CFTR Modulator Effects on Bone & Muscle in Adults With Cystic Fibrosis

Status Active, not recruiting

Clinical Trial Summary

Study is looking at the effects of cystic fibrosis treatment on bone muscle.

Clinical Trial Description

Cystic fibrosis (CF) is a complex multisystem genetic disease, with pulmonary and gastrointestinal consequences dominating the clinical picture. The life-expectancy of CF patients has increased through several therapeutic advances. Although respiratory failure remains the major cause of mortality in CF, musculoskeletal impairments contribute to major morbidity. In the general population, musculoskeletal conditions are among the most common reasons for seeking medical care, and the risk of osteoporotic fracture increases with age. As the CF population ages, the morbidity related to musculoskeletal effects may increase. The etiology of CF related bone disease is multifactorial and includes effects of pancreatic insufficiency, poor nutritional status, vitamin D deficiency, glucocorticoid treatment, inflammation, hypogonadism, and sarcopenia, collectively resulting in attenuated bone mineral accrual and low bone density The effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulating drugs on bone disease in CF has not been evaluated. Effects of CFTR modulators may help counter the bone and muscle consequences of CF either directly by effects on bone or muscle cells, or indirectly by improved lung disease, improved nutritional status, decreased systemic inflammation or glucocorticoid use, or subsequent increases in physical activity. The rationale that underlies the proposed research is that better understanding of the bone and muscle effects of CFTR modulator therapies will help guide strategies to optimize bone accrual, prevent osteoporosis and fractures, and improve functional outcomes in the aging CF population. Set on the backbone of a longitudinal observational cohort study, the study will systematically and comprehensively evaluate changes in bone and muscle mass and strength from baseline to 12 month and 24 month time points among patients receiving CFTR modulator therapies and also among controls not receiving CFTR modulator therapies. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04206436
Study type	Observational
Source	Indiana University
Contact
Status	Active, not recruiting
Phase
Start date	December 18, 2019
Completion date	July 13, 2024

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04696198` - Thoracic Mobility in Cystic Fibrosis Care	N/A
Completed	`NCT00803205` - Study of Ataluren (PTC124™) in Cystic Fibrosis	Phase 3
Terminated	`NCT04921332` - Bright Light Therapy for Depression Symptoms in Adults With Cystic Fibrosis (CF) and COPD	N/A
Completed	`NCT03601637` - Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del	Phase 3
Terminated	`NCT02769637` - Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)
Recruiting	`NCT06030206` - Lung Transplant READY CF 2: A Multi-site RCT	N/A
Recruiting	`NCT06012084` - The Development and Evaluation of iCF-PWR for Healthy Siblings of Individuals With Cystic Fibrosis	N/A
Recruiting	`NCT06032273` - Lung Transplant READY CF 2: CARING CF Ancillary RCT	N/A
Recruiting	`NCT06088485` - The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
Recruiting	`NCT05392855` - Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic Fibrosis (SPACE-CF)	N/A
Recruiting	`NCT04039087` - Sildenafil Exercise: Role of PDE5 Inhibition	Phase 2/Phase 3
Recruiting	`NCT04056702` - Impact of Triple Combination CFTR Therapy on Sinus Disease.
Completed	`NCT04038710` - Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
Completed	`NCT04058548` - Clinical Utility of the 1-minute Sit to Stand Test as a Measure of Submaximal Exercise Tolerance in Patients With Cystic Fibrosis During Acute Pulmonary Exacerbation	N/A
Completed	`NCT03637504` - Feasibility of a Mobile Medication Plan Application in CF Patient Care	N/A
Recruiting	`NCT03506061` - Trikafta in Cystic Fibrosis Patients	Phase 2
Completed	`NCT03566550` - Gut Imaging for Function & Transit in Cystic Fibrosis Study 1
Recruiting	`NCT04828382` - Prospective Study of Pregnancy in Women With Cystic Fibrosis
Completed	`NCT04568980` - Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis
Recruiting	`NCT04010253` - Impact of Bronchial Drainage Technique by the Medical Device Simeox® on Respiratory Function and Symptoms in Adult Patients With Cystic Fibrosis	N/A

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms