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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00475423




Registration number
NCT00475423
Ethics application status
Date submitted
17/05/2007
Date registered
21/05/2007
Date last updated
23/02/2015

Titles & IDs
Public title
A Study of MabThera (Rituximab) in Patients With Idiopathic Thrombocytopenic Purpura.
Scientific 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.
Secondary ID [1] 0 0
ML20948
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Idiopathic Thrombocytopenic Purpura 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - rituximab [MabThera/Rituxan]

Experimental: 1 -


Treatment: Drugs: rituximab [MabThera/Rituxan]
1000mg iv on days 1 and 15

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
Week 8
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
Week 8
Secondary outcome [2] 0 0
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.
Timepoint [2] 0 0
Week 52
Secondary outcome [3] 0 0
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.
Timepoint [3] 0 0
Baseline to Week 52
Secondary outcome [4] 0 0
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.
Timepoint [4] 0 0
Week 52
Secondary outcome [5] 0 0
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.
Timepoint [5] 0 0
Baseline to Week 52
Secondary outcome [6] 0 0
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.
Timepoint [6] 0 0
Week 52
Secondary outcome [7] 0 0
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.
Timepoint [7] 0 0
Baseline to Week 52
Secondary outcome [8] 0 0
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.
Timepoint [8] 0 0
Week 8 to Week 52
Secondary outcome [9] 0 0
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.
Timepoint [9] 0 0
Week 8 to Week 52
Secondary outcome [10] 0 0
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.
Timepoint [10] 0 0
Week 8 to Week 52
Secondary outcome [11] 0 0
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.
Timepoint [11] 0 0
Week 8 to Week 52
Secondary outcome [12] 0 0
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.
Timepoint [12] 0 0
Week 52
Secondary outcome [13] 0 0
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.
Timepoint [13] 0 0
Baseline to Week 52
Secondary outcome [14] 0 0
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.
Timepoint [14] 0 0
Week 26 and Week 52
Secondary outcome [15] 0 0
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.
Timepoint [15] 0 0
Week 26 and Week 52
Secondary outcome [16] 0 0
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.
Timepoint [16] 0 0
Baseline, Weeks 1, 3, and 8, Follow-up Months 4, 6, 8, 10, and 12, and Last Day
Secondary outcome [17] 0 0
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.
Timepoint [17] 0 0
Weeks 3, 8 and Months 4, 6, 8, 10 and 12, and last day

Eligibility
Key inclusion criteria
- adult patients, >=18 years of age;

- refractory, relapsing or chronic idiopathic thrombocytopenic purpura;

- stable therapy during 3 weeks prior to study entry.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- newly diagnosed ITP (<6 weeks);

- prior treatment with MabThera;

- active bleeding requiring platelet transfusion within 7 days prior to entry into
study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
- Adelaide
Recruitment hospital [2] 0 0
- Gosford
Recruitment hospital [3] 0 0
- Randwick
Recruitment hospital [4] 0 0
- Sydney
Recruitment hospital [5] 0 0
- Westmead
Recruitment hospital [6] 0 0
- Woolloongabba
Recruitment hospital [7] 0 0
- Frankston
Recruitment hospital [8] 0 0
- Malvern
Recruitment hospital [9] 0 0
- Melbourne
Recruitment hospital [10] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
5011 - Adelaide
Recruitment postcode(s) [2] 0 0
2250 - Gosford
Recruitment postcode(s) [3] 0 0
NSW 2031 - Randwick
Recruitment postcode(s) [4] 0 0
2139 - Sydney
Recruitment postcode(s) [5] 0 0
2747 - Sydney
Recruitment postcode(s) [6] 0 0
2145 - Westmead
Recruitment postcode(s) [7] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [8] 0 0
5042 - Adelaide
Recruitment postcode(s) [9] 0 0
3199 - Frankston
Recruitment postcode(s) [10] 0 0
3144 - Malvern
Recruitment postcode(s) [11] 0 0
3168 - Melbourne
Recruitment postcode(s) [12] 0 0
3052 - Parkville

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Hoffmann-La Roche
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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. The anticipated
time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.
Trial website
https://clinicaltrials.gov/show/NCT00475423
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Hoffmann-La Roche
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications