Idiopathic Thrombocytopenic PurpuraPhase 2NCT00475423
A Study of MabThera (Rituximab) in Patients With Idiopathic Thrombocytopenic Purpura.
A Phase 2 study testing rituximab [MabThera/Rituxan] in people with Idiopathic Thrombocytopenic Purpura, run by Hoffmann-La Roche at 12 sites in Australia.
Trial summary
This single arm study will evaluate the efficacy and safety of MabThera monotherapy in patients with refractory, relapsing or chronic idiopathic thrombocytopenic purpura (ITP). Patients will receive infusions of MabThera 1000mg i.v. on days 1 and 15. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Idiopathic Thrombocytopenic Purpura
- What is being tested?
- rituximab [MabThera/Rituxan]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 122 participants
- Where
- 12 sitesAustralia
- Study dates
- May 2007to Aug 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- adult patients, >=18 years of age;
- refractory, relapsing or chronic idiopathic thrombocytopenic purpura;
- stable therapy during 3 weeks prior to study entry.
Who cannot join
- newly diagnosed ITP (<6 weeks);
- prior treatment with MabThera;
- active bleeding requiring platelet transfusion within 7 days prior to entry into study.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
Australia12 sites
Study site
Adelaide, New South Wales, 5011
Study site
Adelaide, South Australia, 5042
Study site
Frankston, Victoria, 3199
Study site
Gosford, New South Wales, 2250
Study site
Malvern, Victoria, 3144
Study site
Melbourne, Victoria, 3168
Study site
Parkville, Victoria, 3052
Study site
Randwick, New South Wales, NSW 2031
Study site
Sydney, New South Wales, 2139
Study site
Sydney, New South Wales, 2747
Study site
Westmead, New South Wales, 2145
Study site
Woolloongabba, Queensland, 4102
About this study
Official title: An Open Label Study of a Fixed Dose Regimen of MabThera on Overall Response Rate in Patients With Refractory, Relapsing or Chronic Idiopathic Thrombocytopenic Purpura.
This single arm study will evaluate the efficacy and safety of MabThera monotherapy in patients with refractory, relapsing or chronic idiopathic thrombocytopenic purpura (ITP). Patients will receive infusions of MabThera 1000mg i.v. on days 1 and 15. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Being tested
- rituximab [MabThera/Rituxan]
- 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.
rituximab [MabThera/Rituxan]Drug
1000mg iv on days 1 and 15
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: rituximab [MabThera/Rituxan] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Achieving a Complete Hematological Response (CR) or Confirmed Partial Hematological Response (PR)
Percentage of participants with an overall response at Week 8 achieving a CR or PR as evaluated by platelets, new or increased Idiopathic Thrombocytopenic Purpura (ITP)-related treatments and corticosteroids given for Adverse Events (AEs). CR was defined as a platelet count of greater than (>) 150x10^9/ liters (L) over at least 2 consecutive measurements at least 2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy. PR was defined as platelet count of >50x10^9/L over at least 2 consecutive measurements at least <2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy. Overall response rate (participants who achieved CR or confirmed PR) was evaluated using platelets, new or increased ITP related treatments, and corticosteroids given for AEs.
Measured: Week 8
Secondary outcomes (17)
Percentage of Participants With Hematological CR, PR, or Minor Response (MR)
Percentage of participants with CR, PR, and MR at Week 8 as evaluated by platelet count where CR is greater than or equal to (≥)150x10^9/L, PR ≥ 50x10^9/L, MR equals (=) 30x10^9/L over 2 consecutive measurements at least 2 weeks apart but no more than 60 days apart with no increase in concomitant therapy or initiation of new ITP therapy.
Measured: Week 8
Percentage of Participants Who Achieved CR
CR was defined as platelet counts >150x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, and with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Week 52
Time to CR
Time to CR was defined as the time from the first infusion to the first date on which CR was achieved. CR was defined as platelet counts >150x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, and with no increase in concomitant ITP therapy or initiation of new ITP therapy. Participants without an event were censored at the date of last assessment.
