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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05927467
Other study ID # C15-82
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date May 9, 2017
Est. completion date June 30, 2025

Study information

Verified date June 2023
Source Institut National de la Santé Et de la Recherche Médicale, France
Contact Laurence Heidet, PHD
Phone 0033 1 44 49 43 82
Email laurence.heidet@aphp.fr
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Alport syndrome is a rare, inherited condition characterized by a combination of glomerular nephropathy progressing to kidney failure, deafness, and eye involvement. This disease is associated with mutations in the genes encoding one of the three IV collagen chains expressed in the glomerular basement membrane. Significant progress has been made in understanding the molecular mechanisms responsible for the disease, but relatively little in understanding the progression of renal failure and in the area of therapeutics. We have shown in a retrospective European study that blockers of the renin angiotensin system may slow disease progression, but no controlled studies have been performed. Finally, innovative therapies (anti-micro-RNA, stem cells) have recently shown their effectiveness in animal models of the disease, and industrials are planning to quickly carry out phase 1 trials to test molecules. Carrying out therapeutic trials in humans will require full knowledge of the natural history of the disease (isolated hematuria, microalbuminuria, macroalbuminuria, renal failure and its progression) and gathering a sufficient number of patients, especially in the early stages. These trials and the indications for treatments would be greatly facilitated by the discovery of biomarkers that make it possible to predict the progression to renal failure earlier than the onset of proteinuria. The study aims to: - Establish a European database on Alport syndrome to assess the natural history of the disease. - To investigate the impact of the disease on the educational and professional life of patients and their families, and on the adherence and tolerance to renin-angiotensin system blockers prescribed to proteinuric patients. - Investigate access to molecular diagnostics and genetic counseling, as well as identify biomarkers that can predict progression of kidney disease. This project will be carried out at a French level with the support and participation of the very active renal rare disease sector, in collaboration with various countries wishing to participate.


Recruitment information / eligibility

Status Recruiting
Enrollment 700
Est. completion date June 30, 2025
Est. primary completion date June 30, 2025
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: - Diagnosis of AS based on electron microscopic examination of the renal biopsy and/or molecular studies and/or abnormal expression of type IV collagen chains on skin and/or glomerular basement membranes. - Signed informed consent Exclusion Criteria: - No exclusion criteria

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
France RaDiCo Eurbio-Alport Paris Île-de-France

Sponsors (1)

Lead Sponsor Collaborator
Institut National de la Santé Et de la Recherche Médicale, France

Country where clinical trial is conducted

France, 

Outcome

Type Measure Description Time frame Safety issue
Other Disease stage Stratification of patients according to their disease stage; patients' distribution analysis among countries Throughout the follow-up
Other Urinal concentration of specific molecules Correlation assessment between the urinal concentration of the five molecules recently described by Terzi's lab as predicting progression of CKD (or other putative biomarkers) with the rate of decline of the GFR (according of the estimated GFR) on a 3 year- period Through study completion, at 1 year, 2 year, 3 year
Primary Renal function: eGFR, age at ESRD, requirement of Renal Replacement Therapy (RRT) and type of RRT Through study completion, at 1 year, 2 year, 3 year
Primary Urine bio-analysis results: Presence or not and quantification of hematuria, microalbuminuria and proteinuria Through study completion, at 1 year, 2 year, 3 year
Primary Presence or not of hypertension Through study completion, at 1 year, 2 year, 3 year
Primary Level of Hearing loss Through study completion, at 1 year, 2 year, 3 year
Primary Ocular symptoms (presence or not of lenticonus, cataract, retina and cornea impairment) Through study completion, at 1 year, 2 year, 3 year
Secondary Adverse events for the long-term safety of RAAS blockers treatment Through study completion, at 1 year, 2 year, 3 year
Secondary Quality of life questionnaires Impact of disease on quality of life will be evaluated through scores of quality of life questionnaires SF36 for Adult et SF10 for paediatric patients Through study completion, at 1 year, 2 year, 3 year
Secondary Compliance Compliance will be evaluated using X. Girerd Compliance Questionnaire Throughout the follow-up
See also
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Recruiting NCT04947813 - Genotype-Phenotype Correlations in Patients With Alport Syndrome