Adult Immune Thrombocytopenia (ITP)Phase 3DAPS-ITPNCT02627417
Efficacy and Safety of DAPSone as a Second-line Option in Adult Immune Thrombocytopenia
A Phase 3 study testing Dapsone (Disulone®) and Prednisone (Cortancyl®) in people with Adult Immune Thrombocytopenia (ITP) and Dapsone, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Due to its expected efficacy based on the retrospective data available in ITP, its relatively good safety profile and its very low cost , dapsone could be a good steroid-sparing second-line option for adults with ITP. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adult Immune Thrombocytopenia (ITP), Dapsone
- What is being tested?
- Dapsone (Disulone®) and Prednisone (Cortancyl®)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 216 participants
- Where
- 1 siteFrance
- Study dates
- Dec 2015to May 2019
- Phase
- Phase 3
- 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 primary ITP according to the standard definition and international guidelines.
- Previous transient response to corticosteroids ± intravenous immunoglobulin defined by an increase of the platelet count above 30 x 109/L with at least a twofold increase of the pre-treatment count.
- At least platelet count ≤ 30 x 109/L within the 2 weeks before inclusion with a platelet count at time of inclusion below 50 x 109/L, or platelet count < 50 x 109/L at any time point in patients requiring treatment (i.e., patients with bleeding symptoms, elderly patients with comorbidities and/or patients on aspirin for example, or other reason at the investigator discretion) (modified by amendment 8/11/2016)
- Normal marrow aspirate for patients aged of 60 and above.
- Negative pregnancy test and effective method of contraception for women of childbearing age over the study period.
- Informed consent.
- Patient affiliated to the French National Social Security System
Who cannot join
- Secondary ITP. Patients with positive antinuclear antibodies and/or positive antiphospholipid antibodies not fulfilling the classification criteria for systemic lupus erythematosus and/or antiphospholipid antibody syndrome will not be excluded.
- Platelet count ≥ 50 x 109/L or between 30 and 50 x 109/L and no bleeding symptoms and no need for treatment (modified by amendment 8/11/2016)
- Severe bleeding manifestations defined a bleeding score ≥ 8
- No previous transient response to corticosteroids ± intravenous immunoglobulin.
- Previous ITP treatment other than corticosteroids and intravenous immunoglobulin (including rituximab and splenectomy).
- Active severe infection or history of severe infection within 4 weeks before inclusion.
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- History of allergy to sulfonamides.
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency.
- History of methemoglobinemia
- Hemoglobin level < 11g/dL and/or neutrophil count < 1,500/ gL.
- History of autoimmune (Evans' syndrome) or hereditary haemolytic anemia.
- Liver or kidney function impairment (creatinine clearance < 30 ml/min, ALT, AST >2 times upper normal limit). (modified by amendment 8/11/2017)
- Hepatitis C virus (HCV) Ab, HIV Ab, HBsAg, seropositive status. (modified by amendment 8/11/2017)
- Concomitant medical condition requiring anticoagulation. (modified by amendment 8/11/2017)
- Pregnancy or lactation.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Prospective Multicenter Randomized Open-label Controlled Trial Assessing Efficacy and Safety of DAPSone as a Second-line Option in Adult Immune Thrombocytopenia
Due to its expected efficacy based on the retrospective data available in ITP, its relatively good safety profile and its very low cost , dapsone could be a good steroid-sparing second-line option for adults with ITP.
This study is a phase III prospective multicenter randomized open trial comparing two treatment strategies:
Read more
- Arm A (experimental arm): prednisone at 1 mg/kg for 3 weeks + dapsone at 100 mg per day up to week 52 if an initial response is achieved.
- Arm B (control arm): prednisone alone at 1 mg/kg for 3 weeks followed by monitoring and "standard of care" The aim of the study is to demonstrate the efficacy of dapsone based on the overall response rate (including response and complete response) as a second-line treatment for adults with newly-diagnosed persistent or chronic (modified by amendment 08/11/2016) ITP not achieving a durable response with corticosteroids. The primary endpoint will be the overall response-rate (response or complete response according to standard definitions) in both arms at week 52 (1 year).
The secondary endpoints are the following :
- To assess the safety of dapsone over the study period and especially the incidence of cutaneous reactions.
- To analyze the overall response rate (platelet count > 30 x 109/L with at least a doubling of the pre-treatment count in the absence of any other ITP treatment) in both treatment arms at week 24.
- To compare the rate of complete response and failure in both arms at 24 and 52 weeks.
