Clinical trial NCT01143038: Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim
Official title: A Phase 2 Interventional Single Arm Study Describing Platelet Responses and ITP Remission Rates in Adult Subjects With Immune Thrombocytopenia Purpura Receiving Romiplostim
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Idiopathic Thrombocytopenic Purpura
Keywords: ITP
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2010-06-14
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Number of Months With Platelet Response During the 12-Month Treatment Period
timeFrame: 12 months
Secondary outcomes:
measure: Percentage of Participants With ITP Remission
timeFrame: Up to 24 months
measure: Percentage of Participants With Splenectomy During the 12-month Treatment Period
timeFrame: 12 months
measure: Number of Participants With Adverse Events
timeFrame: From first dose date of romiplostim to end of study (up to 24 months).
measure: Number of Participants Who Developed Antibodies to Romiplostim
timeFrame: Baseline and at end of treatment (based on response to treatment, this could occur between 12 months and approximately 18 months)
Other outcomes:
Locations:
city: Anaheim
country: United States
facility: Research Site
state: California
city: Orange
country: United States
facility: Research Site
state: California
city: Boynton Beach
country: United States
facility: Research Site
state: Florida
city: Bethesda
country: United States
facility: Research Site
state: Maryland
city: Hickory
country: United States
facility: Research Site
state: North Carolina
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Charleston
country: United States
facility: Research Site
state: South Carolina
city: Richlands
country: United States
facility: Research Site
state: Virginia
city: Randwick
country: Australia
facility: Research Site
state: New South Wales
city: Woolloongabba
country: Australia
facility: Research Site
state: Queensland
city: Adelaide
country: Australia
facility: Research Site
state: South Australia
city: Brno
country: Czechia
facility: Research Site
city: Ostrava-Poruba
country: Czechia
facility: Research Site
city: Praha 10
country: Czechia
facility: Research Site
city: Praha 2
country: Czechia
facility: Research Site
city: Praha 2
country: Czechia
facility: Research Site
city: Bondy Cedex
country: France
facility: Research Site
city: Créteil Cedex
country: France
facility: Research Site
city: Dijon
country: France
facility: Research Site
city: Pessac Cedex
country: France
facility: Research Site
city: Toulouse Cedex 9
country: France
facility: Research Site
city: Dresden
country: Germany
facility: Research Site
city: Duisburg
country: Germany
facility: Research Site
city: Düsseldorf
country: Germany
facility: Research Site
city: Köln
country: Germany
facility: Research Site
city: Bari
country: Italy
facility: Research Site
city: Catania
country: Italy
facility: Research Site
city: Monza (MB)
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Novara
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: San Giovanni Rotondo FG
country: Italy
facility: Research Site
city: Vicenza
country: Italy
facility: Research Site
city: Gdansk
country: Poland
facility: Research Site
city: Katowice
country: Poland
facility: Research Site
city: Lublin
country: Poland
facility: Research Site
city: Poznan
country: Poland
facility: Research Site
city: Wroclaw
country: Poland
facility: Research Site
city: Málaga
country: Spain
facility: Research Site
state: Andalucía
city: Barcelona
country: Spain
facility: Research Site
state: Cataluña
city: A Coruña
country: Spain
facility: Research Site
state: Galicia
city: Alcorcon
country: Spain
facility: Research Site
state: Madrid
city: Majadahonda
country: Spain
facility: Research Site
state: Madrid
city: Coventry
country: United Kingdom
facility: Research Site
city: Leeds
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: Oxford
country: United Kingdom
facility: Research Site
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.