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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01143038




Registration number
NCT01143038
Ethics application status
Date submitted
10/06/2010
Date registered
14/06/2010
Date last updated
21/09/2022

Titles & IDs
Public title
Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim
Scientific title
A Phase 2 Interventional Single Arm Study Describing Platelet Responses and ITP Remission Rates in Adult Subjects With Immune Thrombocytopenia Purpura Receiving Romiplostim
Secondary ID [1] 0 0
2010-019987-35
Secondary ID [2] 0 0
20080435
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Idiopathic Thrombocytopenic Purpura 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Romiplostim

Experimental: 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.


Treatment: Other: Romiplostim
Romiplostim will be administered weekly by subcutaneous injection
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Months With Platelet Response During the 12-Month Treatment Period
Timepoint [1] 0 0
12 months
Secondary outcome [1] 0 0
Percentage of Participants With ITP Remission
Timepoint [1] 0 0
Up to 24 months
Secondary outcome [2] 0 0
Percentage of Participants With Splenectomy During the 12-month Treatment Period
Timepoint [2] 0 0
12 months
Secondary outcome [3] 0 0
Number of Participants With Adverse Events
Timepoint [3] 0 0
From first dose date of romiplostim to end of study (up to 24 months).
Secondary outcome [4] 0 0
Number of Participants Who Developed Antibodies to Romiplostim
Timepoint [4] 0 0
Baseline and at end of treatment (based on response to treatment, this could occur between 12 months and approximately 18 months)

Eligibility
Key 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 10?/L at any time during the screening period
* Subject or subject's legally acceptable representative has provided informed consent
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/11/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/12/2013
Sample size
Target
Accrual to date
Final
75
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA
Recruitment hospital [1] 0 0
Research Site - Randwick
Recruitment hospital [2] 0 0
Research Site - Woolloongabba
Recruitment hospital [3] 0 0
Research Site - Adelaide
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
North Carolina
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
South Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Virginia
Country [8] 0 0
Czechia
State/province [8] 0 0
Brno
Country [9] 0 0
Czechia
State/province [9] 0 0
Ostrava-Poruba
Country [10] 0 0
Czechia
State/province [10] 0 0
Praha 10
Country [11] 0 0
Czechia
State/province [11] 0 0
Praha 2
Country [12] 0 0
France
State/province [12] 0 0
Bondy Cedex
Country [13] 0 0
France
State/province [13] 0 0
Créteil Cedex
Country [14] 0 0
France
State/province [14] 0 0
Dijon
Country [15] 0 0
France
State/province [15] 0 0
Pessac Cedex
Country [16] 0 0
France
State/province [16] 0 0
Toulouse Cedex 9
Country [17] 0 0
Germany
State/province [17] 0 0
Dresden
Country [18] 0 0
Germany
State/province [18] 0 0
Duisburg
Country [19] 0 0
Germany
State/province [19] 0 0
Düsseldorf
Country [20] 0 0
Germany
State/province [20] 0 0
Köln
Country [21] 0 0
Italy
State/province [21] 0 0
Bari
Country [22] 0 0
Italy
State/province [22] 0 0
Catania
Country [23] 0 0
Italy
State/province [23] 0 0
Monza (MB)
Country [24] 0 0
Italy
State/province [24] 0 0
Napoli
Country [25] 0 0
Italy
State/province [25] 0 0
Novara
Country [26] 0 0
Italy
State/province [26] 0 0
Roma
Country [27] 0 0
Italy
State/province [27] 0 0
San Giovanni Rotondo FG
Country [28] 0 0
Italy
State/province [28] 0 0
Vicenza
Country [29] 0 0
Poland
State/province [29] 0 0
Gdansk
Country [30] 0 0
Poland
State/province [30] 0 0
Katowice
Country [31] 0 0
Poland
State/province [31] 0 0
Lublin
Country [32] 0 0
Poland
State/province [32] 0 0
Poznan
Country [33] 0 0
Poland
State/province [33] 0 0
Wroclaw
Country [34] 0 0
Spain
State/province [34] 0 0
Andalucía
Country [35] 0 0
Spain
State/province [35] 0 0
Cataluña
Country [36] 0 0
Spain
State/province [36] 0 0
Galicia
Country [37] 0 0
Spain
State/province [37] 0 0
Madrid
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Coventry
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Leeds
Country [40] 0 0
United Kingdom
State/province [40] 0 0
London
Country [41] 0 0
United Kingdom
State/province [41] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Amgen
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT01143038
Trial related presentations / publications
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.
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.
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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
MD
Address 0 0
Amgen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01143038