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

Primary Purpose

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

Status

Completed

Phase

Phase 1

Locations

Study Type

Interventional

Intervention

Placebo

AMG 531

About this trial

This is an interventional treatment trial for Idiopathic Thrombocytopenic Purpura focused on measuring Immune (Idiopathic) Thrombocytopenic Purpura, Pediatric Idiopathic Thrombocytopenic Purpura

Eligibility Criteria

1 Year - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

II.

Arm Description

5 thrombocytopenic (as defined per protocol) subjects

15 thrombocytopenic (as defined per protocol) subjects

Outcomes

Primary Outcome Measures

Adverse Events

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

Secondary Outcome Measures

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

The number of weeks with platelet count ≥ 50 x 10^9/L during the 12 week treatment period.

Bleeding Events (Grade 2 or Higher)

Total number of bleeding events (Grade 2 or higher, i.e., mild to life-threatening, as defined in the protocol) for each participant during Weeks 2-13 (end-of-study visit for non-responders)

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

Participant incidence of achieving a platelet count ≥50 x 10^9/L for two consecutive weeks during the 12 week treatment period.

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

Participant 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.

Requirement for Rescue Therapy (as Defined Per Protocol)

Participant required rescue therapy (as defined per protocol) during the 12 week treatment period.

Full Information

NCT ID

NCT00515203

First Posted

August 9, 2007

Last Updated

July 18, 2014

Sponsor

Amgen

1. Study Identification

Unique Protocol Identification Number

NCT00515203

Brief Title

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

Official 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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2014

Overall Recruitment Status

Completed

Study Start Date

July 2007 (undefined)

Primary Completion Date

March 2009 (Actual)

Study Completion Date

August 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Amgen

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

22 (Actual)

8. Arms, Groups, and Interventions

Arm Title

II.

Arm Type

Placebo Comparator

Arm Description

5 thrombocytopenic (as defined per protocol) subjects

Arm Title

Arm Type

Experimental

Arm Description

15 thrombocytopenic (as defined per protocol) subjects

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

AMG 531

Other Intervention Name(s)

romiplostim

Intervention Description

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

Primary Outcome Measure Information:

Title

Adverse Events

Description

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

Time Frame

12 weeks

Secondary Outcome Measure Information:

Title

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

Description

The number of weeks with platelet count ≥ 50 x 10^9/L during the 12 week treatment period.

Time Frame

12-week treatment period

Title

Bleeding Events (Grade 2 or Higher)

Description

Total number of bleeding events (Grade 2 or higher, i.e., mild to life-threatening, as defined in the protocol) for each participant during Weeks 2-13 (end-of-study visit for non-responders)

Time Frame

12-week treatment period (Weeks 2 - 13)

Title

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

Description

Participant 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

Title

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

Description

Participant 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

Title

Requirement for Rescue Therapy (as Defined Per Protocol)

Description

Participant required rescue therapy (as defined per protocol) during the 12 week treatment period.

Time Frame

12-week treatment period

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Organizational Affiliation

Amgen

Official's Role

Study Director

12. IPD Sharing Statement

Citations:

PubMed Identifier

21502541

Citation

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.

Results Reference

background

PubMed Identifier

21910213

Citation

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.

Results Reference

background

Links:

URL

http://www.amgentrials.com

Description

AmgenTrials clinical trials website

Learn more about this trial

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

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