Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

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

Phase 1 single dose study of ALXN1101 in healthy volunteers.

Study Overview

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

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 Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21225
      • PAREXEL Baltimore EPCU

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: 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.	Drug: ALXN1101; Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.
PLACEBO_COMPARATOR: 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.	Drug: Placebo; Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability of single dose of ALXN1101 in healthy adult subjects	Physical examination, vital signs, ECGs, laboratory evaluations, and Adverse Events.	following the Day 30 visit for the last study subject

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PK parameters of ALXN1101	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).	following the Day 5 visit for the last study subject

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Exploratory biochemical marker assessments	To evaluate urine, serum or plasma concentrations of biochemical markers including S-sulfocysteine, xanthine, uric acid, creatinine, and other exploratory biochemical markers.	following the Day 30 visit for the last study subject

Collaborators and Investigators

• Sponsor: Origin Biosciences

Study record dates

Study Major Dates

• Study Start: June 1, 2013
• Primary Completion (ACTUAL): September 1, 2013
• Study Completion (ACTUAL): September 1, 2013

Study Registration Dates

• First Submitted: June 27, 2013
• First Submitted That Met QC Criteria: July 9, 2013
• First Posted (ESTIMATE): July 10, 2013

Study Record Updates

• Last Update Posted (ACTUAL): March 19, 2019
• Last Update Submitted That Met QC Criteria: March 15, 2019
• Last Verified: March 1, 2019

More Information

Terms related to this study

• Keywords: molybdenum cofactor deficiency (MoCD); deficiency of activity of molybdenum-dependent enzymes
• Other Study ID Numbers: ALXN1101-MCD-101