NCT00116688 · CITED IN SOURCE DOCUMENTS

Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

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

Phase: PHASE3
Status at capture: COMPLETED
Registry last update: 2013-12-18

The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim. Participants must have previously completed a romiplostim ITP study.

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

Number of Participants With Adverse Events

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outcome · POSTED

Number of Participants With a Platelet Response

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outcome · POSTED

Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies

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outcome · POSTED

Change From Baseline in ITP Patient Assessment Questionnaire

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outcome · POSTED

Change From Baseline in Short Form 36 (SF-36)

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outcome · POSTED

Change From Baseline in Euroqol-5D (EQ-5D) Index Score

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outcome · POSTED

Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS)

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outcome · POSTED

Patient Global Assessment

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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: * Must have previously completed a romiplostim ITP study * Platelet count ≤ 50 x 10 \^9/L * Written informed consent Exclusion Criteria: * Bone marrow stem cell disorder or new active malignancies diagnosed since enrollment in the previous romiplostim ITP study * Received any alkylating agents within 4 weeks before screening visit or anticipated use during the time of the proposed study * Currently enrolled in or has not yet completed at least 4 weeks since ending device or drug trial(s) (other than the previous romiplostim ITP study), or subject is receiving other investigational agent(s) other than romiplostim * Not using adequate contraceptive precautions * Not available for follow-up assessments * Has any kind of disorder that compromises the ability of the participant to give informed consent and does not have a legally-acceptable representative and/or is unable to comply with study procedures.
healthy Volunteers: false
minimum Age: 1 Year
sex: ALL
std Ages: CHILD
ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

description
Romiplostim weekly subcutaneous dosing based on screening weight and platelet count. Starting dose of 1 µg/kg up to a maximum dose of 10 µg/kg.
intervention Names
Biological: Romiplostim
label
Romiplostim
type
EXPERIMENTAL

interventions

arm Group Labels
Romiplostim
description
Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.
name
Romiplostim
other Names
AMG 531
Nplate
type
BIOLOGICAL

Study design

allocation

RANDOMIZED

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 313
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Participants with one or more occurrences of one or more adverse events up to 8 weeks after the end of treatment. Participants with more than one event were only counted once.
measure
Number of Participants With Adverse Events
time Frame
Duration of treatment plus 8 weeks (up to 285 weeks)

secondary Outcomes

description
Platelet response was defined as having a platelet count of ≥ 50 x 10\^9/L at any time on study, excluding platelet counts within 8 weeks after receiving any rescue medications.
measure
Number of Participants With a Platelet Response
time Frame
Duration of treatment (up to 277 weeks)
description
The number of participants with a reduction or discontinuation of concurrent immune (idiopathic) thrombocytopenic purpura (ITP) therapies (corticosteroids, danazol, azathioprine) during the study.
measure
Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies
time Frame
Duration of treatment (up to 277 weeks)
description
The ITP Patient Assessment Questionnaire (ITP-PAQ) assesses ITP-specific health-related quality of life (HRQOL). This questionnaire assesses ITP specific health-related quality of life (HRQOL). The questionnaire consists of 44 items and has six domains: These domains assess the impact of ITP on Physical Health, Mental Health, Work, Social Activity, Women's Health and Overall QOL. The impact of ITP on Physical Health consists of four sub-scales, which evaluate ITP related Symptoms, Fatigue, Bother and Activity. The impact of ITP on Mental Health consists of two sub-scales, which evaluate Psychological distress and Fear in a population with ITP. Items are scored from 0-100 with higher scores indicating better HRQOL.
measure
Change From Baseline in ITP Patient Assessment Questionnaire
time Frame
Baseline to Week 48
description
The SF-36 is a widely used generic health-related quality of life measure. It has 36 questions with 8 domains: Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional and Mental Health. Items are scored from 0 to 100 with higher scores indicating better health status.
measure
Change From Baseline in Short Form 36 (SF-36)
time Frame
Baseline to Week 48
description
The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The instrument is applicable to a wide range of health conditions and treatments and results in a single index score and a visual analog scale (VAS) score. The EQ-5D descriptive health profile comprises five dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension comprises three levels (no problems, some/moderate problems, extreme problems). A unique EQ-5D health state is defined by combining one level from each of the five dimensions. EQ-5D index values range from -0.59 to 1.00. Higher EQ-5D Index scores represent better health status.
measure
Change From Baseline in Euroqol-5D (EQ-5D) Index Score
time Frame
Baseline to Week 48
description
The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The EQ-5D VAS records the respondent's self-rated health status on a vertical graduated (0-100) visual analogue scale. Higher EQ-5D VAS scores represent better health status.
measure
Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS)
time Frame
Baseline to Week 48
description
The Patient Global Assessment is two questions which assess the overall health-related quality of life (HRQOL) and symptoms of the patient. Each item is answered on a 15-point Likert scale ranging from 'A very great deal worse' (1) to 'A very great deal better' (15). A higher score indicates that quality of life or symptoms have improved.
measure
Patient Global Assessment
time Frame
Week 1 and Week 48

Full study description

brief Summary: The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim. Participants must have previously completed a romiplostim ITP study.

Source references

references

citation
Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3.
pmid
18981291
type
BACKGROUND
citation
Kuter DJ, Bussel JB, Newland A, Baker RI, Lyons RM, Wasser J, Viallard JF, Macik G, Rummel M, Nie K, Jun S. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013 May;161(3):411-23. doi: 10.1111/bjh.12260. Epub 2013 Feb 22.
pmid
23432528
type
BACKGROUND
citation
Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001500;
type
BACKGROUND
citation
Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001752;
type
BACKGROUND
citation
Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009 Oct 29;114(18):3748-56. doi: 10.1182/blood-2009-05-224766. Epub 2009 Aug 11.
pmid
19671919
type
DERIVED