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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-09-30

Study Completion Date

2016-09-30

Brief Summary

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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).

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Detailed Description

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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.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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ΔF508 Homozygous

QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks.

Group Type EXPERIMENTAL

QR-010

Intervention Type DRUG

Single-stranded RNA antisense oligonucleotide in isoosmolar solution

ΔF508 Compound Heterozygous

QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks.

Group Type EXPERIMENTAL

QR-010

Intervention Type DRUG

Single-stranded RNA antisense oligonucleotide in isoosmolar solution

Interventions

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QR-010

Single-stranded RNA antisense oligonucleotide in isoosmolar solution

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No