Cystic Fibrosis Clinical Trial
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
Verified date | September 2020 |
Source | ProQR Therapeutics |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Completed |
Enrollment | 18 |
Est. completion date | September 2016 |
Est. primary completion date | September 2016 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
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 ?F508 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 |
Country | Name | City | State |
---|---|---|---|
Belgium | U.Z. Leuven | Leuven | |
France | Hopital Necker-Enfants Malades | Paris | |
United States | University of Alabama at Birmingham | Birmingham | Alabama |
United States | Cincinnati Childrens Hospital Medical Center | Cincinnati | Ohio |
United States | National Jewish Health | Denver | Colorado |
Lead Sponsor | Collaborator |
---|---|
ProQR Therapeutics | European Commission |
United States, Belgium, France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD). | The primary endpoint was the within-subject change from baseline in total chloride transport as measured by NPD, after the Chloride-free+isoproterenol solution (Cl-free+iso), and was based on the average measurements of both nostrils. To provide baseline stability, baseline was defined as the average of the two most recent pre-dose values, where each pre-dose value was the average of two nostrils. A negative change from baseline of Cl-free+iso shows an improvement. | Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment. | |
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. | This analysis is the proportion (amount) of subjects with an average Cl-free+iso actual value of -6.6 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The actual value of -6.6 mV is used to discriminate individuals with CF (>-6.6 mV) from normal individuals (= -6.6 mV). | 2 and 4 weeks, and at 3 weeks post-treatment. | |
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. | This analysis is the proportion of subjects with an average Cl-free+iso actual value of -4 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The value of -4 mV is considered a clinically relevant response to treatment based on data from studies of other CFTR therapies. | 2 and 4 weeks, and at 3 weeks post-treatment. | |
Secondary | Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study. | This endpoint was the within-subject change in Sodium transport (average Potential Difference prior to perfusion of any solution); this is the average of Potential Difference measured at five sites in the inferior meatus of the nose. A positive change from baseline shows an improvement. | Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment. | |
Secondary | The Mean Change in CFTR-mediated Total Chloride Transport. | The mean change in CFTR-mediated Total Chloride Transport compared to Baseline, per cohort; this is the mean of Potential Difference measured at five sites in the inferior meatus of the nose measured in millivolts (mV). A negative change from baseline shows an improvement. | 2 and 4 weeks, and at 3 weeks post-treatment. | |
Secondary | Number of Subjects Experiencing Serious Adverse Events From Baseline Through End of Study. | Number of subjects experiencing serious adverse events from baseline through End of Study. | 3 weeks post-treatment. | |
Secondary | Number of Subject Discontinuations Due to AEs From Baseline Through End of Study. | Number of subject discontinuations due to AEs from baseline through End of Study. No discontinuations occurred. | 3 weeks post-treatment. | |
Secondary | Number of Subjects With Abnormalities of Laboratory Parameters From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality in laboratory parameters (chemistry, hematology and urinalysis) that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | 3 weeks post-treatment. | |
Secondary | Number of Subjects With Abnormalities of Vital Signs & Oximetry From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality vital signs & oximetry that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | 3 weeks post-treatment. | |
Secondary | Number of Subjects With Abnormalities of Physical Examinations From Baseline Through End of Study. | Number of subjects experiencing at least one abnormality in physical examination that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study. | 3 weeks post-treatment. | |
Secondary | Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study. | The NERS was performed by site staff prior to each dose and as part of the NPD procedure; it is a set of scales ranging from 0 (no symptoms) to 3 (most severe symptoms) for assessing the severity of each of the following nasal symptoms, separately for each nostril: mucosal disruption, edema, erythema, polyp, secretions. The range of the total sum is 0-30. | 3 weeks post-treatment. | |
Secondary | Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study. | Subjects were asked to score a list of 22 symptoms on the SNOT-22 regarding social and emotional consequences. Outcomes were graded as 0 (no problem), to 5 (problem as bad as it could be). The list included: need to blow nose, sneezing, dripping nose, cough, postnasal drip, dense nasal drip, ear fullness, dizziness, ear pain, facial pain/pressure, difficulty falling asleep, waking at night, lack of a good night's sleep, waking up tired, fatigue, reduced productivity, reduced concentration, frustrated/restless/irritable, sad, embarrassed, decrease in smell and taste, and nasal obstruction. The range of the total sum is 0-110, with higher values indicating worse outcome. | 3 weeks post-treatment. |
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