Daratumumab in Immune-mediated Thrombotic Thrombocytopenic Purpura

NCT07513948 | Recruiting

• 1 other identifier: 6442
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Thrombotic Thrombocytopenic Purpura, Acquired

Keywords

daratumumab; thrombotic thrombocytopenic purpura; ADAMTS13

Outcome Measures

Primary Outcomes (1)

• Proportion of responders to daratumumab

  Number of patients responding to daratumumab / total number of patients treated with daratumumab (response defined as the achievement of ADAMTS13 activity levels above 20% of normal at any timepoint from the first daratumumab administration to 12 weeks after the last dose, without new additional immunosuppressants)

  From the first daratumumab administration to 12 weeks after the last dose

Secondary Outcomes (2)

• Safety of daratumumab

  From the first daratumumab administration to 24 weeks after

• Duration of response

  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.

Study Arms (1)

Adult patients with iTTP

Patients with a confirmed diagnosis of iTTP, aged ≥ 18 years, treated with daratumumab for iTTP

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study includes adult iTTP patients who were treated with daratumumab in the acute phase of disease (i.e., with plasma exchanges and/or caplacizumab ongoing) or during remission (i.e., due to ADAMTS13 relapse or ADAMTS13 activity plasma levels persistently \<20%) from January 2010 to 6 months before the time of data collection. It includes iTTP patients receiving daratumumab either for refractory disease or in ADAMTS13 remission but intolerant to other immune suppressive agents.

You may qualify if:

• 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.

You may not qualify if:

• patients unwilling or unable to provide their informed consent;
• follow-up \< 6 months after daratumumab administration.

Sponsors & Collaborators

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico: lead

Study Sites (1)

Angelo Bianchi Bonomi Hemophilia and Thrombosis Center

Milan, Italy

RECRUITING

MeSH Terms

Conditions

Thrombotic thrombocytopenic purpura, acquired; Purpura, Thrombotic Thrombocytopenic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Central Study Contacts

Juri A Giannotta, M.D.

CONTACT

+390255035273; juri.giannotta@policlinico.mi.it

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2026
• First Posted: April 7, 2026
• Study Start: October 1, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: April 14, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)