Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis

Novartis

Status and phase: Completed; Phase 2; Phase 1

Conditions: Pulmonary Cystic Fibrosis

Treatments: Drug: QBW276; Other: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02566044

CQBW276X2201

2014-004915-35 (EudraCT Number)

Details and patient eligibility

About

This is a study of multiple doses of inhaled QBW276 in patients with cystic fibrosis on top of standard of care. The study was divided into 3 Cohorts. Cohorts 1 and 2 are designed to be a randomized, double-blind, placebo-controlled, parallel arm, multiple dose study to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of inhaled QBW276 over 1 week (cohort 1) or 2 weeks (cohort 2) in patients with cystic fibrosis regardless of their genotype.

The study was terminated after Cohort 2 due to the resource issues.

Enrollment: 16 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Cohorts 1 and 2 = any genotype on any standard of care treatment
• Cohort 3 = F508del homozygotes on standard of care at that time
• FEV₁between 40 and 100%
• LCI2.5 ≥ 8 if FEV₁is more than 80%

Exclusion criteria

• Adrenal or electrolyte abnormalities
• Lung transplant
• Autonomic dysfunction (e.g. recurrent episodes of fainting, palpitations, etc.)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Crossover Assignment

Masking: Double Blind

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16 participants in 2 patient groups, including a placebo group

CQBW276

Experimental group

Description:

Cohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3.

Treatment:

Drug: QBW276

Placebo

Placebo Comparator group

Description:

Cohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3.

Treatment:

Other: Placebo