Effetti del modulatore CFTR su ossa e muscoli negli adulti con fibrosi cistica
Study is looking at the effects of cystic fibrosis treatment on bone muscle.
Panoramica dello studio
Stato
Intervento / Trattamento
Descrizione dettagliata
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.
Tipo di studio
Iscrizione (Effettivo)
Contatti e Sedi
Luoghi di studio
-
-
Indiana
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Indianapolis, Indiana, Stati Uniti, 46202-5149
- Indiana University
-
-
Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Metodo di campionamento
Popolazione di studio
Enrollment will target groups that are frequency matched for age (decade), race, sex, pancreatic insufficiency and glucocorticoid usage (inhaled or systemic as both have skeletal effects in some studies) to control for these factors influencing relevant bone and muscle outcomes. (see statistical description). The study will enroll up to 30 subjects in each group, expecting up to 25% dropout.
Descrizione
Inclusion Criteria:
-
documented, confirmed diagnosis of CF
- Age ≥18 years old
- >21 days since the start of their last pulmonary exacerbation at the baseline visit
- Provide signed written informed consent to participate
Exclusion Criteria:
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• Estimated glomerular filtration rate (eGFR) <30 ml/min/m2 using the CKD-EPI equation,
- Treatment with any osteoporosis medication within 6 months for oral agents or 1 year for intravenous or injectable agents (Subjects may participate if therapy stopped earlier than these time periods).
- Current treatment with growth hormone or IGF-1
- Currently pregnant or lactating or planning plan on becoming pregnant during the duration of the study.
- Life expectancy less than 12 months
- History of lung transplantation
- Conditions that in the opinion of the investigators would interfere with the ability to collect or interpret the data, or put the patient at higher safety risk from study procedures.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Modelli osservazionali: CASE_ONLY
- Prospettive temporali: PROSPECTIVE
Coorti e interventi
Gruppo / Coorte |
Intervento / Trattamento |
|---|---|
|
on CFTR
For patients on or near time of initiation CFTR modulator therapy |
CFTR modulators are drugs used to treat cystic fibrosis |
|
Controls
controls will be patients not eligible for available treatment |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Identify the effects of CFTR modulators on bone mineral density
Lasso di tempo: 12 months
|
Changes from baseline to 12 month in total volumetric BMD and in estimated failure load as measured by HRpQCT at the distal radius and tibia |
12 months
|
|
establish the effect of CFTR modulators on sarcopenia.
Lasso di tempo: 12 months
|
changes from baseline to 12 months in whole body lean mass using body composition software on DXA |
12 months
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Identify the effect of CFTR modulators on bone mineral density
Lasso di tempo: 6, 12 and 24 month
|
Changes from baseline to 24 month in total volumetric BMD and in estimated failure load as measured by HRpQCT at the distal radius and tibia. Changes from baseline to 6, 12, and 24 month in HRpQCT measure of cortical vBMD and trabecular vBMD at both the distal radius and tibia and cortical vBMD and trabecular vBMD at diaphyseal site. Changes from baseline to 6, 12 and 24 month in HRpQCT measure of cortical vBMC and trabecular vBMD at both the distal radius and tibia and cortical vBMD at a diaphyseal site Changes from baseline to 6,12, and 24 month in areal BMD using DXA at eh lumbar spine, total body, femur neck and total hip |
6, 12 and 24 month
|
|
establish the effect of CFTR modulators on sarcopenia.
Lasso di tempo: 6, 12 and 24 month
|
changes from baseline to 6 and 24 months in whole body lean mass using body composition software on DXA Changes from baseline to 6, 12, 24 months in appendicular lean mass relative to height measured using body composition software on DXA |
6, 12 and 24 month
|
Collaboratori e investigatori
Sponsor
Collaboratori
Investigatori
- Investigatore principale: Erik A Imel, MD, Indiana University
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Manifestazioni neurologiche
- Malattie ossee
- Malattie muscoloscheletriche
- Malattie del sistema nervoso
- Manifestazioni neuromuscolari
- Condizioni patologiche, anatomiche
- Malattie genetiche, congenite
- Malattie metaboliche
- Malattie delle vie respiratorie
- Malattie dell'apparato digerente
- Malattie polmonari
- Infante, neonato, malattie
- Malattie pancreatiche
- Atrofia
- Malattie e anomalie congenite, ereditarie e neonatali
- Condizioni patologiche, segni e sintomi
- Malattie nutrizionali e metaboliche
- Segni e sintomi
- Atrofia muscolare
- Fibrosi cistica
- Malattie ossee, metaboliche
Altri numeri di identificazione dello studio
- 1908566174
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