Acquired thrombotic thrombocytopenic purpura
Conditions
Interventions
Trade Name: Mabthera
Product Name: Mabthera
Pharmaceutical Form: Concentrate for solution for infusion
Sponsors
University College London
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female, age>18 2. Antibody-mediated TTP with at least one previous acute TTP episode 3. ADAMTS13 activity dropping to =15% documented on 2 separate occasions on different days, , with platelets>150 and no evidence of microangiopathic haemolytic anaemia and LDH =65 years) yes F.1.3.1 Number of subjects for this age range 7
Exclusion criteria
Exclusion criteria: 1. Females who are pregnant, planning pregnancy or breastfeeding 2. Concurrent and/or recent involvement in other research that is likely to interfere with the intervention within 3 months of study enrolment 3. Known allergies to rituximab 4. Patients currently undergoing plasma infusion/exchange 5. Patients on therapy with other immunosuppressive medication (except steroids) 6. Patients with a current acute severe infection or a history of recurring or chronic infections or underlying conditions which may further predispose patient to serious infection.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: We know that a fall in ADAMTS13 activity in remission can be a sign of upcoming TTP relapse. We plan to see if low dose rituximab is as effective as the standard dose in preventing TTP relapse by looking at the length of time until patients need to be treated again with ritiuximab or other immunosuppression ;Secondary Objective: We will also look to see if low-dose rituximab is as effective as standard dose rituximab in: • the time it takes for ADAMTS13 activity to normalise after rituximab • how long ADAMTS13 stays normal after rituximab • subsequent relapse rates of patients with a clinical TTP episode • subsequent re-treatment rates of patients using rituximab or other immunosuppression and also whether there is a difference in side effects between the 2 doses, either related to the infusion itself or other rituximab-related adverse effects. ;Primary end point(s): The primary outcome will be time to re-treatment with rituximab/other immunosuppression measured in days from day of first rituximab infusion (D1)until D1 of any subsequent course of elective rituximab, or introduction of other immunosuppression initiated with the aim of preventing clinical relapse of TTP. This will be analysed as a time-to-event variable. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Time to normalisation of ADAMTS13 activity–number of days from D1 of first rituximab infusion until ADAMTS13 activity returns to normal range; this is a time-to-event outcome. Duration of ADAMTS13 response – number of days from normalisation of ADAMTS13 activity to ADAMTS13 activity dropping outside of remains in normal range; this is a time-to-event outcome. Subsequent relapse rate with clinical TTP episode (%) – the proportion of patients who experience a clinical relapse of TTP during trial follow-up compared to patients who do not; this is a binary outcome. Subsequent re-treatment rate with rituximab/other immunosuppression (%) – the proportion of patients who are retreated with rituximab or other immunosuppression, for the prevention of clinical relapse of TTP, during trial follow-up compared to patients who are not; this is a binary outcome. Time to B cell depletion – number of days from D1 to CD19 count <0.005x109/L0.5% ; this is a time-to-event outcome . Time to B cell return – number of days from D1 to CD19 count returning to normal range; this is a time-to-event outcome. Infusion-related adverse effects Delayed rituximab-related adverse effects eg serum sickness, abnormal LFTs, cytopenias, infections- not occurring during rituximab infusion | — |
Countries
United Kingdom
Contacts
Public ContactSponsor Regulatory Advisor
University College London Joint
Outcome results
None listed