A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A

Condition(s) studied

Investigational drug(s) / intervention(s)

ARD001: ARD001 will be administered subcutaneously as a single dose of 0.01, 0.1, or 0.3 mg/kg in Phase 1. A multiple-dose regimen is planned for Phase 2 and will be specified in a protocol amendment before Phase 2 initiation

Placebo: The corresponding placebo will be administered subcutaneously as a single dose in Phase 1

Study summary

This study is evaluating ARD001, an investigational medicine being developed for the treatment of hemophilia A. In Phase 1, healthy adult participants will receive a single dose of ARD001 or placebo to assess its safety and tolerability, how it moves through the body, its effects on blood coagulation, and whether the body develops antibodies against it.

If the Phase 1 results support further study and the required regulatory and ethics approvals are obtained, Phase 2 will evaluate repeated doses of ARD001 in adults with hemophilia A. Phase 2 is not currently open for enrollment.

The study is designed to determine whether ARD001 can be administered with acceptable safety and to identify doses suitable for further clinical development.

Eligibility

Primary outcome measure(s)

• Phase 1: Number of Participants With Treatment-Emergent Adverse Events, Serious Adverse Events, and Adverse Events of Special Interest — From first dose through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
  Number and percentage of participants with treatment-emergent adverse events, serious adverse events, and adverse events of special interest will be summarised.

Trial sites (1)

Other trials for the same condition