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NCT05109793: PRONTO
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
trial in GM1 Gangliosidosis in 31 participants. Completed in 16 September 2025.
16 September 2025
Quick facts
| Lead sponsor | Azafaros A.G. |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 31 |
| Start date | 22 February 2022 |
| Primary completion | 16 September 2025 |
| Estimated completion | 16 September 2025 |
| Sites | 13 locations across France, Italy, United Kingdom, Germany, United States, Brazil |
Conditions studied
- GM1 Gangliosidosis — all drugs for GM1 Gangliosidosis →
- Sandhoff Disease — all drugs for Sandhoff Disease →
- Tay-Sachs Disease — all drugs for Tay-Sachs Disease →
Sponsor
Azafaros A.G. — full company profile →
Who can join
Adults 2 to 20, any sex, with GM1 Gangliosidosis or Sandhoff Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).
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 NCT05109793
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for GM1 Gangliosidosis
Currently open trials in the same condition.
- NCT07054515 — A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem · Phase 3 · recruiting
- NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · recruiting
Other Azafaros A.G. trials
Trials by the same sponsor.
- NCT07399704 — A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pi · Phase 2 · recruiting
- NCT07054515 — A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem · Phase 3 · recruiting
- NCT07082543 — A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 · Phase 3 · recruiting
- NCT07082725 — A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem · Phase 3 · 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 NCT05109793 (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 Azafaros A.G.
- Last refreshed: 29 September 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/NCT05109793.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing