Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in ITP (STOP-AGO)
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|ClinicalTrials.gov Identifier: NCT03119974|
Recruitment Status : Recruiting
First Posted : April 19, 2017
Last Update Posted : February 18, 2020
Thrombopoietin-receptor agonists (Tpo-RAs) have profoundly changed the management of ITP. However, today, there are no international recommendations concerning the long-term use of these costly, potentially pro-thrombotic agents, and that could induce bone marrow fibrosis in case of prolonged treatment. Tpo-RAs have been thought to play only a supporting role in ITP management. But our center along with many other research centers, have reported unexpected cases of durable remission after Tpo-RAs discontinuation in adult chronic ITP. In these retrospective studies, more than 20 % of patients were able to achieve prolonged remission.
The purpose of this study is to demonstrate that a substantial proportion of ITP patients may achieve a prolonged response after Tpo-RA discontinuation.
The investigators developed, in this study, a standardized procedure to discontinue Eltrombopag and Romiplostim, wherein the dose will be slowly tapered to limit the risk of bleeding. In case of relapse after Tpo-RA discontinuation, the decision to start a new therapy will be based on the clinician's judgment.
|Condition or disease||Intervention/treatment||Phase|
|Persistent or Chronic ITP||Drug: Tpo-RA discontinuation||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||48 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in Immune Thrombocytopenia: a Prospective Multicenter Open Study|
|Actual Study Start Date :||April 18, 2017|
|Estimated Primary Completion Date :||April 2020|
|Estimated Study Completion Date :||April 2020|
Drug: Tpo-RA discontinuation
a standardized strategy of dosage reduction will be implemented according to a predetermined scheme on persistent and/or chronic ITP patients who have previously achieved a stable and prolonged (> 2 months) complete response (platelets counts > 100 x 109/L) period on Tpo-RA treatment.
- The proportion of patients achieving an overall response (complete response and response) at week 24 (6 months). The criteria of response will be defined according to international terminology [ Time Frame: week 24 (6 months) ]
the criteria of response will be defined as the following:
- Response (R) will be defined as sustained platelet count >30x109/L in the absence of bleeding or use of any other ITP directed therapies between the week 0 (discontinuation) and week 24.
- Complete response (CR) by a platelet count > 100x 109/L in the absence use of any other ITP directed therapies between week 0 and week 24.
Patients will be considered as being non-responders (NR) if:
- Their platelet count is < 30 x 109/L between week 0 and week 24, but also, in the setting of this study if:
- They need a rescue therapy (a new course of corticosteroids and/or intravenous immunoglobulin) after inclusion.
- The rate of overall response after Tpo-RAs discontinuation [ Time Frame: 24 and 52 weeks ]response and complete response
- The duration of overall response after Tpo-RAs discontinuation. [ Time Frame: 24 and 52 weeks ]response and complete response
- The number of bleeding events during the reduction period and along the study period at weeks 4, 8, 12,24,36, 52 [ Time Frame: at weeks 4, 8, 12,24,36, 52 ]Safety assess of Tpo-RAs discontinuation
- The rate of the response to a new course of Tpo-RAs in case of relapse after Tpo-RAs discontinuation at one year [ Time Frame: 52 weeks ]Rate of the response in case of relapse
- The delay of the response to a new course of Tpo-RAs in case of relapse after Tpo-RAs discontinuation [ Time Frame: 52 weeks ]Delay of the response in case of relapse
- To identify predictive factors, for overall prolonged response [ Time Frame: Weeks 24 ]Search for predictive factor of response
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03119974
|Contact: Matthieu Mahevas, MD, PhD||(0)143812076 ext +email@example.com|
|Creteil, France, 94010|
|Principal Investigator:||Matthieu Mahevas, MD, PhD||Assistance Publique - Hôpitaux de Paris|