Measured: Baseline to Week 52
Percentage of Participants Who Achieved PR
PR was defined as platelet counts >50x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Week 52
Time to PR
Time to response was defined as the time from the first infusion to the first date on which PR was achieved. PR was defined as platelet counts > 50x10^9/L over ≥ 2 consecutive measurements ≥ 2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Baseline to Week 52
Percentage of Participants Who Achieved MR
MR was defined as participants registered with chronic ITP with a platelet count of >30x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, with a 50 to 100% reduction in the dose intensity of concomitant ITP therapy compared with that at screening, and with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Week 52
Time to MR
Time to response was defined as the time from the first infusion to the first date on which MR was achieved. MR was defined as participants registered with chronic ITP with a platelet count of >30x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, with a 50 percent (%) to 100% reduction in the dose intensity of concomitant ITP therapy compared with that at screening, and with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Baseline to Week 52
Percentage of Participants With Continued CR From Week 8 to Week 52
The number of participants with a durable CR assessed in CR responders whose responses were sustained from Week 8 through to the end of the study or withdrawal, irrespective of change of treatment. CR was defined as platelet counts >150x10^9/L over ≥2 consecutive measurements ≥ 2 weeks apart, but no more than 60 days apart, and with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Week 8 to Week 52
Duration of CR in Participants With Continued CR From Week 8 Until Week 52
Duration of response was assessed in all responders who reached Week 8 and was defined as the time from Week 8 to the end of CR, irrespective of change of treatment. CR was defined as platelet counts >150x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, and with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Week 8 to Week 52
Duration of PR in Participants With Continued PR From Week 8 Until Week 52
Duration of PR was assessed in all responders who reached Week 8 and was defined as the time from Week 8 to the end of PR, irrespective of change of treatment. PR was defined as platelet counts >50x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Week 8 to Week 52
Duration of MR in Participants With Continued MR From Week 8 Until Week 52
Duration of response was assessed in all responders who reached Week 8 and was defined as the time from Week 8 to the end of MR, irrespective of change of treatment. MR was defined as participants registered with ITP in relapse with a platelet count of > 30x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy. Participants registered with chronic ITP with a platelet count of >30x10^9/L over ≥2 consecutive measurements ≥2 weeks apart, but no more than 60 days apart, with a 50% to 100% reduction in the dose intensity of concomitant ITP therapy compared with that at screening, and with no increase in concomitant ITP therapy or initiation of new ITP therapy.
Measured: Week 8 to Week 52
Time to Inititiation of New ITP Therapy - Percentage of Participants With an Event
Percentage of participants with an event of initiation of new ITP therapy and/or increase in dose of existing ITP therapy, including date on which decision made in relation to splenectomy, from time of first treatment to Week 52.
Measured: Week 52
Time to Initiation of New ITP Therapy
Median time in days to initiation of new ITP therapy and/or increase in dose of existing ITP therapy, including date on which decision made in relation to splenectomy, from time of first treatment to Week 52.
Measured: Baseline to Week 52
Percentage of Therapeutic Responders
Percentage of participants with a therapeutic response, defined as achieving CR, PR, or MR assessed at Week 26 and Week 52 or hematological response, defined as achieving CR, PR, or MR at Week 8. CR was defined as no platelet response or no reduction in the dose intensity of concomitant ITP therapy compared with that at screening. PR response was defined as at least a minor platelet response that enabled a 50% to 99% reduction in the dose intensity of concomitant ITP therapy compared with that at screening. MR was defined as at least a minor platelet response that enabled a 1% to 49% reduction in the dose intensity of concomitant ITP therapy compared with that at screening.
Measured: Week 26 and Week 52
Percentage of Participants With a Therapeutic Response
Number of therapeutic responder participants by CR, PR, MR, or no response (NR) measured at Week 26 and Week 52. CR was defined as no platelet response or no reduction in the dose intensity of concomitant ITP therapy compared with that at screening. PR was defined as at least minor platelet response that enabled a 50% to 99% reduction in the dose intensity of concomitant ITP therapy compared with that at screening. MR was defined as at least minor platelet response that enabled a 1% to 49% reduction in the dose intensity of concomitant ITP therapy compared with that at screening.
Measured: Week 26 and Week 52
Cluster of Differentiation 19 (CD19) B Cell Count
Value of mean CD19+ B cell count at baseline. The standard reference range for CD19 is 0.05 to 0.35 x10^9/L.
Measured: Baseline, Weeks 1, 3, and 8, Follow-up Months 4, 6, 8, 10, and 12, and Last Day
Change From Baseline in CD19 B Cell Count
Actual values of CD19+ mean B cell count assessed at Weeks 3 and 8, and Months 4, 6, 8, 10 and 12, and last day. The standard reference range for CD19 is 0.05 to 0.35 x10^9/L.
Measured: Weeks 3, 8 and Months 4, 6, 8, 10 and 12, and last day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2007 (estimated)
- Study startMay 2007
- Primary completionAug 2011
- Study completionAug 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Idiopathic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe 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
- 122 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00475423
- Sponsor's protocol ID
- ML20948
- Status last verified
- Feb 2015
- First submitted
- 17 May 2007
- Last updated
- 23 Feb 2015
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study of a Fixed Dose Regimen of MabThera on Overall Response Rate in Patients With Refractory, Relapsing or Chronic Idiopathic Thrombocytopenic Purpura.
- ClinicalTrials.gov ID
- NCT00475423
- Acronym / sponsor ID
- ML20948
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 122 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- May 2007
- Primary completion
- Aug 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 21 May 2007 (estimated)
- Results
- Posted 23 Feb 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This single arm study will evaluate the efficacy and safety of MabThera monotherapy in patients with refractory, relapsing or chronic idiopathic thrombocytopenic purpura (ITP). Patients will receive infusions of MabThera 1000mg i.v. on days 1 and 15. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: rituximab [MabThera/Rituxan] |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- adult patients, >=18 years of age;
- refractory, relapsing or chronic idiopathic thrombocytopenic purpura;
- stable therapy during 3 weeks prior to study entry.
Key exclusion criteria (3 listed)
- newly diagnosed ITP (<6 weeks);
- prior treatment with MabThera;
- active bleeding requiring platelet transfusion within 7 days prior to entry into study.
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 |
|---|---|---|
| rituximab [MabThera/Rituxan]1000mg iv on days 1 and 15 | Drug | 1 |
Primary outcome
Percentage of Participants Achieving a Complete Hematological Response (CR) or Confirmed Partial Hematological Response (PR)
Time frame: Week 8
Percentage of participants with an overall response at Week 8 achieving a CR or PR as evaluated by platelets, new or increased Idiopathic Thrombocytopenic Purpura (ITP)-related treatments and corticosteroids given for Adverse Events (AEs). CR was defined as a platelet count of greater than (>) 150x10^9/ liters (L) over at least 2 consecutive measurements at least 2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy. PR was defined as platelet count of >50x10^9/L over at least 2 consecutive measurements at least <2 weeks apart, but no more than 60 days apart, with no increase in concomitant ITP therapy or initiation of new ITP therapy. Overall response rate (participants who achieved CR or confirmed PR) was evaluated using platelets, new or increased ITP related treatments, and corticosteroids given for AEs.
17 secondary outcomes
Percentage of Participants With Hematological CR, PR, or Minor Response (MR)
Time frame: Week 8
Percentage of Participants Who Achieved CR
Time frame: Week 52
Time to CR
Time frame: Baseline to Week 52
Percentage of Participants Who Achieved PR
Time frame: Week 52
Time to PR
Time frame: Baseline to Week 52
Percentage of Participants Who Achieved MR
Time frame: Week 52
Time to MR
Time frame: Baseline to Week 52
Percentage of Participants With Continued CR From Week 8 to Week 52
Time frame: Week 8 to Week 52
Duration of CR in Participants With Continued CR From Week 8 Until Week 52
Time frame: Week 8 to Week 52
Duration of PR in Participants With Continued PR From Week 8 Until Week 52
Time frame: Week 8 to Week 52
Duration of MR in Participants With Continued MR From Week 8 Until Week 52
Time frame: Week 8 to Week 52
Time to Inititiation of New ITP Therapy - Percentage of Participants With an Event
Time frame: Week 52
Time to Initiation of New ITP Therapy
Time frame: Baseline to Week 52
Percentage of Therapeutic Responders
Time frame: Week 26 and Week 52
Percentage of Participants With a Therapeutic Response
Time frame: Week 26 and Week 52
Cluster of Differentiation 19 (CD19) B Cell Count
Time frame: Baseline, Weeks 1, 3, and 8, Follow-up Months 4, 6, 8, 10, and 12, and Last Day
Change From Baseline in CD19 B Cell Count
Time frame: Weeks 3, 8 and Months 4, 6, 8, 10 and 12, and last day
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Clinical Trials study director
Hoffmann-La Roche
12 sites 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 [MabThera/Rituxan] in people with Idiopathic Thrombocytopenic Purpura, run by Hoffmann-La Roche at 12 sites in Australia.
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 3 inclusion and 3 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 23 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Australia. 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?
Yes. Results are posted in the registry (first posted 23 Feb 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants Achieving a Complete Hematological Response (CR) or Confirmed Partial Hematological Response (PR) (time frame: Week 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Feb 2015.