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
|Condition or disease||Intervention/treatment||Phase|
|Idiopathic Thrombocytopenic Purpura||Biological: Romiplostim||Phase 2|
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)|
|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|
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.
Romiplostim will be administered weekly by subcutaneous injection
- 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.
- 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.
- 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.
- 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.
- 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.
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
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