Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ΔF508 Cystic Fibrosis
Status: Completed
Location: See all (26) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 60
Healthy Volunteers: f
View:
• Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of \> 60 mmol/L
• Confirmation of CFTR gene mutations homozygous for the ΔF508 mutation
• Body mass index (BMI) ≥ 17 kg/m2
• Non-smoking for a minimum of two years
• FEV1 ≥70% of predicted normal for age, gender, and height, at Screening
• Stable lung function
• Adequate hepatic and renal function
Locations
United States
California
University of Southern California USC - Keck School of Medicine
Los Angeles
Stanford University
Palo Alto
Illinois
Northwestern University
Chicago
Kansas
University of Kansas Medical Center Research Institute
Kansas City
Massachusetts
Boston Children's Hospital
Boston
Massachusetts General Hospital
Boston
Missouri
Washington University School of Medicine
Saint Louis
Ohio
Nationwide Children's Hospital
Columbus
Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey
South Carolina
Medical University of South Carolina
Charleston
Texas
University of Texas Southwestern Medical Center
Dallas
Washington
University of Washington Medical Center
Seattle
Other Locations
Belgium
Universitair Ziekenhuis Brussel
Brussels
University of Leuven
Leuven
Canada
University of Calgary (Health Sciences Centre)
Calgary
Denmark
Cystic Fibrosis Center Rigshospitalet
Copenhagen
France
HGRL Chu Nantes
Nantes
Hopital Necker- Enfants Malades
Paris
Germany
Charité Universitätsmedizin Berlin
Berlin
Medizinische Hochschule Hannover
Hannover
Munich U. Hospital, Cystic Fibrosis Center for Adults
Munich
Italy
Azienda Ospedaliera Universitaria Integrata di Verona
Verona
Spain
Hospital Vall D'Hebron
Barcelona
United Kingdom
Celerion
Belfast
Royal Brompton Hospital
London
Southampton General Hospital
Southampton
Time Frame
Start Date: 2015-06
Completion Date: 2017-09-14
Participants
Target number of participants: 70
Treatments
Experimental: QR-010
QR-010 administered via inhalation either as a single dose or three times weekly for four weeks.
Placebo_comparator: Placebo
Placebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks.
Authors
Christiane De Boeck, Alicia Casey, Karen McCoy, Isabel Neuringer, Manu Jain, Richard Moss, Raksha Jain, Patrick Flume, Adupa Rao, Moira Aitken, Daniel Rosenbluth, Susanne Naehrig, Nico Derichs, Isabelle Danner-Boucher, Tobias Welte, Robert Vender, Joel Mermis, Anne Malfroot, Stuart Elborn, Javier De Gracia, Isabelle Sermet, Mary Carroll, Tacjana Pressler, Michael Parkins, Jane Davies, Pavel Drevinek, Marco Cipolli
Related Therapeutic Areas
Sponsors
Leads: ProQR Therapeutics
Collaborators: European Commission