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

Phase 2RecruitingNCT07787091

Arditus Pte Ltd20 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Hemophilia A Healthy Adult Participants

Interventions

Eligibility

Sex: MALEMin age: 21 YearsMax age: 45 YearsHealthy volunteers:

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Phase 1: Number of Participants With Treatment-Emergent Adverse Events, Serious Adverse Events, and Adverse Events of Special Interest

Number and percentage of participants with treatment-emergent adverse events, serious adverse events, and adverse events of special interest will be summarised.

Time frame: From first dose through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.

Secondary Outcomes

Observed Body Weight and Change From Baseline

Observed body weight values and changes from baseline will be summarized.

Time frame: Baseline and protocol-specified post-dose assessment time points through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.

Number of Participants With Clinically Significant Changes From Baseline in Hematology Parameters

Hematology assessments include white blood cell count with differential (neutrophils, lymphocytes, monocytes, eosinophils and basophils), red blood cell count and indices (mean corpuscular volume, mean corpuscular hemoglobin and mean corpuscular hemoglobin concentration), hemoglobin, and platelet count. The number of participants with clinically significant changes from baseline in hematology parameters will be summarized. Clinical significance will be determined by the investigator.