Thrombotic Thrombocytopenic PurpuraPhase 2PTTrituxNCT00907751
Rituximab in Adult Acquired Idiopathic Thrombotic Thrombocytopenic Purpura
A Phase 2 study testing rituximab in people with Thrombotic Thrombocytopenic Purpura, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Multicentric non-randomized phase II opened prospective study (10 centres involved). Primary endpoint: To evaluate the kinetics of B-cell depletion by rituximab and its pharmacokinetics in patients treated with rituximab in association with plasma exchanges. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura
- What is being tested?
- rituximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteFrance
- Study dates
- May 2010to Aug 2013
- 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
- Microangiopathic hemolytic anemia (< 12 g/dL) with thrombocytopenia <50 G/L, and mild or no renal failure (Serum creatinine < 150 µmol/L),
- negative Beta HCG and ongoing contraception during treatment and during the 24 months following the last infusion of rituximab,
- refractory TTP (after 4 days of standard treatment)
- > 18 year old
- and signed written informed consent.
Who cannot join
- Hemolytic uremic syndrome (platelet count ³ 50 G/L and serum creatinine ³ 150 micromol/L),
- TTP associated with another condition (HIV infection, cancer and/or chemotherapy, transplantation),
- previous treatment with vincristine or cyclophosphamide or other immunomodulatory drugs (except steroids), within 2 months before inclusion ;
- ongoing or planned pregnancy, lactation
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Association of Rituximab to Plasma Exchange in Adult Acquired Idiopathic Thrombotic Thrombocytopenic Purpura
Multicentric non-randomized phase II opened prospective study (10 centres involved).
Primary endpoint:
- To evaluate the kinetics of B-cell depletion by rituximab and its pharmacokinetics in patients treated with rituximab in association with plasma exchanges.
Secondary endpoints:
- To evaluate the tolerance of rituximab, the volume of plasma and the number of plasma exchange sessions required to achieve a durable complete remission, and to determinate the duration of B-cell depletion.
- To evaluate the incidence of persistent severe acquired ADAMTS13 deficiency following treatment with rituximab, as well as the incidence of relapses.
Read the detailed description
Duration of the study:
3 years and 2 months, including 1 year of inclusion and 2 years of participation for the patient.
Experimental plan:
Patients fulfilling the inclusion criteria of the study will be treated according to the recommendations of the Reference Centre for the management of thrombotic microangiopathies. If patients present refractory TTP, infusions of rituximab (375 mg/m2) will be added to this treatment at day 1, 4 and 15, immediately after plasma exchange sessions.On diagnosis, during treatment and after remission achievement, the following values will be explored: ADAMTS13 activity, ADAMTS13 inhibitors and anti-ADAMTS13 antibodies, B-cell lymphocytes quantification by immunophenotyping, and serum gammaglobulin level by serum protein electrophoresis. Rituximab will also be quantified.
Number of patients:
Each participating centre may recruit and include 1 patient/year.Amongst 10 participating centres, a total number of 10 patients should be included.
Specific activities during the study:
Three infusions of rituximab (375 mg/m2 /infusion), immediately after plasma exchange sessions on day 1, 4 and 15. Blood samplings at day 1, 4 and 15, at 1 month and then every 3 month until month 24.
Expected results and perspectives:
This study should provide evidence as to whether the association of rituximab to plasma exchanges allo an efficient B-cell depletion. If yes, a randomized study should be performed to evaluate the role of rituximab at the acute phase of acquired idiopathic TTP, immediately after the diagnosis was established.
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Being tested
- rituximab
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rituximabDrug
Patients will be treated according to the recommendations of the Reference Centre for the management of thrombotic microangiopathies. Infusions of rituximab (375 mg/m2) will be added to this treatment at day 1, 4 and 15, immediately after plasma exchange sessions.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Microangiopathic hemolytic anemia (< 12 g/dL) with thrombocytopenia (<50 G/L) Receives: Drug: rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the kinetics of B-cell depletion by rituximab and its pharmacokinetics in patients treated with rituximab in association with plasma exchanges
Measured: at 1, 3, 6, 9, 12, 18 and 24 months
Secondary outcomes (2)
To evaluate the tolerance of rituximab, the volume of plasma and the number of plasma exchange sessions required to achieve a durable complete remission, and to determinate the duration of B-cell depletion
Measured: at 1, 3, 6, 9, 12, 18 and 24 months
To evaluate the incidence of persistent severe acquired ADAMTS13 deficiency following treatment with rituximab, as well as the incidence of relapses.
Measured: at 1, 3, 6, 9, 12, 18 and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2009 (estimated)
- Study startMay 2010
- Primary completionAug 2013
- Study completionAug 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Thrombotic 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Paul COPPO, Md Ph D, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00907751
- Sponsor's protocol ID
- P060801
- Acronym
- PTTritux
- Status last verified
- Feb 2014
- First submitted
- 22 May 2009
- Last updated
- 27 Feb 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Benhamou Y, Paintaud G, Azoulay E, Poullin P, Galicier L, Desvignes C, Baudel JL, Peltier J, Mira JP, Pene F, Presne C, Saheb S, Deligny C, Rousseau A, Feger F, Veyradier A, Coppo P; French Reference Center for Thrombotic Microangiopathies. Efficacy of a rituximab regimen based on B cell depletion in thrombotic thrombocytopenic purpura with suboptimal response to standard treatment: Results of a phase II, multicenter noncomparative study. Am J Hematol. 2016 Dec;91(12):1246-1251. doi: 10.1002/ajh.24559. Epub 2016 Nov 8.
PubMed 27643485
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Association of Rituximab to Plasma Exchange in Adult Acquired Idiopathic Thrombotic Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT00907751
- Acronym / sponsor ID
- PTTritux / P060801
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- May 2010
- Primary completion
- Aug 2013 (actual)
- Study completion
- Aug 2013 (actual)
- First posted
- 25 May 2009 (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.
Multicentric non-randomized phase II opened prospective study (10 centres involved).
Primary endpoint:
- To evaluate the kinetics of B-cell depletion by rituximab and its pharmacokinetics in patients treated with rituximab in association with plasma exchanges.
Secondary endpoints:
- To evaluate the tolerance of rituximab, the volume of plasma and the number of plasma exchange sessions required to achieve a durable complete remission, and to determinate the duration of B-cell depletion.
- To evaluate the incidence of persistent severe acquired ADAMTS13 deficiency following treatment with rituximab, as well as the incidence of relapses.
Detailed description
Duration of the study:
3 years and 2 months, including 1 year of inclusion and 2 years of participation for the patient.
Experimental plan:
Patients fulfilling the inclusion criteria of the study will be treated according to the recommendations of the Reference Centre for the management of thrombotic microangiopathies. If patients present refractory TTP, infusions of rituximab (375 mg/m2) will be added to this treatment at day 1, 4 and 15, immediately after plasma exchange sessions.On diagnosis, during treatment and after remission achievement, the following values will be explored: ADAMTS13 activity, ADAMTS13 inhibitors and anti-ADAMTS13 antibodies, B-cell lymphocytes quantification by immunophenotyping, and serum gammaglobulin level by serum protein electrophoresis. Rituximab will also be quantified.
Number of patients:
Each participating centre may recruit and include 1 patient/year.Amongst 10 participating centres, a total number of 10 patients should be included.
Specific activities during the study:
Three infusions of rituximab (375 mg/m2 /infusion), immediately after plasma exchange sessions on day 1, 4 and 15. Blood samplings at day 1, 4 and 15, at 1 month and then every 3 month until month 24.
Expected results and perspectives:
This study should provide evidence as to whether the association of rituximab to plasma exchanges allo an efficient B-cell depletion. If yes, a randomized study should be performed to evaluate the role of rituximab at the acute phase of acquired idiopathic TTP, immediately after the diagnosis was established.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: rituximabMicroangiopathic hemolytic anemia (< 12 g/dL) with thrombocytopenia (<50 G/L) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Microangiopathic hemolytic anemia (< 12 g/dL) with thrombocytopenia <50 G/L, and mild or no renal failure (Serum creatinine < 150 µmol/L),
- negative Beta HCG and ongoing contraception during treatment and during the 24 months following the last infusion of rituximab,
- refractory TTP (after 4 days of standard treatment)
- > 18 year old
- and signed written informed consent.
Key exclusion criteria (4 listed)
- Hemolytic uremic syndrome (platelet count ³ 50 G/L and serum creatinine ³ 150 micromol/L),
- TTP associated with another condition (HIV infection, cancer and/or chemotherapy, transplantation),
- previous treatment with vincristine or cyclophosphamide or other immunomodulatory drugs (except steroids), within 2 months before inclusion ;
- ongoing or planned pregnancy, lactation
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 |
|---|---|---|
| rituximabPatients will be treated according to the recommendations of the Reference Centre for the management of thrombotic microangiopathies. Infusions of rituximab (375 mg/m2) will be added to this treatment at day 1, 4 and 15, immediately after plasma exchange sessions. | Drug | 1 |
Primary outcome
To evaluate the kinetics of B-cell depletion by rituximab and its pharmacokinetics in patients treated with rituximab in association with plasma exchanges
Time frame: at 1, 3, 6, 9, 12, 18 and 24 months
2 secondary outcomes
To evaluate the tolerance of rituximab, the volume of plasma and the number of plasma exchange sessions required to achieve a durable complete remission, and to determinate the duration of B-cell depletion
Time frame: at 1, 3, 6, 9, 12, 18 and 24 months
To evaluate the incidence of persistent severe acquired ADAMTS13 deficiency following treatment with rituximab, as well as the incidence of relapses.
Time frame: at 1, 3, 6, 9, 12, 18 and 24 months
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 27 Feb 2014.
- Derived from this study: PubMed 27643485Benhamou Y, Paintaud G, Azoulay E, Poullin P, Galicier L, Desvignes C, Baudel JL, Peltier J, Mira JP, Pene F, Presne C, Saheb S, Deligny C, Rousseau A, Feger F, Veyradier A, Coppo P; French Reference Center for Thrombotic Microangiopathies. Efficacy of a rituximab regimen based on B cell depletion in thrombotic thrombocytopenic purpura with suboptimal response to standard treatment: Results of a phase II, multicenter noncomparative study. Am J Hematol. 2016 Dec;91(12):1246-1251. doi: 10.1002/ajh.24559. Epub 2016 Nov 8.
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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.
Paul COPPO, Md Ph 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 2 study testing rituximab in people with Thrombotic 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 4 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 27 Feb 2014. 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 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?
To evaluate the kinetics of B-cell depletion by rituximab and its pharmacokinetics in patients treated with rituximab in association with plasma exchanges (time frame: at 1, 3, 6, 9, 12, 18 and 24 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 NCT00907751, which also lists 1 linked publication.
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 Feb 2014.