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

NCT04623879 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 13

Last updated 2021-02-03

No results posted yet for this study

Summary

Background: Lumacaftor/Ivacaftor (LUM-IVA), a CFTR corrector-potentiator combination, was found to improve lung function and reduce pulmonary exacerbations (PEx). However, cystic fibrosis (CF) is a multi-organ disease and therefore there is a need for more information on the systemic effects of CFTR modulators.

Aim: To evaluate pancreatic function, bone metabolism and respiratory changes through a year of LUM-IVA treatment.

Methods: A prospective real world, one-year study on F508del homozygous adult CF patients who commenced treatment with LUM-IVA. Visits were scheduled on the first day of treatment and every 3 months evaluating: symptoms, Body Mass Index (BMI), spirometry, laboratory tests and Quality of life. At baseline and at 12 months, the patients underwent sweat test, oral glucose tolerance test (OGTT), chest CT and dual-energy X-ray absorptiometry (DEXA).

Conditions

Interventions

DRUG

Lumacaftor, Ivacaftor Drug Combination

Sponsors & Collaborators

  • Carmel Medical Center

    lead OTHER

Principal Investigators

  • Karin Yaacoby-Bianu, MD · Carmel Medical Center

Eligibility

Min Age
18 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-12-29
Primary Completion
2019-07-30
Completion
2020-02-26
FDA Drug
Yes

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04623879 on ClinicalTrials.gov