Trial results for pegcetacoplan in patients with transplant-associated thrombotic microangiopathy (TA-TMA) were posted on ClinicalTrials.gov on 2025-11-28. The study detailed pharmacokinetic (PK) parameters, including mean AUC0-tau values up to 36208.37 µg*h/mL.
Background
The study investigated pegcetacoplan for patients diagnosed with transplant-associated thrombotic microangiopathy after hematopoietic stem cell transplantation (HSCT). The trial aimed to assess the pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety profile of the drug in this patient population.
Trial design
The study (NCT05148299) was a Phase 2 trial that enrolled 12 participants diagnosed with transplant-associated thrombotic microangiopathy. The trial investigated pegcetacoplan as the intervention. The study's overall status is COMPLETED.
Key results
The trial detailed several pharmacokinetic parameters for pegcetacoplan:
- For "Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau)":
- Mean value of 14934.65 µg*h/mL (Standard Deviation: 4448.067)
- Mean value of 20089.79 µg*h/mL (Standard Deviation: 5162.653)
- Mean value of 36208.37 µg*h/mL (Standard Deviation: 10685.626)
- For "Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)":
- Mean value of 508.1 µg/mL (Standard Deviation: 134.86)
- Mean value of 579.6 µg/mL (Standard Deviation: 142.11)
- Mean value of 705.2 µg/mL (Standard Deviation: 203.04)
- For "Pegcetacoplan PK Parameter Time to Cmax (Tmax)":
- Median value of 1.0 Hour (Full Range)
- Median value of 1.02 Hour (Full Range)
- Median value of 1.05 Hour (Full Range)
- For "Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough)":
- Geometric Mean value of 365.22 µg/mL (95% Confidence Interval)
- Geometric Mean value of 449.86 µg/mL (95% Confidence Interval)
- Geometric Mean value of 585.71 µg/mL (95% Confidence Interval)
What this means
The posting of these pharmacokinetic results provides important data on the exposure and concentration profiles of pegcetacoplan in patients with transplant-associated thrombotic microangiopathy. These parameters are crucial for understanding the drug's behavior in the body and can inform future dosing strategies and further clinical development. While efficacy and safety outcomes were part of the study's purpose, the publicly available data primarily details the drug's pharmacokinetic characteristics.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05148299, titled "A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation," were posted on 2025-11-28 on clinicaltrials.gov.