Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharma International Sarl
ClinicalTrials.gov Identifier:
NCT01894165
First received: June 27, 2013
Last updated: November 5, 2013
Last verified: November 2013
  Purpose

Phase 1 single dose study of ALXN1101 in healthy volunteers.


Condition Intervention Phase
Molybdenum Cofactor Deficiency (MoCD)
Rare Autosomal Recessive Disorder
Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
Drug: ALXN1101
Drug: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Alexion Pharma International Sarl:

Primary Outcome Measures:
  • Safety and tolerability of single dose of ALXN1101 in healthy adult subjects [ Time Frame: following the Day 30 visit for the last study subject ] [ Designated as safety issue: Yes ]
    Physical examination, vital signs, ECGs, laboratory evaluations, and Adverse Events.


Secondary Outcome Measures:
  • PK parameters of ALXN1101 [ Time Frame: following the Day 5 visit for the last study subject ] [ Designated as safety issue: No ]
    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).


Other Outcome Measures:
  • Exploratory biochemical marker assessments [ Time Frame: following the Day 30 visit for the last study subject ] [ Designated as safety issue: No ]
    To evaluate urine, serum or plasma concentrations of biochemical markers including S-sulfocysteine, xanthine, uric acid, creatinine, and other exploratory biochemical markers.


Enrollment: 24
Study Start Date: June 2013
Study Completion Date: September 2013
Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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.

  Eligibility

Ages Eligible for Study:   18 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01894165

Locations
United States, Maryland
Parexel Baltimore EPCU
Baltimore, Maryland, United States, 21225
Sponsors and Collaborators
Alexion Pharma International Sarl
Investigators
Principal Investigator: Ronald Goldwater, MD Parexel Baltimore Early Phase Clinical Unit
  More Information

No publications provided

Responsible Party: Alexion Pharma International Sarl
ClinicalTrials.gov Identifier: NCT01894165     History of Changes
Other Study ID Numbers: ALXN1101-MCD-101
Study First Received: June 27, 2013
Last Updated: November 5, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Alexion Pharma International Sarl:
molybdenum cofactor deficiency (MoCD)
deficiency of activity of molybdenum-dependent enzymes

Additional relevant MeSH terms:
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on April 16, 2014