- To compare time to treatment failure (TTF) in both arms
- To investigate the mechanisms of action of dapsone in ITP in a subgroup of patients (ancillary study)
- Conditions
- Adult Immune Thrombocytopenia (ITP), Dapsone
- Being tested
- Dapsone (Disulone®), Prednisone (Cortancyl®) alone followed by monitoring and "standard of care", Prednisone (Cortancyl®)
- Keywords
- ITP, Dapsone
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dapsone (Disulone®)Drug
Also called experimental arm
Experimental group will receive dapsone at a fixed dose of 100 mg per day for a total of 12 months unless they fail to respond to the treatment.
Prednisone (Cortancyl®) alone followed by monitoring and "standard of care"Drug
Also called control arm
Patients randomized in the control arm will be treated only prednisone at a daily dose of 1 mg per kg for 2 weeks and then tapered and stopped over a week for a total of 3 consecutive weeks. After this 3 weeks period, patients from arm B will be left without treatment and monitored.
Prednisone (Cortancyl®)Drug
Also called experimental arm
Experimental group will receive prednisone at a daily dose of 1 mg per kg for 2 weeks and then tapered and stopped over a week for a total of 3 consecutive weeks.
| Study group | Role | What they receive |
|---|---|---|
| Dapsone: (Disulone®) | Experimental | Dapsone: Disulone® given orally at 100 mg per day Receives: Drug: Dapsone (Disulone®); Drug: Prednisone (Cortancyl®) |
| Prednisone (Cortancyl®) alone and "standard of care" | Active comparator | Prednisone (Cortancyl®) alone at 1 mg/kg for 3 weeks followed by monitoring and "standard of care" (control arm) Receives: Drug: Prednisone (Cortancyl®) alone followed by monitoring and "standard of care" |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response-rate.
Measured: at week 52
Secondary outcomes (6)
Assess the safety of dapsone especially the incidence of cutaneous adverse effects.
Measured: over the study period (up to 52 weeks)
Compare the overall response rate .
platelet count > 30 x 109/L with at least a doubling of the pre-treatment count in the absence of any other ITP treatment in both arms
Measured: at week 24
Proportion of patients with complete response in both arms, defined as follows: - Proportion of patients with a platelet count > 100 x 109/L in the absence use of any other ITP directed therapies
Measured: at 24 and at 52 weeks
Proportion of non-responders patients (primary failure)
Patients will be considered as being non-responders if:
- Their platelet count at the end of the study is < 30 x 109/L, but also, in the setting of this study if:
- They need a rescue therapy (a new course of corticosteroids and/or intravenous immunoglobulin) more than 6 weeks after inclusion. or
- They receive any other treatment for ITP than dapsone or prednisone (including rituximab, splenectomy and Tpo-R agonists) over the study period
Measured: at 24 and at 52 weeks
Number of days to treatment failure in both arms
Measured: over the study period (up to 52 weeks)
Mechanisms of action of Dapsone: degree of phagocytosis of autologous platelets and red blood cells, cytokines profile expression (including IL-8), whole blood gene expression signature between responders and non-responders.
Measured: over the study period (52 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2015 (estimated)
- Study startDec 2015
- Primary completionMay 2019 (estimated)
- Study completionMay 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Adult Immune Thrombocytopenia (ITP)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 216 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- MARC MICHEL, Prof. M.D, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02627417
- Sponsor's protocol ID
- P140925
- Acronym
- DAPS-ITP
- Status last verified
- Jul 2017
- First submitted
- 3 Nov 2015
- Last updated
- 26 Jul 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Durand JM, Lefevre P, Hovette P, Mongin M, Soubeyrand J. Dapsone for idiopathic autoimmune thrombocytopenic purpura in elderly patients. Br J Haematol. 1991 Jul;78(3):459-60. doi: 10.1111/j.1365-2141.1991.tb04467.x. No abstract available.
PubMed 1873232Godeau B, Oksenhendler E, Bierling P. Dapsone for autoimmune thrombocytopenic purpura. Am J Hematol. 1993 Sep;44(1):70-2. doi: 10.1002/ajh.2830440117.
PubMed 8342569Hernandez F, Linares M, Colomina P, Pastor E, Cervero A, Perez A, Perella M. Dapsone for refractory chronic idiopathic thrombocytopenic purpura. Br J Haematol. 1995 Jun;90(2):473-5. doi: 10.1111/j.1365-2141.1995.tb05179.x.
PubMed 7794776Godeau B, Durand JM, Roudot-Thoraval F, Tenneze A, Oksenhendler E, Kaplanski G, Schaeffer A, Bierling P. Dapsone for chronic autoimmune thrombocytopenic purpura: a report of 66 cases. Br J Haematol. 1997 May;97(2):336-9. doi: 10.1046/j.1365-2141.1997.412687.x.
PubMed 9163598Damodar S, Viswabandya A, George B, Mathews V, Chandy M, Srivastava A. Dapsone for chronic idiopathic thrombocytopenic purpura in children and adults--a report on 90 patients. Eur J Haematol. 2005 Oct;75(4):328-31. doi: 10.1111/j.1600-0609.2005.00545.x.
PubMed 16146539Larue M, Moulis G, Rueter M, Audia S, Comont T, Terriou L, Viallard JF, Pan-Petesch B, Royer B, Bonnotte B, Galicier L, Lambotte O, Lefrere F, Cheze S, Ebbo M, Duong TA, Boutin E, Languille L, Mahevas M, Godeau B, Canoui-Poitrine F, Michel M. Efficacy and safety of dapsone in adult primary immune thrombocytopenia. Blood Adv. 2025 Apr 22;9(8):1976-1983. doi: 10.1182/bloodadvances.2024014939.
PubMed 39874521
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Multicenter Randomized Open-label Controlled Trial Assessing Efficacy and Safety of DAPSone as a Second-line Option in Adult Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT02627417
- Acronym / sponsor ID
- DAPS-ITP / P140925
- Conditions
- Adult Immune Thrombocytopenia (ITP), Dapsone
- Keywords
- ITP, Dapsone
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 216 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Dec 2015
- Primary completion
- May 2019 (estimated)
- Study completion
- May 2019 (estimated)
- First posted
- 11 Dec 2015 (estimated)
- 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.
Due to its expected efficacy based on the retrospective data available in ITP, its relatively good safety profile and its very low cost , dapsone could be a good steroid-sparing second-line option for adults with ITP.
This study is a phase III prospective multicenter randomized open trial comparing two treatment strategies:
- Arm A (experimental arm): prednisone at 1 mg/kg for 3 weeks + dapsone at 100 mg per day up to week 52 if an initial response is achieved.
- Arm B (control arm): prednisone alone at 1 mg/kg for 3 weeks followed by monitoring and "standard of care" The aim of the study is to demonstrate the efficacy of dapsone based on the overall response rate (including response and complete response) as a second-line treatment for adults with newly-diagnosed persistent or chronic (modified by amendment 08/11/2016) ITP not achieving a durable response with corticosteroids. The primary endpoint will be the overall response-rate (response or complete response according to standard definitions) in both arms at week 52 (1 year).
The secondary endpoints are the following :
- To assess the safety of dapsone over the study period and especially the incidence of cutaneous reactions.
- To analyze the overall response rate (platelet count > 30 x 109/L with at least a doubling of the pre-treatment count in the absence of any other ITP treatment) in both treatment arms at week 24.
- To compare the rate of complete response and failure in both arms at 24 and 52 weeks.
- To compare time to treatment failure (TTF) in both arms
- To investigate the mechanisms of action of dapsone in ITP in a subgroup of patients (ancillary study)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dapsone: (Disulone®) | Experimental | Drug: Dapsone (Disulone®), Drug: Prednisone (Cortancyl®)Dapsone: Disulone® given orally at 100 mg per day |
| Prednisone (Cortancyl®) alone and "standard of care" | Active comparator | Drug: Prednisone (Cortancyl®) alone followed by monitoring and "standard of care"Prednisone (Cortancyl®) alone at 1 mg/kg for 3 weeks followed by monitoring and "standard of care" (control arm) |
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 primary ITP according to the standard definition and international guidelines.
- Previous transient response to corticosteroids ± intravenous immunoglobulin defined by an increase of the platelet count above 30 x 109/L with at least a twofold increase of the pre-treatment count.
- At least platelet count ≤ 30 x 109/L within the 2 weeks before inclusion with a platelet count at time of inclusion below 50 x 109/L, or platelet count < 50 x 109/L at any time point in patients requiring treatment (i.e., patients with bleeding symptoms, elderly patients with comorbidities and/or patients on aspirin for example, or other reason at the investigator discretion) (modified by amendment 8/11/2016)
- Normal marrow aspirate for patients aged of 60 and above.
Key exclusion criteria (15 listed)
- Secondary ITP. Patients with positive antinuclear antibodies and/or positive antiphospholipid antibodies not fulfilling the classification criteria for systemic lupus erythematosus and/or antiphospholipid antibody syndrome will not be excluded.
- Platelet count ≥ 50 x 109/L or between 30 and 50 x 109/L and no bleeding symptoms and no need for treatment (modified by amendment 8/11/2016)
- Severe bleeding manifestations defined a bleeding score ≥ 8
- No previous transient response to corticosteroids ± intravenous immunoglobulin.
- Previous ITP treatment other than corticosteroids and intravenous immunoglobulin (including rituximab and splenectomy).
