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

NCT ID: NCT02566044

Last Updated: 2020-12-30

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-09-27

Study Completion Date

2018-04-24

Brief Summary

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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.

Detailed Description

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Conditions

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Pulmonary Cystic Fibrosis

Keywords

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Cystic Fibrosis

Study Design

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Allocation Method

NA

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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CQBW276

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.

Group Type EXPERIMENTAL

QBW276

Intervention Type DRUG

0.3 mg and 1.5 mg strengths

Placebo

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.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo

Interventions

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Placebo

Placebo

Intervention Type OTHER

QBW276

0.3 mg and 1.5 mg strengths

Intervention Type DRUG

Eligibility Criteria

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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.)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

New York, New York, United States

Site Status

Novartis Investigative Site

Dallas, Texas, United States

Site Status

Novartis Investigative Site

Cologne, , Germany

Site Status

Novartis Investigative Site

Essen, , Germany

Site Status

Countries

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United Kingdom United States Germany

Provided Documents

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Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Related Links

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https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=280

A Plain Language Trial Summary is available on novartisclinicatrials.com

Other Identifiers

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2014-004915-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CQBW276X2201

Identifier Type: -

Identifier Source: org_study_id