Follow-up Study for Patients Who Completed Study ALX0681-C301 (Post-HERCULES)

Sanofi

Status and phase: Completed; Phase 3

Conditions: Acquired Thrombotic Thrombocytopenic Purpura

Treatments: Biological: Caplacizumab; Other: Standard of Care

Study type: Interventional

Funder types: Industry

Identifiers

NCT02878603

2016-001503-23 (EudraCT Number)

LTS16371

ALX0681-C302 (Other Identifier)

Details and patient eligibility

About

The objectives of this study were to evaluate long-term safety and efficacy of caplacizumab, to evaluate safety and efficacy of repeated use of caplacizumab and to characterize long-term impact of acquired thrombotic thrombocytopenic purpura (aTTP).

Enrollment: 104 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Completed the Final (28 day) follow-up visit in Study ALX0681-C301.
2. >= 18 years of age at the time of signing the informed consent form.
3. Provided informed consent prior to initiation of any study specific activity/procedure.

Exclusion criteria

1. Not being able/willing to comply with the study protocol procedures.
2. Currently enrolled in a clinical study with another investigational drug or device.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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104 participants in 1 patient group

caplacizumab

Experimental group

Description:

Participants who completed study ALX0681-C301 (NCT02553317) with standard of care (plasma exchange \[PE\], corticosteroid and other immunosuppressive agents) or caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product \[IMP\], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 milligrams (mg) intravenous dose followed by a daily 10 mg subcutaneous injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Treatment:

Biological: Caplacizumab

Other: Standard of Care