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

Thrombocytopenia Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00116688
• Other study ID #: 20030213
• Status: Completed
• Phase: Phase 3
• First received: June 30, 2005
• Last updated: November 25, 2013
• Start date: August 2004
• Est. completion date: January 2010

Study information

• Verified date: November 2013
• Source: Amgen
• Contact: n/a
• Is FDA regulated: No
• Health authority: Belgium: Federal Agency for Medicines and Health Products, FAMHPBelgium: Directorate-General for Medicinal ProductsBelgium: Federal Public Service (FPS) Health, Food Chain Safety and EnvironmentBelgium: FPS of Public Health, Food Chain Security and EnvironmentBelgium: Pharmaceutical InspectorateBelgium: Service Public Federal Sante Publiquest, Securite de la Chaine alimentaire et EnvironnementBelgium: Service Public Fédéral Santé Publique, Sécurité de la Chaîne alimentaire et EnvironnementCanada: Health CanadaCanada: Health Products and Food BranchCanada: Institutional Review BoardCzech Republic: State Institute for Drug ControlCzech Republic: Statni ustav pro kontrolu lecivEU: CHMPEuropean Union: European Medicines AgencyFrance and Sweden: European Medicines AgencyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)France: CCPPRB Central Ethics CommitteeFrance: Ministry of HealthGermany: Federal Institute for Drugs and Medical DevicesGermany: Paul_Ehrlich-Institut Bundesamt fur Sera und ImpfstoffeItaly: Local Ethics CommitteesItaly: Ministry of HealthNetherlands: CCMO (Centrale Commissie Mensgebonden Onderzoek): Central Committee Human Bound ResearchNetherlands: Medicines Evaluation BoardNetherlands: Medisch Centrum Rijnmond_Zuid, lcatie ZuiderAustria: Central Ethics CommitteeAustria: Competant AuthorityAustria: Federal Ministry for Health and WomenAustria: Secretariat of HealthSpain: Agencia Española de Medicamentos y Productos SanitariosSpain: Spanish Agency of MedicinesSpain: Spanish Drug AgencySwitzerland: Agency for Therapeutic ProductsSwitzerland: Local Ethics CommitteeSwitzerland: Swissmedic (Swiss Agency for Therapeutic Products)United Kingdom: Medicines and Healthcare Products Regulatory AgencyPoland: Drug InstitutPoland: Office for Registration of Medicinal Products, Medical Devices and Biocidal ProductsUnited States: Food and Drug AdministrationAustralia: Department of Health and Ageing Therapeutic Goods AdministrationAustralia: Human Research Ethics CommitteeAustralia: Therapeutic Goods AdministrationAustria: AGES - PharmaMed Austria Institut Wissenschaft & InformationAustria: Bundesamt fur Sicherheit im GesundheitswesenAustria: Bundesamt für Sicherheit im Gesundheitswesen
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 313
• Est. completion date: January 2010
• Est. primary completion date: January 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Idiopathic Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia
• Thrombocytosis

Intervention

Biological:
• Romiplostim
Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

References & Publications (4)

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. Erratum in: Blood. 2009 May 7;113(19):4822. — View 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. — View 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;

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;

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events	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.	Duration of treatment plus 8 weeks (up to 285 weeks)	Yes
Secondary	Number of Participants With a Platelet Response	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.	Duration of treatment (up to 277 weeks)	No
Secondary	Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies	The number of participants with a reduction or discontinuation of concurrent immune (idiopathic) thrombocytopenic purpura (ITP) therapies (corticosteroids, danazol, azathioprine) during the study.	Duration of treatment (up to 277 weeks)	No
Secondary	Change From Baseline in ITP Patient Assessment Questionnaire	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.	Baseline to Week 48	No
Secondary	Change From Baseline in Short Form 36 (SF-36)	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.	Baseline to Week 48	No
Secondary	Change From Baseline in Euroqol-5D (EQ-5D) Index Score	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.	Baseline to Week 48	No
Secondary	Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS)	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.	Baseline to Week 48	No
Secondary	Patient Global Assessment	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.	Week 1 and Week 48	No

External Links

•   AmgenTrials clinical trials website