Hemophilia APhase 1/2NCT07787091
A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
A Phase 1/2 study testing ARD001 in people with Hemophilia A and Healthy Adult Participants, run by Arditus Pte Ltd at 1 site in Singapore.
Trial 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… Read more about this study
- Who is it for?
- Ages 21 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Hemophilia A, Healthy Adult Participants
- What is being tested?
- ARD001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteSingapore
- Study dates
- Aug 2026to Mar 2027
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 45 years
- Men only
- Healthy volunteers accepted
- Adult
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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Singapore1 site, 1 recruiting
- Recruiting
SingHealth Investigational Medicine Unit
Singapore
Lavanya Lakshmi Jeeva, +65 63237544, lavanya.lakshmi.jeeva@singhealth.com.sg
Chuen Wen Tan, MB BCh BAO, FRCPath
About this study
Official title: 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.
- Conditions
- Hemophilia A, Healthy Adult Participants
- Being tested
- ARD001, Placebo
- Keywords
- ARD001, Single Ascending Dose, Healthy Volunteers, Hemophilia A, Coagulation
- Medical subject headings
- Hemophilia A, Thrombosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARD001Biological
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
PlaceboOther
The corresponding placebo will be administered subcutaneously as a single dose in Phase 1
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Biological: ARD001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Observed body weight values and changes from baseline will be summarized.
Measured: 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.
Measured: 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
Serum chemistry assessments include creatinine, blood urea nitrogen, sodium, potassium, magnesium, phosphate, calcium, chloride, bicarbonate, fasting blood glucose, high-density lipoprotein, low-density lipoprotein, triglycerides, alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase, total and direct bilirubin, thyroid-stimulating hormone, free thyroxine, lactate dehydrogenase, creatine kinase, C-reactive protein, and uric acid. The number of participants with clinically significant changes from baseline in serum chemistry parameters will be summarized. Clinical significance will be determined by the investigator.
Measured: 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
Urinalysis assessments include dipstick measurements of pH, protein, glucose, ketone, and blood, with reflex comprehensive urinalysis if abnormal as specified in the protocol. The number of participants with clinically significant changes from baseline in urinalysis parameters will be summarized. Clinical significance will be determined by the investigator.
Measured: 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
Coagulation assessments include prothrombin time (PT), international normalized ratio (INR), activated partial thromboplastin time (aPTT), fibrinogen, D-dimer, and thrombin time. The number of participants with clinically significant changes from baseline in coagulation parameters will be summarized. Clinical significance will be determined by the investigator.
Measured: 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.
Observed Values and Change From Baseline in Systolic Blood Pressure
Observed systolic blood pressure values and changes from baseline will be summarised.
Measured: 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.
Observed Values and Change From Baseline in Diastolic Blood Pressure
Measured: 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.
Observed Values and Change From Baseline in Pulse Rate
Measured: 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.
Observed Values and Change From Baseline in Respiratory Rate
Measured: 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.
Observed Values and Change From Baseline in Body Temperature
Measured: 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.
Observed Values and Change From Baseline in PR Interval
Observed PR interval values and changes from baseline obtained from protocol-specified 12-lead electrocardiograms will be summarised.
Measured: 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.
Observed Values and Change From Baseline in QRS Duration
Observed QRS duration values and changes from baseline obtained from protocol-specified 12-lead electrocardiograms will be summarised.
Measured: 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.
Observed Values and Change From Baseline in QT Interval
Observed uncorrected QT interval values and changes from baseline obtained from protocol-specified 12-lead electrocardiograms will be summarised.
Measured: 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.
Observed Values and Change From Baseline in QTcF Interval
Observed QT interval values corrected using Fridericia's formula and changes from baseline obtained from protocol-specified 12-lead electrocardiograms will be summarised.
Measured: 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.
Maximum Observed Serum Concentration of ARD001
Maximum observed serum concentration (Cmax) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Time to Maximum Observed Serum Concentration of ARD001
Area under the serum concentration-time curve from time zero to the last quantifiable concentration (AUC0-last) of ARD001 derived using noncompartmental analysis.
Measured: Time of maximum observed serum concentration (Tmax) of ARD001 derived using noncompartmental analysis.
Area Under the Serum Concentration-Time Curve Extrapolated to Infinity
Area under the serum concentration-time curve from time zero extrapolated to infinity (AUC0-inf) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Mean Residence Time From Time Zero to Infinity of ARD001
Mean residence time from time zero to infinity (MRT0-inf) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Lag Time Before the First Measurable Serum Concentration of ARD001
Time before the first measurable non-zero serum concentration (Tlag) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Terminal Elimination Rate Constant of ARD001
Apparent terminal elimination rate constant (λz) of ARD001 derived from the terminal portion of the serum concentration-time profile using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Apparent Terminal Elimination Half-Life of ARD001
Apparent terminal elimination half-life (t1/2) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Apparent Volume of Distribution During the Terminal Phase of ARD001
Apparent volume of distribution during the terminal phase following subcutaneous administration (Vz/F) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Apparent Clearance of ARD001
Apparent clearance following subcutaneous administration (CL/F) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Area Under the Serum Concentration-Time Curve From Time Zero to the Last Quantifiable Concentration
Area under the serum concentration-time curve from time zero to the last quantifiable concentration (AUC0-last) of ARD001 derived using noncompartmental analysis.
Measured: From pre-dose through protocol-specified pharmacokinetic sampling through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Number of Participants With Treatment-Emergent Anti-Drug Antibodies to ARD001
Number and percentage of participants with treatment-induced or treatment-boosted binding anti-drug antibodies to ARD001.
Measured: From baseline through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Time to First Detection of Anti-Drug Antibodies to ARD001
Time from first dose to the first scheduled post-baseline sample with a positive binding anti-drug antibody result.
Measured: From first dose through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Anti-Drug Antibody Titre to ARD001
Binding anti-drug antibody titre in participants with a positive anti-drug antibody result.
Measured: From baseline through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Persistence of Anti-Drug Antibodies to ARD001
Persistence of binding anti-drug antibodies to ARD001 based on positive results at protocol-specified post-dose assessment time points.
Measured: From baseline through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2026
- Study startAug 2026 (estimated)
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arditus Pte Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07787091
- Sponsor's protocol ID
- ARD001-01
- Status last verified
- Aug 2026
- First submitted
- 2 Aug 2026
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided