Persistent or Chronic ITPPhase 4STOP-AGONCT03119974
Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in ITP
A Phase 4 study testing Tpo-RA discontinuation in people with Persistent or Chronic ITP, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Persistent or Chronic ITP
- What is being tested?
- Tpo-RA discontinuation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteFrance
- Study dates
- 26 Sep 2017to 16 Feb 2021
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
- -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in Immune Thrombocytopenia: a Prospective Multicenter Open Study
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.
- Conditions
- Persistent or Chronic ITP
- Being tested
- Tpo-RA discontinuation
- Keywords
- ITP, TPO-RAS, Thrombopoietin-receptor agonists
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tpo-RA discontinuationDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Tpo-RA discontinuation | Other | Not described Receives: Drug: Tpo-RA discontinuation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
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.
Measured: week 24 (6 months)
Secondary outcomes (6)
The rate of overall response after Tpo-RAs discontinuation
response and complete response
Measured: 24 and 52 weeks
The duration of overall response after Tpo-RAs discontinuation.
response and complete response
Measured: 24 and 52 weeks
The number of bleeding events during the reduction period and along the study period at weeks 4, 8, 12,24,36, 52
Safety assess of Tpo-RAs discontinuation
Measured: at weeks 4, 8, 12,24,36, 52
The rate of the response to a new course of Tpo-RAs in case of relapse after Tpo-RAs discontinuation at one year
Rate of the response in case of relapse
Measured: 52 weeks
The delay of the response to a new course of Tpo-RAs in case of relapse after Tpo-RAs discontinuation
Delay of the response in case of relapse
Measured: 52 weeks
To identify predictive factors, for overall prolonged response
Search for predictive factor of response
Measured: Weeks 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2017
- Study start26 Sep 2017
- Primary completion26 Aug 2020
- Study completion16 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Persistent or Chronic ITPStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Matthieu Mahevas, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03119974
- Sponsor's protocol ID
- P150956
- Other IDs
- 2016-001786-93, 2016
- Acronym
- STOP-AGO
- Status last verified
- Jun 2025
- First submitted
- 23 Dec 2016
- Last updated
- 1 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Cottu A, Guillet S, Viallard JF, Riviere E, Cheze S, Gobert D, Neel A, Graveleau J, Marolleau JP, Lefrere F, Moulis G, Lega JC, Moignet-Autrel A, Robbins A, Crickx E, Boutin E, Noel N, Malphettes M, Galicier L, Audia S, Bonnotte B, Lambotte O, Fain O, Gerfaud-Valentin M, Terriou L, Martis N, Morin AS, Perlat A, Le Gallou T, Roy-Peaud F, Puyade M, Comont T, Limal N, Languille L, Michel M, Godeau B, Mahevas M. Long-term follow-up of the STOPAGO study. Blood. 2025 Jan 9;145(2):244-247. doi: 10.1182/blood.2024025707.
PubMed 39476101Guillet S, Crickx E, Azzaoui I, Chappert P, Boutin E, Viallard JF, Riviere E, Gobert D, Galicier L, Malphettes M, Cheze S, Lefrere F, Audia S, Bonnotte B, Lambotte O, Noel N, Fain O, Moulis G, Hamidou M, Gerfaud-Valentin M, Marolleau JP, Terriou L, Martis N, Morin AS, Perlat A, Le Gallou T, Roy-Peaud F, Robbins A, Lega JC, Puyade M, Comont T, Limal N, Languille L, Zarrour A, Luka M, Menager M, Belmondo T, Hue S, Canoui-Poitrine F, Michel M, Godeau B, Mahevas M. Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study. Blood. 2023 Jun 8;141(23):2867-2877. doi: 10.1182/blood.2022018665.
PubMed 36893453
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in Immune Thrombocytopenia: a Prospective Multicenter Open Study
- ClinicalTrials.gov ID
- NCT03119974
- Acronym / sponsor ID
- STOP-AGO / P150956
- Conditions
- Persistent or Chronic ITP
- Keywords
- ITP, TPO-RAS, Thrombopoietin-receptor agonists
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 26 Sep 2017 (actual)
- Primary completion
- 26 Aug 2020 (actual)
- Study completion
- 16 Feb 2021 (actual)
- First posted
- 19 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tpo-RA discontinuation | Other | Drug: Tpo-RA discontinuation |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (2 listed)
- 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
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tpo-RA discontinuationa 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. | Drug | Tpo-RA discontinuation |
Primary outcome
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.
6 secondary outcomes
The rate of overall response after Tpo-RAs discontinuation
Time frame: 24 and 52 weeks
The duration of overall response after Tpo-RAs discontinuation.
Time frame: 24 and 52 weeks
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
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
The delay of the response to a new course of Tpo-RAs in case of relapse after Tpo-RAs discontinuation
Time frame: 52 weeks
To identify predictive factors, for overall prolonged response
Time frame: Weeks 24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2025.
- Derived from this study: PubMed 39476101Cottu A, Guillet S, Viallard JF, Riviere E, Cheze S, Gobert D, Neel A, Graveleau J, Marolleau JP, Lefrere F, Moulis G, Lega JC, Moignet-Autrel A, Robbins A, Crickx E, Boutin E, Noel N, Malphettes M, Galicier L, Audia S, Bonnotte B, Lambotte O, Fain O, Gerfaud-Valentin M, Terriou L, Martis N, Morin AS, Perlat A, Le Gallou T, Roy-Peaud F, Puyade M, Comont T, Limal N, Languille L, Michel M, Godeau B, Mahevas M. Long-term follow-up of the STOPAGO study. Blood. 2025 Jan 9;145(2):244-247. doi: 10.1182/blood.2024025707.
- Derived from this study: PubMed 36893453Guillet S, Crickx E, Azzaoui I, Chappert P, Boutin E, Viallard JF, Riviere E, Gobert D, Galicier L, Malphettes M, Cheze S, Lefrere F, Audia S, Bonnotte B, Lambotte O, Noel N, Fain O, Moulis G, Hamidou M, Gerfaud-Valentin M, Marolleau JP, Terriou L, Martis N, Morin AS, Perlat A, Le Gallou T, Roy-Peaud F, Robbins A, Lega JC, Puyade M, Comont T, Limal N, Languille L, Zarrour A, Luka M, Menager M, Belmondo T, Hue S, Canoui-Poitrine F, Michel M, Godeau B, Mahevas M. Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study. Blood. 2023 Jun 8;141(23):2867-2877. doi: 10.1182/blood.2022018665.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Matthieu Mahevas, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tpo-RA discontinuation in people with Persistent or Chronic ITP, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03119974, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2025.