Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim

NCT ID: NCT01143038

Last Updated: 2022-09-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 75 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-11-30
• Study Completion Date: 2013-12-26

Brief Summary

The purpose of this study is to describe the number of months with a platelet response over a 12 month treatment period and to describe ITP remission rates in adults with ITP receiving romiplostim.

Detailed Description

The study includes a 4-week screening period, a 12-month romiplostim treatment period, and a romiplostim dose-tapering period.

During the 12-month treatment period romiplostim doses could be increased or decreased to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L. Participants who dose reduce such that they no longer require treatment with romiplostim during the 12-month treatment period will continue with all required study procedures up to 12 months and will be monitored for ITP remission for at least 6 months.

At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10^9/L will enter the tapering period, during which the romiplostim dose will be decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. If a participant maintains a platelet count of ≥ 50 x 10^9/L in the absence of romiplostim and all medications for ITP (concomitant or rescue), the participant will be followed for at least 6 months to confirm the incidence of ITP remission. If a participant's platelet count falls below 50 x 10^9/L and the participant has tapered off treatment with romiplostim, the participant will enter the stabilization period and reinitiate romiplostim for up to 8 weeks.

Conditions

Idiopathic Thrombocytopenic Purpura

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Romiplostim

Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10\^9/L.

Group Type: EXPERIMENTAL

Romiplostim

Intervention Type: BIOLOGICAL

Romiplostim will be administered weekly by subcutaneous injection

Interventions

Romiplostim

Romiplostim will be administered weekly by subcutaneous injection

Intervention Type: BIOLOGICAL

Other Intervention Names

AMG 531; Nplate

Eligibility Criteria

Inclusion Criteria

• Subject has been diagnosed with primary ITP according to the American Society of Hematology (ASH) guidelines (George et al, 1996) and previously received only 1st line therapies.

First line therapy is defined as corticosteroids, immunoglobulin G (IVIG), anti-D and vinca alkaloids (used for the treatment of ITP related thrombocytopenia only). A platelet transfusion at any time during the six month period since the original diagnosis would not exclude the subject from study participation

• Initial diagnosis of primary ITP within 6 months of enrollment
• Age ≥ 18 years at screening
• A single platelet count ≤ 30 x 10⁹/L at any time during the screening period
• Subject or subject's legally acceptable representative has provided informed consent

Exclusion Criteria

• Known history of a bone marrow stem cell disorder
• Surgical resection of the spleen
• Subject has a history of cancer or current malignancy other than basal cell carcinoma or cervical cancer in-situ with active treatment or disease within 5 years of screening
• Known history of congenital thrombocytopenia
• Known history of hepatitis B, hepatitis C, or human immunodeficiency virus
• Positive H. pylori by urea breath test or stool antigen test at screening
• Known history of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia
• Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant
• Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
• Previous history of recurrent venous thromboembolism or thrombotic events or an occurrence within 5 years of enrollment.
• Previous use of romiplostim, pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), eltrombopag, recombinant human thrombopoietin (rHuTPO) or any platelet producing agent
• Rituximab (for any indication) or mercaptopurine (6-MP) or anticipated use during the time of the proposed study
• All hematopoietic growth factors including interleukin-11 (IL-11) (oprelvekin) within 4 weeks before the screening visit
• Alkylating agents use at any time before or during the screening visit or anticipated during the time of the proposed study
• Known hypersensitivity to any recombinant E. coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
• Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s)
• Subject will have any other investigational procedures performed while enrolled in this clinical study
• Subject is pregnant or breast feeding, or planning to become pregnant within 5 weeks after the end of treatment
• Female subject of child bearing potential is not willing to use, in combination with her partner, highly effective contraception during treatment and for 4 weeks after the end of treatment
• Subject has previously enrolled into a romiplostim study
• Subject will not be available for protocol required study visits, to the best of the subject's and investigator's knowledge
• Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amgen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

