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

ProQR Therapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Cystic Fibrosis

Treatments: Drug: QR-010; Drug: Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02532764

PQ-010-001

Details and patient eligibility

About

A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.

Full 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.

Enrollment: 70 patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: Triple Blind

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70 participants in 2 patient groups, including a placebo group

QR-010

Experimental group

Description:

QR-010 administered via inhalation either as a single dose or three times weekly for four weeks.

Treatment:

Drug: QR-010

Placebo

Placebo Comparator group

Description:

Placebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks.

Treatment:

Drug: Placebo