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

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ClinicalTrials.gov Identifier: NCT01143038
Recruitment Status : Completed
First Posted : June 14, 2010
Results First Posted : February 24, 2016
Last Update Posted : August 29, 2018
Sponsor:
Information provided by (Responsible Party):
Amgen

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.

Condition or disease Intervention/treatment Phase
Idiopathic Thrombocytopenic Purpura Biological: Romiplostim Phase 2

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.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 75 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : November 30, 2010
Actual Primary Completion Date : September 20, 2013
Actual Study Completion Date : December 26, 2013


Arm Intervention/treatment
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.
Biological: Romiplostim
Romiplostim will be administered weekly by subcutaneous injection
Other Names:
  • AMG 531
  • Nplate




Primary Outcome Measures :
  1. Number of Months With Platelet Response During the 12-Month Treatment Period [ Time Frame: 12 months ]
    The primary endpoint was the number of months a participant achieved a platelet response during the 12-month treatment period. A platelet response for any 1 month was defined as the median of platelet counts measured in the month ≥ 50 x 10^9/L. Platelet counts within 4 weeks following a rescue medication use or following splenectomy were considered non-response. Months without any platelet count measurement were considered as months with no platelet response.


Secondary Outcome Measures :
  1. Percentage of Participants With ITP Remission [ Time Frame: Up to 24 months ]
    ITP remission was defined as maintaining every platelet count ≥ 50 x 10^9/L for at least 6 months in the absence of romiplostim and any other therapies to treat ITP.

  2. Percentage of Participants With Splenectomy During the 12-month Treatment Period [ Time Frame: 12 months ]
    If treatment with romiplostim was deemed ineffective or intolerable by the investigator, a splenectomy may have been performed.

  3. Number of Participants With Adverse Events [ Time Frame: From first dose date of romiplostim to end of study (up to 24 months). ]
    An adverse event (AE) is defined as any untoward medical occurrence in a clinical trial participant. The event does not necessarily have a causal relationship with study treatment. A serious adverse event is defined as an adverse event that meets at least one of the following serious criteria: • fatal • life threatening • requires in-patient hospitalization or prolongation of existing hospitalization • results in persistent or significant disability/incapacity • congenital anomaly/birth defect • other significant medical hazard. Whether an adverse event was treatment-related (TRAE) or not was determined by investigator.

  4. Number of Participants Who Developed Antibodies to Romiplostim [ Time Frame: Baseline and at end of treatment (based on response to treatment, this could occur between 12 months and approximately 18 months) ]
    The number of participants who developed antibody formation (defined as negative at baseline and positive at post-baseline, transient or persistent) to romiplostim, endogenous thrombopoietin (eTPO), and thrombopoietin mimetic peptide (TMP, the peptide component of romiplostim) was summarized.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01143038


  Show 48 Study Locations
Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen

Additional Information:
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01143038     History of Changes
Other Study ID Numbers: 20080435
2010-019987-35 ( EudraCT Number )
First Posted: June 14, 2010    Key Record Dates
Results First Posted: February 24, 2016
Last Update Posted: August 29, 2018
Last Verified: August 2018

Keywords provided by Amgen:
ITP

Additional relevant MeSH terms:
Thrombocytopenia
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases