Autoimmune Thrombocytopenic PurpuraPhase 2NCT00225875
Study of the Effectiveness of Rituximab in Adults With Chronic and Severe Immune Thrombocytopenic Purpura and Candidate for a Splenectomy
A Phase 2 study testing Mabthéra in people with Autoimmune Thrombocytopenic Purpura, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The goal of this study is to evaluate the clinical effectiveness of the rituximab at the adults with a chronic immune thrombocytopenic purpura (>=6 months of evolution) and severe (platelets <= 30x109/L) and candidate to a splenectomy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Thrombocytopenic Purpura
- What is being tested?
- Mabthéra
- How many people
- 65 participants
- Where
- 1 siteFrance
- Study dates
- Sep 2003to Jul 2007
- Phase
- Phase 2
- 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
- Platelets <= 30x109/L in the absence of agglutinat
- Evolution of the PTAI >= 6 months starting from the date of the diagnosis
- Myélogramme normal and rich in mégacaryocytes
- Age >=18 years
- Among patients at which the treatments prescribed before (and in particular corticoids or intravenous immunoglobulins) did not have any effectiveness, even transitory, the diagnosis of ITP will have to be confirmed by an isotopic study of the 1/2 life of the plates.
Who cannot join
- Refusal of informed and enlightened assent written.
- Intermittent ITP defined by which has occurred of transitory periods of remissions variable length of the thrombocytopenia.
- Sick splenectomized whatever is the reason
- Splénomégalie
- Absence of vaccination against the pneumococcus
- Absence of vaccination against Haemophilus influenzae
Show all 19Show fewer
- Previous of treatment by the rituximab
- Administration of a treatment known as active during the ITP other than corticoids in the 30 days which precede inclusion
- CIVD and/or weakens haemolytic with schizocytes
- Serology VIH or positive VHC, Ag positive HBs
- Rate of ALAT or ASAT higher than twice the higher limit of the normal of the laboratory
- Associated autoimmune anomalies:
- Anti DNA and/or anti ECT (ENA) and/or anti Ro (SSA)
- The presence isolated from antibody anti cores (nuclear anti factors) is not a criterion of exclusion.
- Anticoagulant circulating of lupic type and/or antibody anticardiolipines with antecedent of thrombosis or spontaneous miscarriages with repetition (their isolated presence is not a criterion of exclusion)
- Other autoimmune diseases: lupus (with at least 4 criteria of the ACR), polyarthrite chronic evolutionary, disease of Biermer, affected thyroid, weakens haemolytic autoimmune.
- Pregnant woman, breast feeding, woman in genital working life in the effective absence of contraception throughout treatment and 12 month after stop of the treatment.
- Evolutionary or previous cancer of malignant hemopathy
- Over-sensitiveness with murine proteins
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation De l'Efficacité Du Rituximab (Mabthéra) Chez l'Adulte Atteint d'Un Purpura thrombopénique Auto-Immun Chronique Et sévère Et Candidat à La splénectomie
The goal of this study is to evaluate the clinical effectiveness of the rituximab at the adults with a chronic immune thrombocytopenic purpura (>=6 months of evolution) and severe (platelets <= 30x109/L) and candidate to a splenectomy. The objective is to obtain after a treatment by the rituximab a satisfactory response to one year, defined by a number of platelets higher than 50x109/L and at least 2 times superior with the persistent initial figure without treatment during one year after the end of the treatment.
Read the detailed description
Adults immune thrombocytopenic purpura has an evolution which is generally chronic defined by the persistence of the thrombocytopenia 6 months after the diagnosis. The treatment is then based on the splenectomy which is proposed by the majority of the teams when the platelets are lower than 30x109/L. The splenectomy is effective at 70 to 80 % of the patients whereas no medicamentous treatment makes it possible to obtain a comparable result. Nevertheless, it exposes to immediate post-operative complications and to a risk of mortal fulminant infections by encapsulated germs, in particular the pneumococcus. However, its long-term effectiveness is discussed with a risk of relapse which would reach 50 % for certain teams.
