Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40.
ID
Source
Brief title
Condition
- Respiratory disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in
1 Second (ppFEV1).
Secondary outcome
1. Absolute change from Baseline in Sweat Chloride (SwCl).
2. Absolute change from Baseline in forced vital capacity [FVC].
3. Absolute change from Baseline in forced expiratory flow at mid-lung capacity
[FEF25-75].
4. Relative changes from Baseline in Percent Predicted Forced Expiratory Volume
in 1 Second (ppFEV1).
5. Relative changes from Baseline in forced vital capacity [FVC].
6. Relative changes from Baseline in Forced Expiratory Flow Between 25% and 75%
of Exhaled Volume (FEF25-75).
7. Absolute change in CF Questionnaire-Revised (CFQ-R) respiratory domain score
from Baseline.
Background summary
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects
the lungs and digestive system, significantly impairing the quality of life,
with those affected having a median age of death at 40.
Study objective
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects
the lungs and digestive system, significantly impairing the quality of life,
with those affected having a median age of death at 40.
Study design
Randomized, Double-blind, Parallel cohort study
Intervention
Participants in arm 1 will receive oral capsules of galicaftor/navocaftor dual
combination for 28 days followed by galicaftor/navocaftor/ABBV-119 triple
combination for 28 days. All other participants will receive the
galicaftor/navocaftor/ABBV-119 triple combination or placebo for 28 days. For
all study arms, galicaftor, navocaftor, will be given once daily and ABBV-119
twice a day.
Study burden and risks
There may be higher treatment burden for participants inthis trial compared to
their standard of care. Participants will attend regular visits during the
study at a hospital or clinic. The effect of the treatment will be checked by
medical assessments, blood tests, checking for side effects and completing
questionnaires.
Knollstrasse 50
Ludwigshafen 67061
DE
Knollstrasse 50
Ludwigshafen 67061
DE
Listed location countries
Age
Inclusion criteria
1. Confirmed clinical diagnosis of CF, and genotype homozygous for the F508del
CFTR mutation for Cohort 1 and Cohort 3, heterozygous for F508del CFTR mutation
and a minimal function mutation for Cohort 2 and Cohort 3.
2. ppFEV1 >= 40% and <= 90% of predicted normal for age, gender, and height
(Global Lung Function Initiative [GLI] equations) at Screening.
3. No clinically significant laboratory values at Screening that would pose
undue risk for the subject or interfere with safety assessments (per the
investigator).
4. Absence of clinically significant abnormality detected on ECG regarding
rate, rhythm, or conduction (e.g., QT interval corrected for heart rate using
Fridericia's formula [QTcF] should be < 450 msec for males and < 460 msec
for females).
5. Stable pulmonary status, i.e., no respiratory infections or exacerbations
requiring a change in therapy (including antimicrobials) or causing an acute
decline in ppFEV1 of >10% from usual ppFEV1 level within 4 weeks.
6. SwCl at screening visit must be >= 60 mmol/L for Cohort 1 and Cohort 2, and
this criteria does not apply to Cohort 3.
7. No history of diseases aggravated or triggered by ultraviolet radiation and
no history of abnormal reaction photosensitivity or photoallergy to sunlight,
or artificial source of intense light, especially ultraviolet light.
Exclusion criteria
1. Cirrhosis with or without portal hypertension (e.g., splenomegaly,
esophageal varices) or history of clinically significant liver disease.
3. History of malignancy within past 5 years (except for excised basal cell
carcinoma of the skin with no recurrence, or treated carcinoma in situ of the
cervix with no recurrence).
4. Recent (within the past 6 months) history of drug or alcohol abuse that
might preclude adherence to the protocol, in the opinion of the investigator.
5. Smoking or vaping tobacco or cannabis products within 6 months before
Screening.
6. History of solid organ or hematopoietic transplantation.
7. History of known sensitivity to any component of the study drug.
8. Need for supplemental oxygen while awake, or >2 L/minute while sleeping.
10. Evidence of active SARS-CoV-2 infection. If a subject has signs/symptoms
suggestive of SARS CoV-2 infection, they should undergo molecular (e.g.,
polymerase chain reaction [PCR]) testing to rule out SARS-CoV-2 infection.
Subjects who do not meet SARS-CoV-2 infection eligibility criteria must be
screen failed and may only rescreen after they meet the SARS-CoV-2 infection
viral clearance criteria listed in the protocol.
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 | EUCTR2020-005805-25-NL |
| ClinicalTrials.gov | NCT04853368 |
| CCMO | NL78519.056.21 |