Completed
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
What is being tested
QR-010
+ Placebo
Drug
Who is being recruted
Cystic Fibrosis+5
+ Digestive System Diseases
+ Infant, Newborn, Diseases
From 18 to 60 Years
+12 Eligibility Criteria
How is the trial designed
Treatment Study
Placebo-ControlledPhase 1 & 2
Interventional
Study Start: June 2015
Summary
Principal SponsorProQR Therapeutics
Last updated: January 27, 2026
Sourced from a government-validated database.Claim as a partner
Study start date: June 1, 2015
Actual date on which the first participant was enrolled.The purpose of this study is to evaluate the safety, tolerability, and to determine the pharmacokinetics of QR-010 administered via inhalation in adult homozygous for ΔF508 Cystic Fibrosis.
Official TitlePhase 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
Principal SponsorProQR Therapeutics
Last updated: January 27, 2026
Sourced from a government-validated database.Claim as a partner
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.Design Details
70 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Conditions
Criteria
Any sex
Biological sex of participants that are eligible to enroll.From 18 to 60 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Cystic FibrosisDigestive System DiseasesInfant, Newborn, DiseasesLung DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPancreatic DiseasesRespiratory Tract DiseasesGenetic Diseases, Inborn
Criteria
7 inclusion criteria required to participate
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
Show More Criteria
5 exclusion criteria prevent from participating
Breast-feeding or pregnant
Use of lumacaftor or ivacaftor
Use of any investigational drug or device
History of lung transplantation
Show More Criteria
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.Treatment Groups
Study Objectives
2 intervention groups are designated in this study
50% chance of being blinded to the placebo group
Treatment Groups
Group I
ExperimentalQR-010 administered via inhalation either as a single dose or three times weekly for four weeks.
Group II
PlaceboPlacebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks.
Study Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 27 locations
Suspended
University of Southern California USC - Keck School of Medicine
Los Angeles, United StatesOpen University of Southern California USC - Keck School of Medicine in Google MapsSuspended
Stanford University
Palo Alto, United StatesSuspended
Northwestern University
Chicago, United StatesSuspended
University of Kansas Medical Center Research Institute
Kansas City, United StatesCompleted27 Study Centers