Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

Origin Biosciences

Status and phase: Completed; Phase 1

Conditions: Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase); Molybdenum Cofactor Deficiency (MoCD); Rare Autosomal Recessive Disorder

Treatments: Drug: ALXN1101; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01894165

ALXN1101-MCD-101

Details and patient eligibility

About

Phase 1 single dose study of ALXN1101 in healthy volunteers.

Full 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.

Enrollment: 24 patients

Sex: All

Ages: 18 to 60 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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24 participants in 2 patient groups, including a placebo group

ALXN1101

Experimental group

Description:

Four cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV.

Treatment:

Drug: ALXN1101

Placebo

Placebo Comparator group

Description:

Four cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV.

Treatment:

Drug: Placebo