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Trial record 2 of 3 for:    ProQR

Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

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ClinicalTrials.gov Identifier: NCT02564354
Recruitment Status : Completed
First Posted : September 30, 2015
Last Update Posted : November 10, 2016
Sponsor:
Collaborator:
European Commission
Information provided by (Responsible Party):
ProQR Therapeutics

Brief Summary:
Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).

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

Detailed Description:
This is an open-label, multi-center, exploratory study to estimate the effect of intranasal administration of QR-010 on the nasal mucosa in the restoration of CFTR function, as measured by nasal potential difference (NPD), in the nasal epithelium of adult subjects with CF who are homozygous or compound heterozygous for the ΔF508 CFTR mutation.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Open-Label, Exploratory Study to Evaluate the Effects of QR-010 on Nasal Potential Difference in Subjects With CF With the ΔF508 CFTR Mutation
Study Start Date : September 2015
Actual Primary Completion Date : September 2016
Actual Study Completion Date : September 2016

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: ΔF508 Homozygous
QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks.
Drug: QR-010
Single-stranded RNA antisense oligonucleotide in isoosmolar solution
Experimental: ΔF508 Compound Heterozygous
QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks.
Drug: QR-010
Single-stranded RNA antisense oligonucleotide in isoosmolar solution



Primary Outcome Measures :
  1. Intra-subject change of CFTR-mediated total chloride transport as measured by nasal potential difference (NPD) from baseline through End of Study [ Time Frame: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment ]
    Exploratory Efficacy


Secondary Outcome Measures :
  1. Number of subjects with a -6.6 mV or more negative change in CFTR-mediated total chloride transport, and after different treatment durations from baseline through End of Study [ Time Frame: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment ]
    Key Exploratory Efficacy

  2. Compare CFTR-mediated total chloride transport between dF508 homozygous and compound heterozygous CF subjects from baseline through End of Study [ Time Frame: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment ]
    Exploratory Efficacy

  3. Incidence of (serious) adverse events from baseline through End of Study [ Time Frame: 3 weeks post-treatment ]
    Safety and tolerability

  4. Incidence of discontinuations due to AEs from baseline through End of Study [ Time Frame: 3 weeks post-treatment ]
    Safety and tolerability

  5. Abnormalities of laboratory parameters (chemistry, hematology and urinalysis) from baseline through End of Study [ Time Frame: 3 weeks post-treatment ]
    Safety and tolerability

  6. Abnormalities of vital signs & oximetry from baseline through End of Study [ Time Frame: 3 weeks post-treatment ]
    Safety and tolerability

  7. Abnormalities of physical examinations from baseline through End of Study [ Time Frame: 3 weeks post-treatment ]
    Safety and tolerability

  8. Changes in nasal symptoms (based on the NERS and SNOT-22) from baseline through End of Study [ Time Frame: 3 weeks post-treatment ]
    Safety and tolerability


Other Outcome Measures:
  1. Serum concentration of QR-010 after intranasal administration [ Time Frame: 30 minutes post dose Baseline, 2 & 4 weeks ]
    Pharmacokinetics

  2. Serum concentration of QR-010 after intranasal administration if persistent AE(s) are present [ Time Frame: 3 weeks post-treatment ]
    Pharmacokinetics

  3. Changes in systemic effect of QR-010 as measured by pilocarpine iontopheresis sweat chloride [ Time Frame: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment ]
    Pharmacokinetics

  4. Explore correlation between change in nasal potential difference (NPD), sweat chloride (mmol/L), and serum concentration of QR-010 [ Time Frame: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment ]
    Exploratory Efficacy



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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
  • Nasal potential difference (NPD) measurement at Screening consistent with CF
  • Confirmation of CFTR gene mutations homozygous or compound heterozygous for the ΔF508 mutation
  • Body mass index (BMI) of ≥ 18 kg/m2
  • Non-smoking for a minimum of 2 years
  • Stable lung function
  • FEV1 ≥40% of predicted normal for age, gender, and height at Screening

Exclusion Criteria:

  • Breast-feeding or pregnant
  • Acute allergy or infection affecting nasal conditions not resolved within 14 days prior Screening
  • Use of lumacaftor or ivacaftor
  • Use of any investigational drug or device
  • Hemoptysis

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): NCT02564354


Locations
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, Colorado
National Jewish Health
Denver, Colorado, United States, 80206
United States, Ohio
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Belgium
U.Z. Leuven
Leuven, Belgium, 3000
France
Hopital Necker-Enfants Malades
Paris, France, 75743
Sponsors and Collaborators
ProQR Therapeutics
European Commission
Investigators
Principal Investigator: John P Clancy, MD Cincinnati Childrens Hospital Medical Center

Responsible Party: ProQR Therapeutics
ClinicalTrials.gov Identifier: NCT02564354     History of Changes
Other Study ID Numbers: PQ-010-002
First Posted: September 30, 2015    Key Record Dates
Last Update Posted: November 10, 2016
Last Verified: November 2016

Keywords provided by ProQR Therapeutics:
Cystic Fibrosis
CF
ΔF508
CFTR
QR-010
antisense oligonucleotide
RNA therapy
F508del
NPD
nasal potential difference

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