Real Life Evaluation of the Multi-organ Effects of Lumacaftor/Ivacaftor on F508del Homozygous Cystic Fibrosis Patients.

• Register: ClinicalTrials.gov
• Last refreshed on: 16 February 2021
• Main ID: NCT04623879
• Date of registration: 12/10/2020
• Prospective Registration: No
• Primary sponsor: Carmel Medical Center
• Public title: Real Life Evaluation of the Multi-organ Effects of Lumacaftor/Ivacaftor on F508del Homozygous Cystic Fibrosis Patients.
• Scientific title: Real Life Evaluation of the Multi-organ Effects of Lumacaftor/Ivacaftor on F508del Homozygous Cystic Fibrosis Patients.
• Date of first enrolment: December 29, 2016
• Target sample size: 13
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04623879
• Study type: Observational

Contacts

• Name: Karin Yaacoby-Bianu, MD
• Affiliation: Carmel Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subjects who meet all of the following inclusion criteria will be eligible for this
study:

1. Willing and able to comply with scheduled visits, treatment plan, study
restrictions, laboratory tests, and other study procedures.

2. Subjects (males and females), will be aged 18 years or older

3. Confirmed diagnosis of CF

4. Homozygous for the F508del-CFTR mutation.

5. Willing to remain on a stable CF medication regimen through 12 months.

Exclusion Criteria:

- Subjects who meet any of the following exclusion criteria will not be eligible for
this study:

1. History of any comorbidity that, in the opinion of the investigator, might
confound the results of the study or pose an additional risk in administering
study drug to the subject.

For example:

• History of cirrhosis with portal hypertension, and/or history of risk factors
for Torsade de Pointes (e.g., familial long QT syndrome, hypokalemia, heart
failure, left ventricular hypertrophy, bradycardia, myocardial infarction,
cardiomyopathy, history of arrhythmia [ventricular and atrial fibrillation]),
obesity, acute neurologic events.

2. Any of the following abnormal laboratory values at Screening:

- Abnormal liver function defined as any 2 or more of the following: =3 ×
upper limit of normal (ULN) aspartate aminotransferase (AST), =3 × ULN
alanine aminotransferase (ALT), =3 × ULN gamma-glutamyl transpeptidase
(GGT), =3 × ULN alkaline phosphatase (ALP), or =2 × ULN total bilirubin

- Abnormal liver function defined as any increase of =5 × ULN AST or ALT

- Abnormal renal function defined as glomerular filtration rate =50
mL/min/1.73 m2

3. An acute upper or lower respiratory infection, pulmonary exacerbation, or changes
in therapy (including antibiotics) for pulmonary disease within 28 days before
Day 1 (first dose of study drug).

4. History of solid organ or hematological transplantation.

5. History or evidence of cataract, lens opacity, Y-suture, or lamellar rings
determined to be clinically significant by the ophthalmologist during the
ophthalmologic examination during the Screening Period.

6. History of alcohol or drug abuse in the past year, including but not limited to
cannabis, cocaine, and opiates, as deemed by the investigator.

7. Pregnant or nursing females: Females of childbearing potential must have a
negative pregnancy test at Screening and Day 1.



Age minimum: 18 Years
Age maximum: 99 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Lumacaftor, Ivacaftor Drug Combination

Primary Outcome(s)

Pulmonary function tests [Time Frame: 1 year]

Secondary Outcome(s)

Additional Pulmonary function tests [Time Frame: 1 year]

bone health factors [Time Frame: 1 year]

BMI [Time Frame: 1 year]

bone parameters [Time Frame: 1 year]

Glucose metabolism [Time Frame: The change between 12 months (under treatment) to screening]

Secondary ID(s)

CMC-16-0108-CTIL

Source(s) of Monetary Support

Please refer to primary and secondary sponsors