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 |
|---|---|---|
| Dapsone (Disulone®)Also: experimental armExperimental group will receive dapsone at a fixed dose of 100 mg per day for a total of 12 months unless they fail to respond to the treatment. | Drug | Dapsone: (Disulone®) |
| Prednisone (Cortancyl®) alone followed by monitoring and "standard of care"Also: control armPatients randomized in the control arm will be treated only prednisone at a daily dose of 1 mg per kg for 2 weeks and then tapered and stopped over a week for a total of 3 consecutive weeks. After this 3 weeks period, patients from arm B will be left without treatment and monitored. | Drug | Prednisone (Cortancyl®) alone and "standard of care" |
| Prednisone (Cortancyl®)Also: experimental armExperimental group will receive prednisone at a daily dose of 1 mg per kg for 2 weeks and then tapered and stopped over a week for a total of 3 consecutive weeks. | Drug | Dapsone: (Disulone®) |
Primary outcome
Overall response-rate.
Time frame: at week 52
6 secondary outcomes
Assess the safety of dapsone especially the incidence of cutaneous adverse effects.
Time frame: over the study period (up to 52 weeks)
Compare the overall response rate .
Time frame: at week 24
Proportion of patients with complete response in both arms, defined as follows: - Proportion of patients with a platelet count > 100 x 109/L in the absence use of any other ITP directed therapies
Time frame: at 24 and at 52 weeks
Proportion of non-responders patients (primary failure)
Time frame: at 24 and at 52 weeks
Number of days to treatment failure in both arms
Time frame: over the study period (up to 52 weeks)
Mechanisms of action of Dapsone: degree of phagocytosis of autologous platelets and red blood cells, cytokines profile expression (including IL-8), whole blood gene expression signature between responders and non-responders.
Time frame: over the study period (52 weeks)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2017.
- Background: PubMed 1873232Durand JM, Lefevre P, Hovette P, Mongin M, Soubeyrand J. Dapsone for idiopathic autoimmune thrombocytopenic purpura in elderly patients. Br J Haematol. 1991 Jul;78(3):459-60. doi: 10.1111/j.1365-2141.1991.tb04467.x. No abstract available.
- Background: PubMed 8342569Godeau B, Oksenhendler E, Bierling P. Dapsone for autoimmune thrombocytopenic purpura. Am J Hematol. 1993 Sep;44(1):70-2. doi: 10.1002/ajh.2830440117.
- Background: PubMed 7794776Hernandez F, Linares M, Colomina P, Pastor E, Cervero A, Perez A, Perella M. Dapsone for refractory chronic idiopathic thrombocytopenic purpura. Br J Haematol. 1995 Jun;90(2):473-5. doi: 10.1111/j.1365-2141.1995.tb05179.x.
- Background: PubMed 9163598Godeau B, Durand JM, Roudot-Thoraval F, Tenneze A, Oksenhendler E, Kaplanski G, Schaeffer A, Bierling P. Dapsone for chronic autoimmune thrombocytopenic purpura: a report of 66 cases. Br J Haematol. 1997 May;97(2):336-9. doi: 10.1046/j.1365-2141.1997.412687.x.
- Background: PubMed 16146539Damodar S, Viswabandya A, George B, Mathews V, Chandy M, Srivastava A. Dapsone for chronic idiopathic thrombocytopenic purpura in children and adults--a report on 90 patients. Eur J Haematol. 2005 Oct;75(4):328-31. doi: 10.1111/j.1600-0609.2005.00545.x.
- Derived from this study: PubMed 39874521Larue M, Moulis G, Rueter M, Audia S, Comont T, Terriou L, Viallard JF, Pan-Petesch B, Royer B, Bonnotte B, Galicier L, Lambotte O, Lefrere F, Cheze S, Ebbo M, Duong TA, Boutin E, Languille L, Mahevas M, Godeau B, Canoui-Poitrine F, Michel M. Efficacy and safety of dapsone in adult primary immune thrombocytopenia. Blood Adv. 2025 Apr 22;9(8):1976-1983. doi: 10.1182/bloodadvances.2024014939.
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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.
MARC MICHEL, Prof. M.D 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 3 study testing Dapsone (Disulone®) and Prednisone (Cortancyl®) in people with Adult Immune Thrombocytopenia (ITP) and Dapsone, 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 15 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: status not confirmed, as of the sponsor's last update on 26 Jul 2017. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact MARC MICHEL, Prof. M.D ((0)1 49 81 20 76 ext. +33, marc.michel2@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response-rate. (time frame: at week 52). 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 NCT02627417, which also lists 6 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 26 Jul 2017.