Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients
NCT ID: NCT02532764
Last Updated: 2019-02-06
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
70 participants
INTERVENTIONAL
2015-06-30
2017-09-14
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SINGLE_GROUP
TREATMENT
TRIPLE
Study Groups
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QR-010
QR-010 administered via inhalation either as a single dose or three times weekly for four weeks.
QR-010
Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
Placebo
Placebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks.
Placebo
Normal Saline
Interventions
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QR-010
Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
Placebo
Normal Saline
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* Use of lumacaftor or ivacaftor
* Use of any investigational drug or device
* History of lung transplantation
* Hemoptysis
18 Years
60 Years
ALL
No
Sponsors
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European Commission
OTHER
ProQR Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Stuart Elborn, MD
Role: PRINCIPAL_INVESTIGATOR
Trust and Queen's University Belfast
Locations
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University of Southern California USC - Keck School of Medicine
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
Northwestern University
Chicago, Illinois, United States
University of Kansas Medical Center Research Institute
Kansas City, Kansas, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University School of Medicine
St Louis, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Washington Medical Center
Seattle, Washington, United States
Universitair Ziekenhuis Brussel
Brussels, , Belgium
University of Leuven
Leuven, , Belgium
University of Calgary (Health Sciences Centre)
Calgary, Alberta, Canada
Motol University Hospital
Prague, , Czechia
Cystic Fibrosis Center Rigshospitalet
Copenhagen, , Denmark
HGRL Chu Nantes
Nantes, , France
Hopital Necker- Enfants Malades
Paris, , France
Charité Universitätsmedizin Berlin
Berlin, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Munich U. Hospital, Cystic Fibrosis Center for Adults
Munich, , Germany
Azienda Ospedaliera Universitaria Integrata di Verona
Verona, , Italy
Hospital Vall D'Hebron
Barcelona, , Spain
Celerion
Belfast, Northern Ireland, United Kingdom
Royal Brompton Hospital
London, , United Kingdom
Southampton General Hospital
Southampton, , United Kingdom
Countries
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References
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Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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PQ-010-001
Identifier Type: -
Identifier Source: org_study_id
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