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NCT06985212: FASMD
National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
trial in Acid Sphingomyelinase Deficiency (ASMD) in 200 participants. Not yet recruiting.
15 April 2035
Quick facts
| Lead sponsor | Wladimir MAUHIN, Dr |
|---|---|
| Status | Not yet recruiting |
| Study type | OBSERVATIONAL |
| Enrollment | 200 |
| Start date | 15 May 2025 |
| Primary completion | 15 April 2035 |
| Estimated completion | 15 April 2035 |
| Sites | 1 location across France |
Conditions studied
- Acid Sphingomyelinase Deficiency (ASMD) — all drugs for Acid Sphingomyelinase Deficiency (ASMD) →
- Niemann Pick Disease — all drugs for Niemann Pick Disease →
Sponsor
Wladimir MAUHIN, Dr
Who can join
2 and older, any sex, with Acid Sphingomyelinase Deficiency (ASMD) or Niemann Pick Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The goal of this study is to describe the natural history of ASMD in adult and paediatric patients with or without specific treatment in order to assess the impact of the disease on their daily lives and quality of life. The population concerned corresponds to patients aged at least 2 years, with a definite diagnosis of ASMD as determined by a confirmed low acid sphingomyelinase assay and who have not expressed their opposition to participating in this research (patients and/or parental authority).
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT06985212
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Acid Sphingomyelinase Deficiency (ASMD)
Currently open trials in the same condition.
- NCT07274826 — Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD) · active not recruiting
Other Wladimir MAUHIN, Dr trials
Trials by the same sponsor.
- NCT07277361 — Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment · 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 NCT06985212 (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 Wladimir MAUHIN, Dr
- Last refreshed: 22 May 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/NCT06985212.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing