In Part 1 of the study, a fixed dose of ABBV-3067 (potentiator) will be co-administered with ABBV-2222 (corrector) in a dose range-finding manner to enable a dose selection for ABBV-2222 for Part 2 and future combination studies. In addition, ABBV-…
ID
Source
Brief title
Condition
- Respiratory disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Change from Baseline through Day 29 in overall lung function.
Secondary outcome
1) Change from Baseline through Day 29 in sweat chloride
2) Change from Baseline through Day 29 in other spirometric measures
3) Change from Baseline through Day 29 in lung function
Background summary
Cystic fibrosis (CF) is a genetic disease that affects many organs including
the lungs, in which sticky mucus and persistent infections cause increased
breathing difficulty over time. CF is caused by mutations in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene that affect the
production and function of the CFTR protein. When the CFTR protein is not
produced correctly, it affects the balance of salt and fluids inside and
outside of the cell. This imbalance leads to thick, sticky mucus in the lungs,
and loss of organ functions in the pancreas and other organs. ABBV-3067 is a
type of CFTR modulator called a potentiator. Potentiators help facilitate the
opening of the CFTR protein on the cell surface to allow chloride and sodium
(salt) to move in and out of the cell. ABBV-2222 is a CFTR corrector.
Correctors help the CFTR protein form the right shape so that it can move to
the cell surface.
Study objective
In Part 1 of the study, a fixed dose of ABBV-3067 (potentiator) will be
co-administered with ABBV-2222 (corrector) in a dose range-finding manner to
enable a dose selection for ABBV-2222 for Part 2 and future combination
studies. In addition, ABBV-3067 will be given alone (i.e. with placebo for
ABBV-2222) to evaluate the safety and efficacy of ABBV-3067.
In Part 2 of the study, the dose of ABBV-2222 selected from Part 1 will be
co-administered with ABBV-3067 in a dose-ranging manner to enable selection of
the ABBV-3067 dose for future combination studies.
Study design
Randomised, double-blind, placebo controlled parallel arm study
Intervention
ABBV-3067 and/or ABBV-2222 in different doses, placebo controlled.
Study burden and risks
Risks associated with participation are risks involving study procedures and
risks associated with the study drug.
The duration of participation can take up to three months, with a screening
period, a treatment period and a follow up period. The participant will need to
attend study visits and a phone call. The participant does not need to complete
diaries or questionnaires.
Knollstrasse 70
Ludwigshafen 67061
DE
Knollstrasse 70
Ludwigshafen 67061
DE
Listed location countries
Age
Inclusion criteria
*Adults 18 years or older
*Confirmed diagnosis of CF and homozygous for F508del CFTR mutation
*Lung function >= 40% and <= 90% of predicted normal for age, gender, and height
*Stable pulmonary status
Exclusion criteria
*Cirrhosis with portal hypertension
*History of solid organ or hematopoietic transplantation
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2019-000750-63-NL |
| ClinicalTrials.gov | NCT03969888 |
| CCMO | NL70226.041.19 |