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

NCT00415532 | Completed Phase 3 Results

• 1 other identifier: 20060131
• Study Type: interventional
• Target: 234
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Idiopathic Thrombocytopenic Purpura; Thrombocytopenia; Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP); Thrombocytopenic Purpura

Keywords

splenectomy; platelet; AMG 531; thrombopoietin; blood disorder; bleeding disorder; immune thrombocytopenic purpura; idiopathic thrombocytopenic purpura; immune (idiopathic) thrombocytopenic purpura; TPO; thrombopoietic protein

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (6)

• Time to Splenectomy

  52 weeks

• Percentage of Participants With Platelet Response

  Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52

• Change in ITP-PAQ Physical Health Domain of Symptoms

  Baseline and 52 weeks

• Change in ITP-PAQ Physical Health Domain of Fatigue

  Baseline and 52 weeks

• Change in ITP-PAQ Physical Health Domain of Bother

  Baseline and 52 weeks

Study Arms (2)

Romiplostim

EXPERIMENTAL

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

Standard of Care

OTHER

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

Interventions

Medical Standard of Care for ITP DRUG

Standard of Care

Romiplostim BIOLOGICAL

Also known as: AMG 531

Romiplostim

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amgen: lead

Related 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.

  PMID: 22460421 BACKGROUND

• 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.

  PMID: 21067381 BACKGROUND

Related Links

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

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Purpura, Thrombocytopenic; Jacobs syndrome; Hematologic Diseases; Hemostatic Disorders

Interventions

Inosine Triphosphate; romiplostim

Condition Hierarchy (Ancestors)

Purpura; Blood Coagulation Disorders; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Inosine Nucleotides; Purine Nucleotides; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Nucleotides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleotides

Results Point of Contact

• Title: Study Director
• Organization: Amgen Inc.

866-572-6436

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2006
• First Posted: December 25, 2006
• Study Start: December 1, 2006
• Primary Completion: November 7, 2008
• Study Completion: May 11, 2009
• Last Updated: November 8, 2022
• Results First Posted: July 8, 2013

Record last verified: 2022-11