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

P

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

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

Trial documents
2

Trial contacts and locations

27

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Data sourced from clinicaltrials.gov

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