Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase), Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder
Conditions
Keywords
molybdenum cofactor deficiency (MoCD), deficiency of activity of molybdenum-dependent enzymes
Brief summary
Phase 1 single dose study of ALXN1101 in healthy volunteers.
Detailed description
This is a first-in-human (FIH), randomized, blinded, placebo-controlled, single-dose, sequential-cohort, dose-escalation study to evaluate the safety, tolerability, and pharmacokinetics (PK) of a single dose of ALXN1101 in healthy adult subjects.
Interventions
DRUGALXN1101
Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.
DRUGPlacebo
Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.
Sponsors
Origin Biosciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
Yes
Inclusion criteria
Key Inclusion Criteria: 1. Male or female subjects ≥ 18 and ≤ 60 years of age and weight ≥ 55 kg and ≤ 100 kg 2. Willing and able to give written informed consent 3. Female subjects of child bearing potential must have a negative serum pregnancy test or must be practicing an approved contraceptive regimen for the duration of the study 4. Male subjects must be practicing an acceptable barrier method of contraception Key
Exclusion criteria
1. Pregnant or nursing female subjects 2. QTcF \> 450 msec for males and \> 470 msec for females, or a family history of Long QT Syndrome. 3. CrCl \< 80 mL/min 4. CBC in acceptable range; SGOT or SGPT above the ULN 5. HIV, Hepatitis B or Hepatitis C virus infection 6. Other active systemic infection or malignancy 7. Investigational drug study within 60 days 8. Major surgery within the prior 90 days 9. History of illicit drug use or chronic alcohol dependence within 2 years prior to this study 10. Positive urine drug toxicology screen or serum alcohol test 11. Alcohol consumption within 48 hours prior to study drug administration 12. Recently donated or lost ≥ 499 mL of blood 13. Recent hormone replacement therapy or use of prescription medications
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability of single dose of ALXN1101 in healthy adult subjects | following the Day 30 visit for the last study subject | Physical examination, vital signs, ECGs, laboratory evaluations, and Adverse Events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| PK parameters of ALXN1101 | following the Day 5 visit for the last study subject | PK parameters of ALXN1101 will be estimated including, but not limited to,maximum observed plasma concentration (Cmax), time to maximum observed plasma concentration (tmax), terminal elimination halflife (t½), area under the plasma concentration-time curve (AUC), total body clearance (CL), and volume of distribution (Vd). |
Other
| Measure | Time frame | Description |
|---|---|---|
| Exploratory biochemical marker assessments | following the Day 30 visit for the last study subject | To evaluate urine, serum or plasma concentrations of biochemical markers including S-sulfocysteine, xanthine, uric acid, creatinine, and other exploratory biochemical markers. |
Countries
United States
Outcome results
None listed