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

NCT07787091 | Recruiting Phase 1 DMC

• 1 other identifier: ARD001-01
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hemophilia A; Healthy Adult Participants

Keywords

ARD001; Single Ascending Dose; Healthy Volunteers; Hemophilia A; Coagulation

Outcome Measures

Primary Outcomes (1)

• 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.

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

Secondary Outcomes (28)

• Observed Body Weight and Change From Baseline

  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

  At baseline and at 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 Serum Chemistry Parameters

  At baseline and at 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 Urinalysis Parameters

  At baseline and at 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 Coagulation Parameters

  At baseline and at protocol-specified post-dose assessment time points through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.

Study Arms (2)

Placebo

PLACEBO COMPARATOR

In Phase 1, healthy adult participants randomized to placebo will receive a single subcutaneous dose of the corresponding placebo within one of the three sequential dose cohorts. No placebo treatment is planned for Phase 2

Other: Placebo

ARD001

EXPERIMENTAL

In Phase 1, healthy adult participants randomized to ARD001 will receive a single subcutaneous dose of 0.01, 0.1, or 0.3 mg/kg in one of three sequential ascending-dose cohorts. In Phase 2, adults with hemophilia A are planned to receive multiple subcutaneous doses of ARD001. The Phase 2 dose and regimen will be defined in a protocol amendment before Phase 2 initiation

Biological: ARD001

Interventions

ARD001 BIOLOGICAL

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

ARD001

Placebo OTHER

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

Placebo

Eligibility Criteria

• Age: 21 Years - 45 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Arditus Pte Ltd: lead

Study Sites (1)

SingHealth Investigational Medicine Unit

Singapore, Singapore

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Thrombosis

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Embolism and Thrombosis; Vascular Diseases; Cardiovascular Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Phase 1 is blinded to participants, investigators and designated clinical care personnel. Designated unblinded pharmacy personnel will prepare and dispense study treatment. Phase 2 is planned to be open-label.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase 1 comprises three sequentially single-ascending dose cohorts in healthy adults, with participants randomized within each cohort to ARD001 or Placebo. Phase 2 is planned as an open-label, multiple-dose study in adults with Hemophilia A and will be further defined in a protocol amendment before initiation.

Study Record Dates

• First Submitted: August 2, 2026
• First Posted: August 26, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: August 26, 2026

Record last verified: 2026-08

Locations

SG (1)