Cystinosis and Mitochondrial Metabolism study
Official title: Evaluation of Mitochondrial Metabolism in Patients With Cystinosis: CYSTI-MITO Project
Run by Hospices Civils de Lyon · NCT07319091 · Data updated 2026-11-12
This study tests a treatment approach targeting mitochondrial metabolism in people with nephropathic cystinosis to see if it improves kidney-related outcomes.
Quick facts
| Study type | Interventional |
|---|---|
| Ages | 2 Years and up |
| Sex | All |
| Enrollment | 25 (estimated) |
| Sponsor | Hospices Civils de Lyon Academic / other |
| Locations | 9 sites (Bron and 8 other locations) |
| Status | Recruiting |
| Started | December 8, 2025 |
| Main results expected | December 2027 |
| Study ends | July 2028 |
| Registry record updated | August 3, 2026 |
| Trial ID | NCT07319091 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are patients with genetically confirmed nephropathic cystinosis, aged 2 years or older, on conservative native kidney care and taking cysteamine.
✕Probably not a fit
Patients unlikely to benefit include those with recent transplant or dialysis, those not adhering to procedures, or those not affiliated with social security where required.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This interventional study focuses on individuals with nephropathic cystinosis to examine the effects of modulating mitochondrial metabolism. Eligible participants are patients with genetically confirmed cystinosis who are on conservative native kidney treatment and taking oral cysteamine, aged 2 years and older. The trial assesses how altering mitochondrial metabolism might influence disease-related complications, including renal and systemic manifestations. Enrollment requires informed consent and adherence to study procedures, with sites in Lyon and Lille, France. The study does not report a phase designation.
Why it matters
Potential benefit: If successful, participants may experience improved kidney function or broader metabolic benefits related to cystinosis.
How similar studies have gone: Previous cystinosis treatments have shown benefits with cysteamine, but direct mitochondrial-targeted approaches in this condition are less established and exploratory.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patient with genetically confirmed nephropathic cystinosis * Men and women, children and adults with cystinosis * Undergoing conservative treatment on native kidneys * Age ≥ 2 years * Patients receiving oral cysteamine * Patients with social security coverage * Informed consent signed by the participant or parents or legal guardians before participating in the study Exclusion Criteria: * Patient not complying with study procedures * Transplant or dialysis patient * Patient on anticalcineurin * Pregnant or breast-feeding woman * Person deprived of liberty by a judicial or administrative decision * Person not affiliated to a social security scheme or beneficiaries of a similar scheme
Where it's running
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Service de néphrologie pédiatrique, Hôpital Femme Mère Enfant, Hospices Civils de Lyon
Bron, France Recruiting -
Service de Néphrologie pédiatrique, Hôpital Jeanne de Flandre
Lille, France Recruiting -
Service de néphrologie et exploration fonctionnelle rénale, Hôpital Edouard Herriot, Hospices Civils de Lyon
Lyon, France Recruiting -
Service de Néphrologie pédiatrique, Hôpital de la Timone
Marseille, France Recruiting -
Service de Néphologie et endocrinologie pédiatrique, Hôpital Arnaud de Villeneuve
Montpellier, France Recruiting -
Service de Néphrologie pédiatrique, Hôpital Necker-Enfants Malades
Paris, France Recruiting
Show 3 more locations
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Service de Néphrologie-transplantation rénale adultes, Hôpital Necker-Enfants Malades
Paris, France Recruiting -
Service de Néphrologie pédiatrique, Hôpital Robert Debré
Paris, France Recruiting -
Service de Néphrologie-Dialyse-Transplantation pédiatrique, Hôpital d'enfants Brabois
Vandœuvre-lès-Nancy, France Recruiting
Study contacts
- Study coordinator: Justine BACCHETTA, MD
- Email: justine.bacchetta@chu-lyon.fr
- Phone: 4 27 85 61 30
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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National Registry for Rare Kidney Diseases
This study is collecting health information from people with rare kidney diseases to help improve treatments and create better care guidelines.
-
Cohort study on cystinosis in Europe
This study looks at how cystinosis affects the lives of patients in Europe and how different treatments impact their health and well-being.
-
Transplanting stem cells and kidneys from the same donor to prevent rejection
This study is testing if giving patients stem cells and a kidney from the same donor can help prevent kidney rejection without needing lifelong medication to suppress their immune system.
-
Registry for patients with rare diseases
This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.
-
Using cysteamine to treat cystinosis
This study is testing how well the medication cysteamine works for people with cystinosis and what long-term effects it has on their health.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.