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

Thrombocytopenia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00415532
• Other study ID #: 20060131
• Status: Completed
• Phase: Phase 3
• Start date: December 1, 2006
• Est. completion date: May 11, 2009

Study information

• Verified date: November 2022
• Source: Amgen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 234
• Est. completion date: May 11, 2009
• Est. primary completion date: November 7, 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Idiopathic Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia
• Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
• Thrombocytopenic Purpura

Intervention

Drug:
• Medical Standard of Care for ITP

Biological:
• Romiplostim

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

References & Publications (2)

Kuter DJ, Mathias SD, Rummel M, Mandanas R, Giagounidis AA, Wang X, Deuson RR. Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. Am J Hematol. 2012 May;87(5):558-61. doi: 10.1002/ajh.23163. Epub 2012 Mar 28. — View Citation

Kuter DJ, Rummel M, Boccia R, Macik BG, Pabinger I, Selleslag D, Rodeghiero F, Chong BH, Wang X, Berger DP. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med. 2010 Nov 11;363(20):1889-99. doi: 10.1056/NEJMoa1002625. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Splenectomy During 52-Week Treatment Period	Occurrence of a splenectomy. Participants who discontinued study during the treatment period prior to reporting a splenectomy were considered as having had a splenectomy.	52 weeks
Primary	Number of Participants With Treatment Failure During 52-Week Treatment Period	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.	52 weeks
Secondary	Time to Splenectomy	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.	52 weeks
Secondary	Percentage of Participants With Platelet Response	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.	Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52
Secondary	Change in ITP-PAQ Physical Health Domain of Symptoms	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.	Baseline and 52 weeks
Secondary	Change in ITP-PAQ Physical Health Domain of Fatigue	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.	Baseline and 52 weeks
Secondary	Change in ITP-PAQ Physical Health Domain of Bother	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.	Baseline and 52 weeks
Secondary	Change in ITP-PAQ Physical Health Domain of Activity	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.	Baseline and 52 weeks

External Links

•   AmgenTrials clinical trials website
•   Notice regarding posted summaries of trial results