A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation

Part of paid clinical trials in Duarte, California.

Sponsor
Swedish Orphan Biovitrum
Study ID
NCT05148299
Phase
PHASE2
Status
Completed

Conditions

  • Transplant-Associated Thrombotic Microangiopathy

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.

Key Dates

Start date
Feb 1, 2022
Status verified
Sep 2025
Primary completion
Dec 8, 2024
Completion
Dec 8, 2024

Study Design

Enrollment
12 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Pegcetacoplan
    sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.

Primary Outcome Measure

Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) [ Time Frame: Week 1 ]

Locations (2)

FacilityCityStateZIPSite coordinators
City of HopeDuarteCalifornia91010-
Mayo Clinic - RochesterRochesterMinnesota55905-

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