Renal Impact of KAFTRIO in Mucoviscidose Population (IRT-MUCO)

The objective of this study is to describe the renal impact of Elexacaftor-Teacaftor-Ivacaftor, a triple modulator therapy of CFTR channel, in patients with cystic fibrosis. This new treatment acts on the CFTR channel, which is expressed at the level of the nephrons. The objective is to study the changes in plasma and urinary parameters, including metabolic explorations of urolithiasis, change in volemic parameters, renal function, urinary sediment and nutritional and glycemic parameters, in newly treated patients, through the data collected at introduction of the treatment and during the follow-up.

Study Overview

• Status: Recruiting
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Biological: Evaluation of the parameters of the lithiasis assessment before and after KAFTRIO initiation; Drug: Collection of Arterial pressure, metabolic data, kidney function, proteinuria and hematuria before and after KATRIO initiation

• Study Type: Observational
• Enrollment (Estimated): 20

Contacts and Locations

• Study Contact: Name: Etienne NOVEL CATIN; Phone Number: +33 04.72.67.87.37; Email: etienne.novel-catin@chu-lyon.fr

Study Locations

• France
  • Pierre-Bénite, France, 69310
    • Recruiting
    • Centre Hospitalier Lyon Sud
    • Contact: Etienne NOVEL-CATIN, MD; Phone Number: 04.72.67.87.37; Email: etienne.novel-catin@chu-lyon.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Probability Sample

Study Population

Patient with cystic fibrosis followed at Centre Hospitalier Lyon Sud (in the reference and competence center for cystic fibrosis)

Description

Inclusion Criteria:

• Patient with cystic fibrosis
• More than 18 years old
• Eligible for KAFTRIO

Exclusion Criteria:

• KAFTRIO contraindication
• Patient less than 18 years old
• Dialysis patient
• Pregnant woman
• Greffed patient
• Patient opposition to data collection

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Patient with cystic fibrosis, eligible for KAFTRIO therapy; Patient with cystic fibrosis, eligible for KATRIO, over 18 years old.

Biological: Evaluation of the parameters of the lithiasis assessment before and after KAFTRIO initiation; Collection of Uricuria, phosphaturie, calciuria, magnesuria, glycosuria, citraturia, oxaluria, before and after initiation of KAFTRIO; Drug: Collection of Arterial pressure, metabolic data, kidney function, proteinuria and hematuria before and after KATRIO initiation; Arterial pressure, glycemia, glycated hemoglobin, lipid profile, weight, serum and urinary electrolytes, hepatic check, complete blood count, calcium phosphate balance and urinary sediment analysis before and after initiation of KAFTRIO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Modification of the parameters of the lithiasis assessment	Change in blood phosphorus and calcium metabolism ; Change in calciuria mmol/d ;Change in urinary pH, urinary specific gravity (g/mL) and crystalluria	1 month after initiation of KAFTRIO
Modification of the parameters of the lithiasis assessment	Change in blood phosphorus and calcium metabolism ; Change in calciuria mmol/d ;Change in urinary pH, urinary specific gravity (g/mL) and crystalluria	7 months after initiation of KAFTRIO

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in volemic parameter	body mass index (BMI) (kg/m²), systolic and diastolic blood pressure (mmHg), presence of orthostatic hypotension, renin and aldosterone levels and ratio (ng/L and pmol/L respectively), natremia (mmol/L), natriuresis (mmol/d), kalemia (mmol/L), kaliuresis (mmol/d), chloremia (mmol/L), bicarbonatemia (mmol/L), brain natriuretic peptid (ng/L), hemoglobin (g/L) and sodium intake estimated by the dietician and calculated thanks to natriuresis (g/d)	1 month after initiation of KAFTRIO
Change in volemic parameter	body mass index (BMI) (kg/m²), systolic and diastolic blood pressure (mmHg), presence of orthostatic hypotension, renin and aldosterone levels and ratio (ng/L and pmol/L respectively), natremia (mmol/L), natriuresis (mmol/d), kalemia (mmol/L), kaliuresis (mmol/d), chloremia (mmol/L), bicarbonatemia (mmol/L), brain natriuretic peptid (ng/L), hemoglobin (g/L) and sodium intake estimated by the dietician and calculated thanks to natriuresis (g/d)	7 month after initiation of KAFTRIO
Change in renal function	(measured using creatinine (µmol/L) and cystatin C (mg/L)combined to report 2021 CKD-EPIcreatinineand CKD-EPIcreatinine-cystatin C formulas) and in plasma urea levels (mmol/L).	1 month after initiation of KAFTRIO
Change in renal function	(measured using creatinine (µmol/L) and cystatin C (mg/L)combined to report 2021 CKD-EPIcreatinineand CKD-EPIcreatinine-cystatin C formulas) and in plasma urea levels (mmol/L).	7 month after initiation of KAFTRIO
Change in urinary sediment	, represented by proteinuria (g/day), albuminuria/creatininuria ratio (mg/g) and urinary beta-2-microglobulin level (mg/day), after collecting 24-hours urine sa	1 month after initiation of KAFTRIO
Change in urinary sediment	, represented by proteinuria (g/day), albuminuria/creatininuria ratio (mg/g) and urinary beta-2-microglobulin level (mg/day), after collecting 24-hours urine sa	7 month after initiation of KAFTRIO
Change in glycemic profile	(fasting blood glucose level (mmol/L) and glycated hemoglobin (%)) and nutritional profile (albumin and pre-albumin in g/L).	1 month after initiation of KAFTRIO
Change in glycemic profile	(fasting blood glucose level (mmol/L) and glycated hemoglobin (%)) and nutritional profile (albumin and pre-albumin in g/L).	7 month after initiation of KAFTRIO
Change in lung function	predicted forced expiratory volume in 1 second (FEV1) (%) and peak expiratory flow (%)).	1 month after initiation of KAFTRIO
Change in lung function	predicted forced expiratory volume in 1 second (FEV1) (%) and peak expiratory flow (%)).	7 month after initiation of KAFTRIO

Collaborators and Investigators

• Sponsor: Hospices Civils de Lyon
• Investigators: Principal Investigator: Etienne NOVEL-CATIN, MD, Hospices civils de Lyon

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2022
• Primary Completion (Estimated): December 31, 2023
• Study Completion (Estimated): December 31, 2023

Study Registration Dates

• First Submitted: December 11, 2023
• First Submitted That Met QC Criteria: January 8, 2024
• First Posted (Actual): January 9, 2024

Study Record Updates

• Last Update Posted (Actual): January 9, 2024
• Last Update Submitted That Met QC Criteria: January 8, 2024
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: cystic fibrosis; lithiasis; KAFTRIO; renal impact
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: 22-5080; 69HCL23_1169 (Other Identifier: Easydore)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No