Safety and Efficacy Study of Romiplostim to Treat ITP in Pediatric Subjects - Full Text View

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:

Genetics Home Reference related topics: thrombotic thrombocytopenic purpura

Drug Information available for: Romiplostim

U.S. FDA Resources

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 2014
Estimated Primary Completion Date:	October 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 40 thrombocytopenic (as defined per protocol) subjects	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: 20 thrombocytopenic (as defined per protocol) subjects	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.

Arms

Assigned Interventions

Experimental: 40 thrombocytopenic (as defined per protocol) subjects

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: 20 thrombocytopenic (as defined per protocol) subjects

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

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Sponsors and Collaborators

Amgen

Investigators

Study Director:

MD

Amgen

More Information

Additional Information:

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

No publications provided

Responsible Party:	Amgen
ClinicalTrials.gov Identifier:	NCT01444417 History of Changes
Other Study ID Numbers:	20080279
Study First Received:	September 29, 2011
Last Updated:	May 6, 2013
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:

Purpura
Purpura, Thrombocytopenic
Thrombocytopenia
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 May 21, 2013