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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 : May 23, 2017
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief Summary:

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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Tpo-RA discontinuation 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.




Primary Outcome Measures :
  1. 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:

      1. Their platelet count is < 30 x 109/L between week 0 and week 24, but also, in the setting of this study if:
      2. They need a rescue therapy (a new course of corticosteroids and/or intravenous immunoglobulin) after inclusion.


Secondary Outcome Measures :
  1. The rate of overall response after Tpo-RAs discontinuation [ Time Frame: 24 and 52 weeks ]
    response and complete response

  2. The duration of overall response after Tpo-RAs discontinuation. [ Time Frame: 24 and 52 weeks ]
    response and complete response

  3. 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

  4. 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

  5. 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

  6. To identify predictive factors, for overall prolonged response [ Time Frame: Weeks 24 ]
    Search for predictive factor of response



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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:

  1. Age >= 18 years
  2. Diagnosis of ITP according to the standard definition
  3. Disease duration of more than 3 months at Tpo-RA initiation
  4. Platelet count > 100 x 109/L for more than 2 months on Tpo-RA Therapy, with at least 3 platelet counts > 100 x 109/L
  5. Blood count lasting for less than 7 days
  6. Normal marrow aspirate for patients aged of 60 and over
  7. Informed signed consent
  8. Treatment with Tpo-RA for at least 3 months

Exclusion Criteria:

1) Anticoagulation or anti-platelet treatment 2) Recent treatment with corticosteroids ± intravenous immunoglobulins (less than 2 months) 3) Rituximab or splenectomy within the 2 months preceding the Tpo-RA initiation 4) Rituximab or splenectomy after Tpo-RA initiation/RA initiation 5) Previous failure of Tpo-RA discontinuation 6) Pregnant or breastfeeding women 7) No affiliation to a social security scheme or other social protection scheme 8) Inability or refusal to understand or refusal to sign the informed consent from study participation 9) Patient deprived of freedom or under legal protection (guardianship, curatorship) 10) Hypersensitivity to Romiplostin or to any of the excipients or to E. coli derived proteins


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): NCT03119974


Contacts
Contact: Matthieu Mahevas, MD, PhD (0)143812076 ext +33 matthieu.mahevas@aphp.fr

Locations
France
Henri-Mondor Hospital Recruiting
Creteil, France, 94010
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Matthieu Mahevas, MD, PhD Assistance Publique - Hôpitaux de Paris

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT03119974     History of Changes
Other Study ID Numbers: P150956
2016-001786-93 ( EudraCT Number )
First Posted: April 19, 2017    Key Record Dates
Last Update Posted: May 23, 2017
Last Verified: March 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Assistance Publique - Hôpitaux de Paris:
ITP
TPO-RAS
Thrombopoietin-receptor agonists