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Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

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ClinicalTrials.gov Identifier: NCT02532764
Recruitment Status : Active, not recruiting
First Posted : August 26, 2015
Last Update Posted : August 18, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: QR-010 Drug: Placebo Phase 1 Phase 2

Detailed Description:
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.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : June 2015
Estimated Primary Completion Date : September 2017
Estimated Study Completion Date : September 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: QR-010
QR-010 administered via inhalation either as a single dose or three times weekly for four weeks.
Drug: QR-010
Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
Placebo Comparator: Placebo
Placebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks.
Drug: Placebo
Normal Saline


Outcome Measures

Primary Outcome Measures :
  1. Incidence of adverse events from baseline through End of Study Visit [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Safety and Tolerability

  2. Severity of adverse events from baseline through End of Study [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Safety and Tolerability

  3. Occurrence of dose-limiting toxicities (DLT) in each dose cohort from baseline through End of Study Visit. [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Safety and Tolerability


Secondary Outcome Measures :
  1. Changes from baseline to end-of-treatment or presence of abnormalities regarding laboratory parameters, vital signs, ECG, spirometry, and physical findings. [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Safety

  2. Changes from baseline to end-of-treatment or presence of abnormalities regarding laboratory parameters using descriptive statistics, shift tables, and frequencies and percentage of subjects with treatment emergent abnormalities for each timepoint. [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Safety

  3. Changes from baseline to end-of-treatment or presence of abnormalities in vital signs will be summarized descriptively. [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Safety

  4. Changes from baseline to end-of-treatment or presence of abnormalities regarding ECG, spirometry, and physical findings will be summarized and listed as specified in the Statistical Analysis Plan. [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Safety

  5. Maximum Serum Concentration (Cmax) [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Pharmacokinetics

  6. Time to Maximum Serum Concentration (Tmax) [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Pharmacokinetics

  7. Terminal Half-life (T1/2) [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Pharmacokinetics

  8. Area Under the Curve to final sample [AUC(0-last)] [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Pharmacokinetics

  9. Area Under the Curve to infinity [AUC(0-∞)] [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Pharmacokinetics

  10. Serum Clearance (CL) [ Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts ]
    Pharmacokinetics


Other Outcome Measures:
  1. Change in FEV1 [ Time Frame: 8 weeks for Multiple-dose cohorts ]
    Exploratory Efficacy

  2. Change in patient reported outcome measure CFQ-R Respiratory Symptom Score (CFQ-R RSS) [ Time Frame: 8 weeks for Multiple-dose cohorts ]
    Exploratory Efficacy

  3. Change in body weight [ Time Frame: 8 weeks for Multiple-dose cohorts ]
    Exploratory Efficacy

  4. Change in Sweat chloride [ Time Frame: 8 weeks for Multiple-dose cohorts ]
    Exploratory Efficacy


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Breast‐feeding or pregnant
  • Use of lumacaftor or ivacaftor
  • Use of any investigational drug or device
  • History of lung transplantation
  • Hemoptysis
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02532764


  Show 27 Study Locations
Sponsors and Collaborators
ProQR Therapeutics
European Commission
Investigators
Principal Investigator: Stuart Elborn, MD Trust and Queen's University Belfast
More Information

Responsible Party: ProQR Therapeutics
ClinicalTrials.gov Identifier: NCT02532764     History of Changes
Other Study ID Numbers: PQ-010-001
First Posted: August 26, 2015    Key Record Dates
Last Update Posted: August 18, 2017
Last Verified: November 2016

Keywords provided by ProQR Therapeutics:
cystic fibrosis
ΔF508
RNA therapies
antisense oligonucleotide
CFTR
F508del
CF
RNA therapy

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases