Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients
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
1 other identifier
interventional
70
10 countries
27
Brief Summary
A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Jun 2015
Typical duration for phase_1
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2015
CompletedFirst Submitted
Initial submission to the registry
August 13, 2015
CompletedFirst Posted
Study publicly available on registry
August 26, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 14, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
September 14, 2017
CompletedResults Posted
Study results publicly available
February 6, 2019
CompletedFebruary 6, 2019
January 1, 2019
2.3 years
August 13, 2015
December 11, 2018
January 15, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study
Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Assessment of severity of treatment emergent adverse events (TEAEs). Severity is graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Modified for CF (CTCAE v4.03). For events not present in this listing the following grading was applied: Mild: Asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Moderate: Minimal, local, or noninvasive intervention indicated; discomfort sufficient to reduce or interfere with daily activities; Severe: Medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization may be indicated; disabling; limits self-care with significant interference with daily activities; incapacitating with inability to perform self care activities of daily living; Life-threatening: Urgent intervention indicated; immediate risk of death.
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.
DLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Secondary Outcomes (7)
Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Maximum Serum Concentration
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Time to Maximum Serum Concentration
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Terminal Half-life (T1/2)
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Area Under the Curve to Final Sample [AUC(0-last)]
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
- +2 more secondary outcomes
Other Outcomes (8)
Adjusted Mean Change From Baseline in CFQ-R RSS
Day 15, Day 33, Day 54
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo
Day 15, Day 33, Day 54
Adjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)
Day 15, Day 33, Day 54
- +5 more other outcomes
Study Arms (2)
QR-010
EXPERIMENTALQR-010 administered via inhalation either as a single dose or three times weekly for four weeks.
Placebo
PLACEBO COMPARATORPlacebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- Breast-feeding or pregnant
- Use of lumacaftor or ivacaftor
- Use of any investigational drug or device
- History of lung transplantation
- Hemoptysis
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- ProQR Therapeuticslead
- European Commissioncollaborator
Study Sites (27)
University of Southern California USC - Keck School of Medicine
Los Angeles, California, 90033, United States
Stanford University
Palo Alto, California, 940304, United States
Northwestern University
Chicago, Illinois, 60611, United States
University of Kansas Medical Center Research Institute
Kansas City, Kansas, 66160, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Washington University School of Medicine
St Louis, Missouri, 63110-1032, United States
Nationwide Children's Hospital
Columbus, Ohio, 66160, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
University of Washington Medical Center
Seattle, Washington, 98195-6522, United States
Universitair Ziekenhuis Brussel
Brussels, 01090, Belgium
University of Leuven
Leuven, 03000, Belgium
University of Calgary (Health Sciences Centre)
Calgary, Alberta, T2N 4N1, Canada
Motol University Hospital
Prague, 15006, Czechia
Cystic Fibrosis Center Rigshospitalet
Copenhagen, 02100, Denmark
HGRL Chu Nantes
Nantes, 44300, France
Hopital Necker- Enfants Malades
Paris, 75743, France
Charité Universitätsmedizin Berlin
Berlin, 13353, Germany
Medizinische Hochschule Hannover
Hanover, 30625, Germany
Munich U. Hospital, Cystic Fibrosis Center for Adults
Munich, 80336, Germany
Azienda Ospedaliera Universitaria Integrata di Verona
Verona, 37134, Italy
Hospital Vall D'Hebron
Barcelona, 08035, Spain
Celerion
Belfast, Northern Ireland, BT9 6AD, United Kingdom
Royal Brompton Hospital
London, SW3 6NP, United Kingdom
Southampton General Hospital
Southampton, SO16 6YD, United Kingdom
Related Publications (1)
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.
PMID: 31215818DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- ProQR Therapeutics
Study Officials
- PRINCIPAL INVESTIGATOR
Stuart Elborn, MD
Trust and Queen's University Belfast
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 13, 2015
First Posted
August 26, 2015
Study Start
June 1, 2015
Primary Completion
September 14, 2017
Study Completion
September 14, 2017
Last Updated
February 6, 2019
Results First Posted
February 6, 2019
Record last verified: 2019-01