The rituximab could be an alternative to the splenectomy because of its great frequency of effectiveness and its good tolerance in the short and medium term. None the medicamentous treatments usually suggested in alternative to the splenectomy (disulone, danazol, immunosuppressors) indeed makes it possible to obtain an answer prolonged after the stop of therapeutic in a significant number of cases. Moreover, the use of the immunosuppressors such as the cyclophosphamide, the azathioprine or the ciclosporine appears contestable at this stage of the disease because of potential severity their side effects.The primary endpoint is satisfactory response to one year, defined by a figure of plates >=50x109/L and at least 2 times superior in the initial, and persistent figure without treatment during one year after the stop of the treatment by rituximab. Secondary objectives are incomplete response to one year, defined by a figure of platelets >= 30x109/L and < 50x109/L and at least twice the figure initial or > 50x109/L but lower than twice the persistent initial figure without treatment during one year after the end of the treatment by rituximab. Splenectomy at one year satisfactory Response to 2 years incomplete Response to 2 years Splenectomies at 2 years Tolerance of the treatment.
- Conditions
- Autoimmune Thrombocytopenic Purpura
- Being tested
- Mabthéra
- Keywords
- Autoimmune thrombocytopenic purpura, Rituximab
- 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.
MabthéraDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Satisfactory response to one year, platelets >=50x109/L and at least 2 times superior in the initial, and persistent figure without treatment during one year after the stop of the treatment.
Secondary outcomes (2)
Incomplete response to one year,platelets >= 30x109/L and < 50x109/L and at least twice the figure initial or > 50x109/L but lower than twice the persistent initial figure without treatment during one year after the end of the treatment.
Splenectomy at one year satisfactory Response to 2 years Incomplete Response to 2 years Splenectomy at 2 years Tolerance of the treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2005 (estimated)
- Study startSep 2003
- Study completionJul 2007
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 Autoimmune Thrombocytopenic PurpuraStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Etablissement Français du Sang
- Investigators
- Bertrand Godeau, Professor, Principal investigator, Henri Mondor University HospitalPhilippe Bierling, Professor, Principal investigator, EFS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00225875
- Sponsor's protocol ID
- PTAI
- Status last verified
- Mar 2005
- First submitted
- 20 Sep 2005
- Last updated
- 27 Oct 2005
- Results posted
- No
Publications
Michel M, Chanet V, Galicier L, Ruivard M, Levy Y, Hermine O, Oksenhendler E, Schaeffer A, Bierling P, Godeau B. Autoimmune thrombocytopenic purpura and common variable immunodeficiency: analysis of 21 cases and review of the literature. Medicine (Baltimore). 2004 Jul;83(4):254-263. doi: 10.1097/01.md.0000133624.65946.40.
PubMed 15232313Godeau B, Porcher R, Fain O, Lefrere F, Fenaux P, Cheze S, Vekhoff A, Chauveheid MP, Stirnemann J, Galicier L, Bourgeois E, Haiat S, Varet B, Leporrier M, Papo T, Khellaf M, Michel M, Bierling P. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood. 2008 Aug 15;112(4):999-1004. doi: 10.1182/blood-2008-01-131029. Epub 2008 May 7.
PubMed 18463354
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation De l'Efficacité Du Rituximab (Mabthéra) Chez l'Adulte Atteint d'Un Purpura thrombopénique Auto-Immun Chronique Et sévère Et Candidat à La splénectomie
- ClinicalTrials.gov ID
- NCT00225875
- Acronym / sponsor ID
- PTAI
- Conditions
- Autoimmune Thrombocytopenic Purpura
- Keywords
- Autoimmune thrombocytopenic purpura, Rituximab
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 65 participants
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Etablissement Français du Sang
- Study start
- Sep 2003
- Study completion
- Jul 2007
- First posted
- 26 Sep 2005 (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.
The goal of this study is to evaluate the clinical effectiveness of the rituximab at the adults with a chronic immune thrombocytopenic purpura (>=6 months of evolution) and severe (platelets <= 30x109/L) and candidate to a splenectomy. The objective is to obtain after a treatment by the rituximab a satisfactory response to one year, defined by a number of platelets higher than 50x109/L and at least 2 times superior with the persistent initial figure without treatment during one year after the end of the treatment.
