A Phase 2, Randomized, Double-blind, Placebo-Controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease
Status: Terminated
Location: See all (23) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD). The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:
• 18 to 75 years, inclusive, at the time of consent.
• Documented diagnosis of PiZZ-type alpha-1 antitrypsin deficiency, confirmed by genotyping. Historical genotyping data may be used, if available.
• AATD-associated liver disease documented by liver biopsy at Screening.
• Consent to undergo paired liver biopsies.
• Lung, renal and liver function within acceptable limits
• Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Locations
United States
California
University of California - San Diego
La Jolla
Florida
University of Florida
Gainesville
South Carolina
Medical University of South Carolina
Charleston
Other Locations
Australia
St Vincent's Hospital Melbourne
Melbourne
Austria
Medizinische Universitaet Innsbruck
Innsbruck
Belgium
Universitaire Ziekenhuizen Leuven
Leuven
Canada
Centre Hospitalier de l'Universite de Montreal (CHUM)
Montreal
France
CHU Bordeaux - Hopital Haut-Leveque - Centre François Magendie
Pessac
Germany
Universitaetsklinikum Aachen, AoeR
Aachen
Universitaetsklinikum Schleswig-Holstein Campus Kiel
Kiel
Ireland
Beaumont Hospital
Dublin
Netherlands
Leiden University Medical Center
Leiden
New Zealand
Auckland Clinical Studies
Grafton
Waikato Hospital
Hamilton
Portugal
Hospital da Senhora da Oliveira - Guimaraes
Creixomil
Centro Hospitalar Universitario de Sao Joao
Porto
Centro Hospitalar de Trás-os-Montes e Alto Douro, EPE
Vila Real
Spain
Hospital Universitario La Paz
Madrid
Hospital Universitario Marques de Valdecilla Santander
Santander
Sweden
CTC Clinical Trial Consultants AB Uppsala
Uppsala
United Kingdom
Addenbrooke's Hospital, Cambridge University
Cambridge
Leeds Teaching Hospitals NHS Trust
Leeds
Royal Free London NHS Foundation Trust, Royal Free Hospital