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Completed

NCT Number: NCT03119974

Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in ITP

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.

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Key information

Conditions

Persistent or Chronic ITP

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Henri-Mondor Hospital

Créteil, 94010, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age >= 18 years
  • Diagnosis of ITP according to the standard definition
  • Disease duration of more than 3 months at Tpo-RA initiation
  • 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
  • Blood count lasting for less than 7 days
  • Normal marrow aspirate for patients aged of 60 and over
  • Informed signed consent
  • Treatment with Tpo-RA for at least 3 months

Exclusion criteria

  • 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

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Treatment and study plan

Tpo-RA discontinuation

Drug

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 outcomes

  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:
    • 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.

Secondary outcomes

  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

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Official study title

Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in Immune Thrombocytopenia: a Prospective Multicenter Open Study

Acronym: STOP-AGO

Important dates

Study start
2017
Primary completion
2020
Study completion
2021
First posted
Apr 19, 2017
Registry last updated
Jul 1, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.