Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

NCT01894165 | Completed Phase 1

• 1 other identifier: ALXN1101-MCD-101
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

Phase 1 single dose study of ALXN1101 in healthy volunteers.

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• PK parameters of ALXN1101

  following the Day 5 visit for the last study subject

Other Outcomes (1)

• Exploratory biochemical marker assessments

  following the Day 30 visit for the last study subject

Study Arms (2)

ALXN1101

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Interventions

ALXN1101 DRUG

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

ALXN1101

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female subjects ≥ 18 and ≤ 60 years of age and weight ≥ 55 kg and ≤ 100 kg
• Willing and able to give written informed consent
• 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
• Male subjects must be practicing an acceptable barrier method of contraception

You may not qualify if:

• Pregnant or nursing female subjects
• QTcF \> 450 msec for males and \> 470 msec for females, or a family history of Long QT Syndrome.
• CrCl \< 80 mL/min
• CBC in acceptable range; SGOT or SGPT above the ULN
• HIV, Hepatitis B or Hepatitis C virus infection
• Other active systemic infection or malignancy
• Investigational drug study within 60 days
• Major surgery within the prior 90 days
• History of illicit drug use or chronic alcohol dependence within 2 years prior to this study
• Positive urine drug toxicology screen or serum alcohol test
• Alcohol consumption within 48 hours prior to study drug administration
• Recently donated or lost ≥ 499 mL of blood
• Recent hormone replacement therapy or use of prescription medications

Sponsors & Collaborators

• Origin Biosciences: lead

Study Sites (1)

Parexel Baltimore EPCU

Baltimore, Maryland, 21225, United States

Location

MeSH Terms

Conditions

Molybdenum cofactor deficiency

Interventions

nulibry

Study Officials

• Ronald Goldwater, MD

  Parexel Baltimore Early Phase Clinical Unit

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2013
• First Posted: July 10, 2013
• Study Start: June 1, 2013
• Primary Completion: September 1, 2013
• Study Completion: September 1, 2013
• Last Updated: March 19, 2019

Record last verified: 2019-03

Locations

US (1)