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Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT00415532
First received: December 21, 2006
Last updated: July 18, 2014
Last verified: July 2014
  Purpose

This is a phase 3b, multi-center, randomized, Standard of Care (SOC)-controlled, open-label, 52-week treatment study to compare romiplostim to medical SOC for Idiopathic Thrombocytopenia Purpura (ITP), with a 6-month Safety Follow-up. Patients randomized to romiplostim must complete the taper or discontinuation of medical SOC for ITP as soon as medically feasible after the initiation of romiplostim. After the completion or discontinuation of the study treatment period, any participant who does not transfer in to another romiplostim study will complete a 6-month Safety Follow-up period.


Condition Intervention Phase
Idiopathic Thrombocytopenic Purpura
Thrombocytopenia
Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenic Purpura
Drug: Medical Standard of Care for ITP
Biological: Romiplostim
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Randomized, Controlled, Open-label Study Evaluating the Efficacy and Tolerability of AMG 531 Versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects With Immune (Idiopathic) Thrombocytopenic Purpura

Resource links provided by NLM:


Further study details as provided by Amgen:

Primary Outcome Measures:
  • Number of Participants With Splenectomy During 52-Week Treatment Period [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Occurrence of a splenectomy. Participants who discontinued study during the treatment period prior to reporting a splenectomy were considered as having had a splenectomy.

  • Number of Participants With Treatment Failure During 52-Week Treatment Period [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
    Treatment failure was defined by platelet counts ≤ 20 x 10^9/L for 4 consecutive weeks at the highest recommended dose and schedule, a major bleeding event, or change in therapy due to an intolerable side effect or bleeding symptom.


Secondary Outcome Measures:
  • Time to Splenectomy [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Time to splenectomy in days calculated from date of randomization to date of splenectomy, or censored at date of end of treatment visit if no splenectomy was done during treatment period.

  • Percentage of Participants With Platelet Response [ Time Frame: Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52 ] [ Designated as safety issue: No ]
    Platelet response was defined as platelet counts > 50 x 10^9/L, measured at each study visit (excluding those within 8 weeks of prior rescue medication use) up to the time of splenectomy or the end of initial treatment period, whichever occurred first.

  • Change in ITP-PAQ Physical Health Domain of Symptoms [ Time Frame: Baseline and 52 weeks ] [ Designated as safety issue: No ]

    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of symptoms. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life.

    Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.


  • Change in ITP-PAQ Physical Health Domain of Fatigue [ Time Frame: Baseline and 52 weeks ] [ Designated as safety issue: No ]
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of fatigue. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.

  • Change in ITP-PAQ Physical Health Domain of Bother [ Time Frame: Baseline and 52 weeks ] [ Designated as safety issue: No ]
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of bother. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.

  • Change in ITP-PAQ Physical Health Domain of Activity [ Time Frame: Baseline and 52 weeks ] [ Designated as safety issue: No ]

    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of activity. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life.

    Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.



Enrollment: 234
Study Start Date: December 2006
Study Completion Date: July 2009
Primary Completion Date: May 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Romiplostim
Romiplostim administered by subcutaneous injection once weekly at a starting dose of 3 μg/kg, adjusted to a maximum dose of 10 μg/kg to maintain a platelet count between 50 and 200 x 10^9/L for up to 52 weeks.
Biological: Romiplostim
Other Name: AMG 531
Standard of Care
Medical standard of care treatments were selected and prescribed by the investigator according to standard institutional practices or therapeutic guidelines and administered for up to 52 weeks.
Drug: Medical Standard of Care for ITP

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subject is ≥ 18 years of age
  • Subject has a diagnosis of Idiopathic Thrombocytopenia Purpura (ITP) according to the American Society of Hematology (ASH) guidelines
  • If subject is > 60 years of age, subject has a written bone marrow biopsy report confirming the diagnosis of ITP
  • Subject has received at least 1 prior therapy for ITP
  • Subject has a platelet count < 50,000 or their platelet count falls to < 50,000 during or after a clinically-indicated taper or discontinuation of current ITP therapy
  • Before any study-specific procedure, the appropriate written informed consent must be obtained

Exclusion Criteria:

  • Subject has had a splenectomy for any reason
  • Subject has an active malignancy
  • Subject has a history of cancer, other than basal cell carcinoma or cervical carcinoma in situ, with treatment or active disease within 5 years
  • Subject has a known history of bone marrow stem cell disorder
  • Subject has participated in any study evaluating polyethylene glycol-conjugated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), AMG 531, or a thrombopoietic protein
  • Subject is receiving other investigational agents or procedures
  • Subject is currently enrolled in, or has completed within the last 30 days, another investigational device or drug study
  • Subject is pregnant or breast feeding
  • Subject is not using adequate contraceptive precautions
  • Subject has known sensitivity to any recombinant E. coli-derived product
  • Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative
  • Subject has any kind of disorder that compromises the ability of the subject to comply with study procedures
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00415532

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

Additional Information:
No publications provided by Amgen

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT00415532     History of Changes
Other Study ID Numbers: 20060131
Study First Received: December 21, 2006
Results First Received: October 1, 2010
Last Updated: July 18, 2014
Health Authority: 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
Canada: Health Canada
Canada: Health Products and Food Branch
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: Ministry of Health
Germany: Federal Institute for Drugs and Medical Devices
Germany: Paul_Ehrlich-Institut Bundesamt fur Sera und Impfstoffe
Hungary: National Institute of Pharmacy
Italy: Ministry of Health
Netherlands: Medicines Evaluation Board
Netherlands: Medisch Centrum Rijnmond_Zuid, lcatie Zuider
Poland: Drug Institut
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Portugal: National Institute of Pharmacy and Medicines
Spain: Spanish Agency of Medicines
Spain: Spanish Drug Agency
Sweden: Medical Products Agency
Switzerland: Agency for Therapeutic Products
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration
United States: Institutional Review Board
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: Federal Ministry for Health and Women
Austria: Secretariat of Health
Belgium: Federal Agency for Medicines and Health Products, FAMHP

Keywords provided by Amgen:
immune thrombocytopenic purpura
idiopathic thrombocytopenic purpura
immune (idiopathic) thrombocytopenic purpura
splenectomy
platelet
AMG 531
thrombopoietin
blood disorder
bleeding disorder
TPO
thrombopoietic protein

Additional relevant MeSH terms:
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Purpura
Thrombocytopenia
Autoimmune Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Hematologic Diseases
Hemorrhage
Hemorrhagic Disorders
Immune System Diseases
Pathologic Processes
Signs and Symptoms
Skin Manifestations
Thrombotic Microangiopathies

ClinicalTrials.gov processed this record on November 25, 2014