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

A Phase 1 Randomized, Double-Blind, Placebo-Controlled, Single-Ascending-Dose Study Investigating the Safety and Tolerability of ARD001 in Healthy Adult Subjects and a Phase 2 Open-Label, Multiple-Dose Study in Adult Subjects With Hemophilia A

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.

Phase 1 Inclusion Criteria:

• Healthy adults within the specified age and body-weight ranges
• Able and willing to provide informed consent
• Considered healthy based on medical history, physical examination, vital signs, electrocardiogram and laboratory assessments
• Other essential criteria from the approved protocol

Phase 1 Exclusion Criteria:

• History of severe drug or food allergy, or known allergy to ARD001 or its components
• Acute infection within 14 days before dosing or fever on the planned dosing day
• Clinically significant cardiovascular, coagulation, hepatic, renal or other medical abnormality
• Recent use of prohibited medications within the protocol-defined washout periods
• Other major exclusions from the approved protocol