Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

Phase 1

Completed

• Conditions: Molybdenum Cofactor Deficiency (MoCD); Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase); Rare Autosomal Recessive Disorder
• Interventions: Drug: Placebo; Drug: ALXN1101

• Registration Number: NCT01894165
• Lead Sponsor: Origin Biosciences

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.

Study Timeline

• Study Start: 2013-06
• Study First Submitted: 2013-06-27
• Study First Submitted QC: 2013-07-09
• Study First Posted: 2013-07-10
• Primary Completion: 2013-09
• Study Completion: 2013-09
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-15
• Last Update Posted: 2019-03-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	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.
ALXN1101	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.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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).

Trial Locations

Locations (1)

Parexel Baltimore EPCU; 🇺🇸 Baltimore, Maryland, United States