A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexMale
Age21-45
SponsorArditus Pte Ltd
About this trial
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 criteria
This trial accepts healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
ARD001
Biological/VaccineARD001 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
Other interventionThe corresponding placebo will be administered subcutaneously as a single dose in Phase 1
Treatment groups
20 Participants
are divided into 2 treatment groupsGroup A: PlaceboPlacebo comparator 1 intervention
Group B: ARD001Experimental treatment 1 intervention
Trial outcomes
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.
Time frame
From first dose through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.Secondary outcomes
1
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 A3.2
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.
Time frame
At baseline and at protocol-specified post-dose assessment time points through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.3
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.
Time frame
At baseline and at protocol-specified post-dose assessment time points through Day 29 for Cohort A1 and Day 113 for Cohorts A2 and A3.4
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.
Time frame
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.Sponsors and contacts
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Source ClinicalTrials.gov