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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT00116688
First received: June 30, 2005
Last updated: November 25, 2013
Last verified: November 2013
  Purpose

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.


Condition Intervention Phase
Thrombocytopenia
Idiopathic Thrombocytopenic Purpura
Biological: Romiplostim
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of AMG 531 in Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Resource links provided by NLM:


Further study details as provided by Amgen:

Primary Outcome Measures:
  • Number of Participants With Adverse Events [ Time Frame: Duration of treatment plus 8 weeks (up to 285 weeks) ] [ Designated as safety issue: Yes ]
    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.


Secondary Outcome Measures:
  • Number of Participants With a Platelet Response [ Time Frame: Duration of treatment (up to 277 weeks) ] [ Designated as safety issue: No ]
    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.

  • Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies [ Time Frame: Duration of treatment (up to 277 weeks) ] [ Designated as safety issue: No ]
    The number of participants with a reduction or discontinuation of concurrent immune (idiopathic) thrombocytopenic purpura (ITP) therapies (corticosteroids, danazol, azathioprine) during the study.

  • Change From Baseline in ITP Patient Assessment Questionnaire [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Short Form 36 (SF-36) [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Euroqol-5D (EQ-5D) Index Score [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]
    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.

  • Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS) [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]
    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.

  • Patient Global Assessment [ Time Frame: Week 1 and Week 48 ] [ Designated as safety issue: No ]
    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.


Enrollment: 313
Study Start Date: August 2004
Study Completion Date: January 2010
Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Romiplostim
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.
Biological: Romiplostim
Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.
Other Names:
  • AMG 531
  • Nplate

  Eligibility

Ages Eligible for Study:   1 Year and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00116688

Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen
  More Information

Additional Information:
Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT00116688     History of Changes
Other Study ID Numbers: 20030213
Study First Received: June 30, 2005
Results First Received: March 4, 2011
Last Updated: November 25, 2013
Health Authority: Belgium: Federal Agency for Medicines and Health Products, FAMHP
Belgium: Directorate-General for Medicinal Products
Belgium: Federal Public Service (FPS) Health, Food Chain Safety and Environment
Belgium: FPS of Public Health, Food Chain Security and Environment
Belgium: Pharmaceutical Inspectorate
Belgium: Service Public Federal Sante Publiquest, Securite de la Chaine alimentaire et Environnement
Belgium: Service Public Fédéral Santé Publique, Sécurité de la Chaîne alimentaire et Environnement
Canada: Health Canada
Canada: Health Products and Food Branch
Canada: Institutional Review Board
Czech Republic: State Institute for Drug Control
Czech Republic: Statni ustav pro kontrolu leciv
EU: CHMP
European Union: European Medicines Agency
France and Sweden: European Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
France: CCPPRB Central Ethics Committee
France: Ministry of Health
Germany: Federal Institute for Drugs and Medical Devices
Germany: Paul_Ehrlich-Institut Bundesamt fur Sera und Impfstoffe
Italy: Local Ethics Committees
Italy: Ministry of Health
Netherlands: CCMO (Centrale Commissie Mensgebonden Onderzoek): Central Committee Human Bound Research
Netherlands: Medicines Evaluation Board
Netherlands: Medisch Centrum Rijnmond_Zuid, lcatie Zuider
Austria: Central Ethics Committee
Austria: Competant Authority
Austria: Federal Ministry for Health and Women
Austria: Secretariat of Health
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Spain: Spanish Agency of Medicines
Spain: Spanish Drug Agency
Switzerland: Agency for Therapeutic Products
Switzerland: Local Ethics Committee
Switzerland: Swissmedic (Swiss Agency for Therapeutic Products)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Poland: Drug Institut
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
Australia: Human Research Ethics Committee
Australia: Therapeutic Goods Administration
Austria: AGES - PharmaMed Austria Institut Wissenschaft & Information
Austria: Bundesamt fur Sicherheit im Gesundheitswesen
Austria: Bundesamt für Sicherheit im Gesundheitswesen

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

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 April 23, 2014