NCT00515203 · CITED IN SOURCE DOCUMENTS

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

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase: PHASE1, PHASE2
Status at capture: COMPLETED
Registry last update: 2014-07-25

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

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

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outcome · POSTED

Bleeding Events (Grade 2 or Higher)

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outcome · POSTED

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

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outcome · POSTED

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

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outcome · POSTED

Requirement for Rescue Therapy (as Defined Per Protocol)

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outcome · POSTED

Adverse Events

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

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
healthy Volunteers: false
maximum Age: 17 Years
minimum Age: 1 Year
sex: ALL
std Ages: CHILD

Treatment arms and interventions

arm Groups

description
5 thrombocytopenic (as defined per protocol) subjects
intervention Names
Drug: Placebo
label
II.
type
PLACEBO_COMPARATOR
description
15 thrombocytopenic (as defined per protocol) subjects
intervention Names
Drug: AMG 531
label
I.
type
EXPERIMENTAL

interventions

arm Group Labels
II.
description
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.
name
Placebo
type
DRUG
arm Group Labels
I.
description
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.
name
AMG 531
other Names
romiplostim
type
DRUG

Study design

allocation

RANDOMIZED

intervention Model

PARALLEL

masking Info

masking: TRIPLE
who Masked: PARTICIPANT
INVESTIGATOR
OUTCOMES_ASSESSOR

primary Purpose

TREATMENT

Enrollment

count: 22
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Occurrence of one or more adverse events in the participant during the 12-week treatment period
measure
Adverse Events
time Frame
12 weeks

secondary Outcomes

description
The number of weeks with platelet count ≥ 50 x 10\^9/L during the 12 week treatment period.
measure
Weeks With Platelet Count ≥ 50 x 10^9/L
time Frame
12-week treatment period
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)
measure
Bleeding Events (Grade 2 or Higher)
time Frame
12-week treatment period (Weeks 2 - 13)
description
Participant incidence of achieving a platelet count ≥50 x 10\^9/L for two consecutive weeks during the 12 week treatment period.
measure
Platelet Count ≥ 50 x 10^9/L for Two Consecutive Weeks
time Frame
12-week treatment period
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.
measure
Increase in Platelet Count ≥ 20 x 10^9/L Above Baseline for Two Consecutive Weeks
time Frame
12-week treatment period
description
Participant required rescue therapy (as defined per protocol) during the 12 week treatment period.
measure
Requirement for Rescue Therapy (as Defined Per Protocol)
time Frame
12-week treatment period

Full 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.

Source references

references

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.
pmid
21502541
type
BACKGROUND
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.
pmid
21910213
type
BACKGROUND