symptomatic emphysema secondary to alpha-1-antitrypsin deficiency
Conditions
Interventions
Product Name: Not applicable
Product Code: RO3300074
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: None assigned
Other descriptive name: Retinoic Acid Receptor gamma Agonist
Concentration un
Sponsors
F. Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Men >= 30 years of age with AATD of the Pi Z or Pi Null phenotype. 2. Women >= 30 years of age with AATD of the Pi Z or Pi Null phenotype beyond child-bearing potential defined as women who are more than 5 years post menopausal unless they have had a hysterectomy or bilateral oophorectomy. 3. Non-smokers with clinical diagnosis of emphysema with confirmation by radiographic imaging (ex-smokers must have stopped smoking for ? 6 months prior to enrollment). 4. TLco (KCO ) =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. AATD patients on concomitant Prolastin replacement therapy. 2. Psychiatric disorders requiring medication or hospitalization, or with suicide attempt within the last 7 years. 3. More than 3 exacerbations of pulmonary symptoms, requiring treatment with oral steroids, or antibiotics, within 12 months prior to this study. 4. Patients with giant bullous disease. 5. Significant other medical conditions, which in the opinion of the investigator, will interfere with the patient’s ability to perform the study tests. 6. Hypertriglyceridemia ? 300mg/dl with therapy 7. Concomitant medications which are inhibitors or inducers of CYP 450 3A4 activity
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: Secondary objectives include assessment of lung function including spirometry, TLco, lung volumes, arterial blood gases, exercise, quality of life, assessments of dyspnea, and plasma biomarkers.;Main Objective: This is a phase II proof of concept study to investigate efficacy, safety and tolerability of 1.0 mg of RO3300074 when administered once daily for 12 months (52 weeks) compared with placebo. The primary efficacy measure is the change from baseline in 12 months of the 15th percentile of the lung density adjusted for the lung volume as obtained by computer tomography (spiral CT scanner) using lung density histograms;Primary end point(s): The primary efficacy measure is the change from baseline in 12 months of the 15th percentile of the lung density adjusted for the lung volume as obtained by computer tomography (spiral CT scanner) using lung density histograms. The CT densitometry measurements will be performed at baseline, after 28 and after 52 weeks of treatment. | — |
Countries
Denmark, Spain, United Kingdom
Outcome results
None listed