Phase 1
N=18
Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT02564354 ↗Enrolled (actual)
18
Serious AEs
0.0%
Results posted
Sep 2020
Primary outcome: Primary: Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD). — 1.86; -0.75; -1.11; 1.66 mV
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- QR-010 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- ProQR Therapeutics
- Primary completion
- Sep 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD). |
1.86; -0.75; -1.11; 1.66; -1.92; 1.43 | — |
| SECONDARY 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. |
1; 0; 1; 0; 2; 0 | — |
| SECONDARY Number of Subjects With a -4 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study. |
3; 1; 2; 1; 4; 0 | — |
| SECONDARY Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study. |
-31.92; -27.02; -33.80; -27.48; -25.14; -25.52 | — |
| SECONDARY The Mean Change in CFTR-mediated Total Chloride Transport. |
-2.44; 1.88; -3.46; 1.65; -3.19; 2.49 | — |
| SECONDARY Number of Subjects Experiencing Serious Adverse Events From Baseline Through End of Study. |
0; 0 | — |
| SECONDARY Number of Subject Discontinuations Due to AEs From Baseline Through End of Study. |
0; 0 | — |
| SECONDARY Number of Subjects With Abnormalities of Laboratory Parameters From Baseline Through End of Study. |
0; 0 | — |
| SECONDARY Number of Subjects With Abnormalities of Vital Signs & Oximetry From Baseline Through End of Study. |
0; 0 | — |
| SECONDARY Number of Subjects With Abnormalities of Physical Examinations From Baseline Through End of Study. |
3; 4 | — |
| SECONDARY Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study. |
12.20; 11.50; 11.60; 13.38; 11.50; 12.25 | — |
| SECONDARY Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study. |
14.90; 19.13; 13.60; 15.38; 14.50; 14.13 | — |
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).
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT02564354). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.