MD

Role: STUDY_DIRECTOR

Amgen

Locations

Research Site

Anaheim, California, United States

Research Site

Orange, California, United States

Research Site

Boynton Beach, Florida, United States

Research Site

Bethesda, Maryland, United States

Research Site

Hickory, North Carolina, United States

Research Site

Philadelphia, Pennsylvania, United States

Research Site

Charleston, South Carolina, United States

Research Site

Richlands, Virginia, United States

Research Site

Randwick, New South Wales, Australia

Research Site

Woolloongabba, Queensland, Australia

Research Site

Adelaide, South Australia, Australia

Research Site

Brno, , Czechia

Research Site

Ostrava-Poruba, , Czechia

Research Site

Prague, , Czechia

Research Site

Prague, , Czechia

Research Site

Prague, , Czechia

Research Site

Bondy, , France

Research Site

Créteil, , France

Research Site

Dijon, , France

Research Site

Pessac, , France

Research Site

Toulouse, , France

Research Site

Cologne, , Germany

Research Site

Dresden, , Germany

Research Site

Duisburg, , Germany

Research Site

Düsseldorf, , Germany

Research Site

Bari, , Italy

Research Site

Catania, , Italy

Research Site

Monza (MB), , Italy

Research Site

Napoli, , Italy

Research Site

Novara, , Italy

Research Site

Roma, , Italy

Research Site

San Giovanni Rotondo FG, , Italy

Research Site

Vicenza, , Italy

Research Site

Gdansk, , Poland

Research Site

Katowice, , Poland

Research Site

Lublin, , Poland

Research Site

Poznan, , Poland

Research Site

Wroclaw, , Poland

Research Site

Málaga, Andalusia, Spain

Research Site

Barcelona, Catalonia, Spain

Research Site

A Coruña, Galicia, Spain

Research Site

Alcorcón, Madrid, Spain

Research Site

Majadahonda, Madrid, Spain

Research Site

Coventry, , United Kingdom

Research Site

Leeds, , United Kingdom

Research Site

London, , United Kingdom

Research Site

London, , United Kingdom

Research Site

Oxford, , United Kingdom

Countries

United States; Australia; Czechia; France; Germany; Italy; Poland; Spain; United Kingdom

References

Newland A, Godeau B, Priego V, Viallard JF, Lopez Fernandez MF, Orejudos A, Eisen M. Remission and platelet responses with romiplostim in primary immune thrombocytopenia: final results from a phase 2 study. Br J Haematol. 2016 Jan;172(2):262-73. doi: 10.1111/bjh.13827. Epub 2015 Nov 5.

Reference Type: BACKGROUND

PMID: 26537623 (View on PubMed)

Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, Lyons R, Garcia-Chavez J, Carpenter N, Wang X, Eisen M. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017 Aug;102(8):1342-1351. doi: 10.3324/haematol.2016.161968. Epub 2017 Apr 14.

Reference Type: BACKGROUND

PMID: 28411254 (View on PubMed)

Kuter DJ, Newland A, Chong BH, Rodeghiero F, Romero MT, Pabinger I, Chen Y, Wang K, Mehta B, Eisen M. Romiplostim in adult patients with newly diagnosed or persistent immune thrombocytopenia (ITP) for up to 1 year and in those with chronic ITP for more than 1 year: a subgroup analysis of integrated data from completed romiplostim studies. Br J Haematol. 2019 May;185(3):503-513. doi: 10.1111/bjh.15803. Epub 2019 Feb 21.

Reference Type: BACKGROUND

PMID: 30793285 (View on PubMed)

Kuter DJ, Arnold DM, Rodeghiero F, Janssens A, Selleslag D, Bird R, Newland A, Mayer J, Wang K, Olie R. Safety and efficacy of self-administered romiplostim in patients with immune thrombocytopenia: Results of an integrated database of five clinical trials. Am J Hematol. 2020 Jun;95(6):643-651. doi: 10.1002/ajh.25776. Epub 2020 Mar 21.

Reference Type: BACKGROUND

PMID: 32129511 (View on PubMed)

Related Links

http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

2010-019987-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

20080435

Identifier Type: -

Identifier Source: org_study_id