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

ProQR Therapeutics70 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Cystic Fibrosis

Interventions

QR-010DRUG

Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton

PlaceboDRUG

Normal Saline

Eligibility

Sex: ALLMin age: 18 YearsMax age: 60 Years

Medical Language ↔ Plain English

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

Locations (27)

Outcomes

Primary Outcomes

Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study

Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Severity of Treatment Emergent Adverse Events From Baseline Through End of Study

Assessment of severity of treatment emergent adverse events (TEAEs). Severity is graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Modified for CF (CTCAE v4.03). For events not present in this listing the following grading was applied: Mild: Asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Moderate: Minimal, local, or noninvasive intervention indicated; discomfort sufficient to reduce or interfere with daily activities; Severe: Medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization may be indicated; disabling; limits self-care with significant interference with daily activities; incapacitating with inability to perform self care activities of daily living; Life-threatening: Urgent intervention indicated; immediate risk of death.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.

DLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Secondary Outcomes

Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.

Number of subjects experiencing at least one abnormality for the categories laboratory parameters, vital signs, ECG, spirometry and physical findings that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely.

Data from ClinicalTrials.gov

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