Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

NCT ID: NCT01894165

Last Updated: 2019-03-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-06-30
• Study Completion Date: 2013-09-30

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.

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

ALXN1101

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.

Group Type: EXPERIMENTAL

ALXN1101

Intervention Type: DRUG

Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.

Placebo

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.

Interventions

ALXN1101

Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.

Intervention Type: DRUG

Placebo

Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.

Intervention Type: DRUG

Eligibility Criteria

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

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Origin Biosciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ronald Goldwater, MD

Role: PRINCIPAL_INVESTIGATOR

Parexel Baltimore Early Phase Clinical Unit

Locations

Parexel Baltimore EPCU

Baltimore, Maryland, United States

Countries

United States

Other Identifiers

ALXN1101-MCD-101

Identifier Type: -

Identifier Source: org_study_id