Detailed description
Adults immune thrombocytopenic purpura has an evolution which is generally chronic defined by the persistence of the thrombocytopenia 6 months after the diagnosis. The treatment is then based on the splenectomy which is proposed by the majority of the teams when the platelets are lower than 30x109/L. The splenectomy is effective at 70 to 80 % of the patients whereas no medicamentous treatment makes it possible to obtain a comparable result. Nevertheless, it exposes to immediate post-operative complications and to a risk of mortal fulminant infections by encapsulated germs, in particular the pneumococcus. However, its long-term effectiveness is discussed with a risk of relapse which would reach 50 % for certain teams.
The rituximab could be an alternative to the splenectomy because of its great frequency of effectiveness and its good tolerance in the short and medium term. None the medicamentous treatments usually suggested in alternative to the splenectomy (disulone, danazol, immunosuppressors) indeed makes it possible to obtain an answer prolonged after the stop of therapeutic in a significant number of cases. Moreover, the use of the immunosuppressors such as the cyclophosphamide, the azathioprine or the ciclosporine appears contestable at this stage of the disease because of potential severity their side effects.The primary endpoint is satisfactory response to one year, defined by a figure of plates >=50x109/L and at least 2 times superior in the initial, and persistent figure without treatment during one year after the stop of the treatment by rituximab. Secondary objectives are incomplete response to one year, defined by a figure of platelets >= 30x109/L and < 50x109/L and at least twice the figure initial or > 50x109/L but lower than twice the persistent initial figure without treatment during one year after the end of the treatment by rituximab. Splenectomy at one year satisfactory Response to 2 years incomplete Response to 2 years Splenectomies at 2 years Tolerance of the treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Platelets <= 30x109/L in the absence of agglutinat
- Evolution of the PTAI >= 6 months starting from the date of the diagnosis
- Myélogramme normal and rich in mégacaryocytes
- Age >=18 years
- Among patients at which the treatments prescribed before (and in particular corticoids or intravenous immunoglobulins) did not have any effectiveness, even transitory, the diagnosis of ITP will have to be confirmed by an isotopic study of the 1/2 life of the plates.
Key exclusion criteria (19 listed)
- Refusal of informed and enlightened assent written.
- Intermittent ITP defined by which has occurred of transitory periods of remissions variable length of the thrombocytopenia.
- Sick splenectomized whatever is the reason
- Splénomégalie
- Absence of vaccination against the pneumococcus
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 |
|---|---|---|
| Mabthéra | Drug | – |
Primary outcome
Satisfactory response to one year, platelets >=50x109/L and at least 2 times superior in the initial, and persistent figure without treatment during one year after the stop of the treatment.
2 secondary outcomes
Incomplete response to one year,platelets >= 30x109/L and < 50x109/L and at least twice the figure initial or > 50x109/L but lower than twice the persistent initial figure without treatment during one year after the end of the treatment.
Splenectomy at one year satisfactory Response to 2 years Incomplete Response to 2 years Splenectomy at 2 years Tolerance of the treatment.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2005.
- Background: PubMed 15232313Michel M, Chanet V, Galicier L, Ruivard M, Levy Y, Hermine O, Oksenhendler E, Schaeffer A, Bierling P, Godeau B. Autoimmune thrombocytopenic purpura and common variable immunodeficiency: analysis of 21 cases and review of the literature. Medicine (Baltimore). 2004 Jul;83(4):254-263. doi: 10.1097/01.md.0000133624.65946.40.
- Derived from this study: PubMed 18463354Godeau B, Porcher R, Fain O, Lefrere F, Fenaux P, Cheze S, Vekhoff A, Chauveheid MP, Stirnemann J, Galicier L, Bourgeois E, Haiat S, Varet B, Leporrier M, Papo T, Khellaf M, Michel M, Bierling P. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood. 2008 Aug 15;112(4):999-1004. doi: 10.1182/blood-2008-01-131029. Epub 2008 May 7.
Related trials for Autoimmune Thrombocytopenic Purpura
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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.
Bertrand Godeau, Professor principal investigator
Henri Mondor University Hospital
Philippe Bierling, Professor principal investigator
EFS
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mabthéra in people with Autoimmune Thrombocytopenic Purpura, 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 5 inclusion and 19 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: stopped early, as of the sponsor's last update on 27 Oct 2005. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early. Results are not available in the source record.
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?
Satisfactory response to one year, platelets >=50x109/L and at least 2 times superior in the initial, and persistent figure without treatment during one year after the stop of the treatment.. 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 NCT00225875, 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 27 Oct 2005.