 week treatment period plus one week ] [ Designated as safety issue: No ]
  • The subject incidence of achieving a platelet count ≥50 x 10^9/L for two consecutive weeks during the 12 week treatment period. [ Time Frame: 12 week treatment period ] [ Designated as safety issue: No ]
  • The subject 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. [ Time Frame: 12 week treatment period ] [ Designated as safety issue: No ]
  • The subject incidence of requiring rescue therapy (as defined per protocol) during the 12 week treatment period. [ Time Frame: 12 week treatment period ] [ Designated as safety issue: No ]

Enrollment: 22
Study Start Date: July 2007
Estimated Study Completion Date: June 2009
Estimated Primary Completion Date: March 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
II.: Placebo Comparator
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.
I.: Experimental
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.

  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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00515203

Locations
United States, Arkansas
Research Site
Little Rock, Arkansas, United States
United States, California
Research Site
San Diego, California, United States
Research Site
Orange, California, United States
United States, Massachusetts
Research Site
Boston, Massachusetts, United States
United States, Michigan
Research Site
Detroit, Michigan, United States
United States, Nebraska
Research Site
Omaha, Nebraska, United States
United States, New York
Research Site
New York, New York, United States
United States, Pennsylvania
Research Site
Philadelphia, Pennsylvania, United States
United States, South Carolina
Research Site
Charleston, South Carolina, United States
United States, Tennessee
Research Site
Memphis, Tennessee, United States
Research Site
Nashville, Tennessee, United States
United States, Texas
Research Site
Houston, Texas, United States
Research Site
Dallas, Texas, United States
Australia
Research Site
Herston, Australia
Canada, Ontario
Research Site
Ottawa, Ontario, Canada
Spain
Research Site
Barcelona, Spain
Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen
  More Information

AmgenTrials clinical trials website  This link exits the ClinicalTrials.gov site

Responsible Party: Amgen Inc. ( Global Development Leader )
Study ID Numbers: 20060195
Study First Received: August 9, 2007
Last Updated: January 6, 2009
ClinicalTrials.gov Identifier: NCT00515203  
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration;   Australia: Human Research Ethics Committee;   Australia: Therapeutic Goods Administration;   Canada: Health Canada;   France: Afssaps - French Health Products Safety Agency;   Spain: Agencia Española de Medicamentos y Productos Sanitarios;   United States: Food and Drug Administration;   United States: Institutional Review Board

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

Study placed in the following topic categories:
Purpura
Autoimmune Diseases
Hematologic Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Hemostatic Disorders
Purpura, Thrombocytopenic
Signs and Symptoms
Thrombocytopathy
Thrombocytopenia
Hemorrhagic Disorders
Thrombocytopenic purpura, autoimmune
Purpura, Thrombocytopenic, Idiopathic

Additional relevant MeSH terms:
Skin Manifestations
Immune System Diseases

ClinicalTrials.gov processed this record on January 16, 2009