Trial information
REPAIR Tier 2 - (Retinoids in Emphysema Patients on the Alpha-1-Antitrypsin International Registry)
Status: No longer recruiting
Protocol number: NA17598
Sponsor: Hoffmann-La Roche Ltd/Inc/AG/Roche Global Development
Company division: Pharmaceutical
Official Scientific Title: A randomized, double-blind study of the effect of RAR Gamma on lung function in patients with symptomatic emphysema secondary to alpha-1 antitrypsin deficiency
Brief summary: This is a placebo-controlled trial to investigate the efficacy, safety, and tolerability of 5mg RAR Gamma administered once daily in patients with symptomatic emphysema secondary to alpha-1 antitrypsin deficiency. The anticipated time on study treatment is 3-12 months and the target sample size is 100-500 individuals.
Target sample size is
300.
Study phase: II
Study type: Interventional; Treatment; Randomized; Double Blind; Placebo; Parallel; Safety/Efficacy study
Condition: Emphysema
Intervention type: Drug
Intervention name: RAR Gamma
Primary outcome: 1. Efficacy: Change from baseline in lung density adjusted for lung volume
Key secondary outcomes: 1. Efficacy: Spirometry, arterial blood gas, gas transfer, TLco, lung volume, exercise test, dyspnea index, QoL, plasma biomarkers
Safety: Adverse events, skin effects, serum lipids, liver enzymes, psychiatric effects, and other retinoid side effects
Inclusion criteria:
- men >=30 years of age;
- women >=30 years of age, and beyond child-bearing potential;
- non-smokers with a clinical diagnosis of emphysema secondary to alpha-1-antitrypsin deficient emphysema;
- no oral corticosteroids for >60 days before study.
Exclusion criteria:
- concomitant Prolastin replacement therapy;
- treatment with oral corticosteroids or antibiotics for pulmonary symptoms >3 times in 12 months before study;
- current use of any CYP 450 3A4 inhibitors or inducers.
Gender: Males or Females
Age limits: Min: 30 Years Max: N/A (No limit)
Accepts healthy volunteers: No
Anticipated start date: October, 2003
Trial registration date: 07/26/2005
Date last updated: 12/2/2008
Link to trial result
This trial was conducted at the following locations:
Australia
Belgium
Canada
Denmark
Germany
Netherlands
New Zealand
Spain
Sweden
Switzerland
United Kingdom
- Birmingham
- Cambridge
- Edinburgh
