Clinical trials: End Stage Lung Disease
Cystic Fibrosis
Study of Ataluren (PTC124™) in Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
- Trial name
- NCT00803205
- Testing
- Ataluren
- Where
- 38 sites in United States, Belgium and 9 more
- Volunteers
- 238 people
- Sponsor
- PTC Therapeutics
Cystic Fibrosis
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein.
- Trial name
- NCT00458341
- Testing
- Ataluren
- Where
- 3 sites in Belgium, France
- Volunteers
- 30 people
- Sponsor
- PTC Therapeutics
Primary Immunodeficiency, PID
Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched CD3+/CD19+ depleted bone marrow transplant (BMT) is safe and effective for individuals…
- Trial name
- BOLT-BMT (NCT03330795)
- Testing
- CD3/CD19 neg allogeneic BMT
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)