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NCT05545774: Cystinose
Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
trial in Cystinosis in 20 participants. Completed in 11 September 2025.
11 September 2025
Quick facts
| Lead sponsor | Assistance Publique - Hôpitaux de Paris |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 20 |
| Start date | 22 September 2022 |
| Primary completion | 11 September 2025 |
| Estimated completion | 11 September 2025 |
| Sites | 2 locations across France |
Conditions studied
- Cystinosis — all drugs for Cystinosis →
Sponsor
Assistance Publique - Hôpitaux de Paris — full company profile →
Who can join
16 and older, any sex, with Cystinosis. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The primary objective of the study is to evaluate the change in motor function of patients with cystinosis. The secondary objectives of the study: * assessment of the respiratory function; * assessment of the muscle function; * assessment of swallowing disorders; * assessment radiologically of the muscular efficiency; * assessment of lean mass / fat mass ratio; * assessment of sleeping disorders; * annual assessment of evolution of above functions. All patients will be examined by experienced neuromuscular specialist (Pr Pascal Laforêt) and pulmonologist specialized in neuromuscular disorders (Pr Hélène Prigent). All evaluations will be performed in Raymond-Poincaré hospital (Teaching hospital of Assistance Publique - Hopitaux de Paris (APHP) and University of Paris-Saclay) neuromuscular center, coordinated by Pr Pascal Laforêt.
Publications & conference data
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
Verify or expand the search:
- PubMed search for NCT05545774
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Cystinosis
Currently open trials in the same condition.
- NCT05959668 — Development of Health-related Quality of Life Instrument for Patients With Cystinosis · recruiting
- NCT05843851 — Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria · NA · recruiting
- NCT06065852 — National Registry of Rare Kidney Diseases · recruiting
- NCT00359684 — Use of Cysteamine in the Treatment of Cystinosis · recruiting
Other Assistance Publique - Hôpitaux de Paris trials
Trials by the same sponsor.
- NCT07443436 — Immunomodulatory Treatment of Interstitial Lung Disease Associated With Surfactant Related Gene Variants · Phase 2 · not yet recruiting
- NCT07499492 — Red Blood Cell Transfusion to Optimize Extubation · NA · not yet recruiting
- NCT07379918 — Real-life Evaluation of Endopredict® in Early HR+/HER2- Breast Cancer · recruiting
- NCT07473869 — Smartphone Application for Automated Measurement of Capillary Refill Time (CRT) · not yet recruiting
- NCT07505394 — Efficacy of a Prediction Model-based Algorithm to PREVENT Drug-induced Impulse Control Disorders in Parkinson's Disease · NA · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05545774 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Assistance Publique - Hôpitaux de Paris
- Last refreshed: 24 November 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05545774.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing