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About
The purpose of the study is to assess PK, Pharmacodynamics (PD), Efficacy and safety of pegcetacoplan in patients with TA-TMA after HSCT.
Full description
This is a pilot study and the sample size is based on practical rather than statistical aspects.
A total of 12 patients will be included and treated in the study. With 12 patients included, it is estimated that 9 patients will complete at least 4 weeks of treatment, which is deemed sufficient to characterize the PK of pegcetacoplan in patients with TA-TMA to an appropriate precision. In addition, 12 patients will provide a 72 % probability to observe a response rate of at least 8 responders of the 12 patients recruited (assuming the true response rates is 70 %).
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
Received allogeneic HSCT.
Diagnosis of TA-TMA established, as per laboratory markers indicating TMA.
Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
Have rUPCR ≥ 1 mg/mg.
Women of childbearing potential, defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative serum pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last IMP dose.
Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
Men must agree to the following for the duration of the study and 8 weeks after their last dose of IMP:
Patient and/or legally authorized representative must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF.
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
12 participants in 1 patient group
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Central trial contact
Luis Lopez Lazaro, MD
Data sourced from clinicaltrials.gov
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