Clinical trial NCT01894165: Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers
Official title: A Phase 1, Randomized, Blinded, Placebo-Controlled, Single-Dose, Sequential-Cohort, Dose-Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ALXN1101 in Healthy Adult Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Molybdenum Cofactor Deficiency (MoCD)
Rare Autosomal Recessive Disorder
Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
Keywords: molybdenum cofactor deficiency (MoCD)
deficiency of activity of molybdenum-dependent enzymes
Lead sponsor: Origin Biosciences
Sponsor class: INDUSTRY
First posted: 2013-07-10
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Safety and tolerability of single dose of ALXN1101 in healthy adult subjects
timeFrame: following the Day 30 visit for the last study subject
Secondary outcomes:
measure: PK parameters of ALXN1101
timeFrame: following the Day 5 visit for the last study subject
Other outcomes:
measure: Exploratory biochemical marker assessments
timeFrame: following the Day 30 visit for the last study subject
Locations:
city: Baltimore
country: United States
facility: Parexel Baltimore EPCU
state: Maryland
Study officials:
affiliation: Parexel Baltimore Early Phase Clinical Unit
name: Ronald Goldwater, MD
role: PRINCIPAL_INVESTIGATOR
References: