Daratumumab in Immune-mediated Thrombotic Thrombocytopenic Purpura
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Thrombotic Thrombocytopenic Purpura, Acquired. Basic parameters: 18 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
DarTTP: an Observational, International, Multicentric Study on Daratumumab in Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Overview
Data about efficacy and safety of daratumumab in iTTP refractory or intolerant to standard immunosuppressive treatments are scarce. Therefore, the investigators aim at collecting evidence on a larger number of patients through a collaborative, international study.
Detailed description
iTTP in an autoimmune disease caused by autoantibodies directed against the metalloproteinase ADAMTS13. Rituximab is the standard immune suppressive treatment suggested from international guidelines. However, 10-15% of patients do not achieve a sustained ADAMTS13 remission with rituximab, and a significant portion of responders eventually need re-treatment after 12 months or less. Other therapeutic options are scarce and based on old immunosuppressive agents or splenectomy, all burdened by relevant toxicity and lack of solid efficacy data. Recently, targeting CD20-negative long-lived plasma cells appears to be a promising strategy in refractory iTTP. The anti-CD38 monoclonal antibody daratumumab has been employed in selected iTTP patients with good results. However, evidence stems only from isolated case reports.
The DarTTP study aims to collect evidence on a larger number of patients about the efficacy and safety of daratumumab in iTTP subjects who are refractory or intolerant to previous immunosuppressive treatments. The primary endpoint is the proportion of patients with ADAMTS13 activity levels above 20% of normal at 6 months from the first daratumumab administration.
Primary outcome measures
- Proportion of responders to daratumumab [Time frame: From the first daratumumab administration to 12 weeks after the last dose]
Secondary outcome measures (2)
- Safety of daratumumab [Time frame: From the first daratumumab administration to 24 weeks after]
- Duration of response [Time frame: From the date of the first documented ADAMTS13 activity >20% after the first dose of daratumumab until the date of ADAMTS13 relapse (i.e., ADAMTS13 activity <20%), assessed up to 3 years.]
Eligibility criteria
Inclusion criteria
- patients with a confirmed diagnosis of iTTP (i.e., ADAMTS13 activity <10% with anti-ADAMTS13 antibodies detected);
- aged ≥ 18 years;
- male and female patients;
- treated with daratumumab for iTTP.
Exclusion criteria
- patients unwilling or unable to provide their informed consent;
- follow-up < 6 months after daratumumab administration.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
Italy · 1 center
- Angelo Bianchi Bonomi Hemophilia and Thrombosis Center — Milan
Identifiers
NCT: NCT07513948 · 6442