Interviews and Video Capture in Patients With GM1 Gangliosidosis
- Conditions
- GM1 Gangliosidosis
- First Posted Date
- 2020-03-17
- Last Posted Date
- 2023-06-08
- Lead Sponsor
- LYSOGENE
- Target Recruit Count
- 25
- Registration Number
- NCT04310163
- Locations
- 🇺🇸
Casimir Trials, Plymouth, Massachusetts, United States
A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis
- Conditions
- GM1 Gangliosidosis
- Interventions
- Genetic: LYS-GM101
- First Posted Date
- 2020-02-18
- Last Posted Date
- 2023-06-09
- Lead Sponsor
- LYSOGENE
- Target Recruit Count
- 5
- Registration Number
- NCT04273269
- Locations
- 🇺🇸
Children's Hospital of Orange County (CHOC), Orange, California, United States
🇫🇷Hôpital Armand-Trousseau, Centre de Référence des Maladies Lysosomales (CRML), Service de Neuropédiatrie, Paris, France
🇬🇧Manchester University NHS Foundation Trust, Manchester, United Kingdom
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
- First Posted Date
- 2018-08-02
- Last Posted Date
- 2021-08-31
- Lead Sponsor
- LYSOGENE
- Target Recruit Count
- 20
- Registration Number
- NCT03612869
- Locations
- 🇺🇸
Weill Cornell Medical College, New York, New York, United States
🇳🇱Amsterdam UMC, Amsterdam, Netherlands
🇺🇸University of Minnesota, Minneapolis, Minnesota, United States
Natural History Study of Patients With MPS IIIA
- Conditions
- Mucopolysaccharidosis IIIA
- First Posted Date
- 2016-04-21
- Last Posted Date
- 2021-08-31
- Lead Sponsor
- LYSOGENE
- Target Recruit Count
- 23
- Registration Number
- NCT02746341
- Locations
- 🇳🇱
Academic Medical Center, Emma Children's Hospital, Amsterdam, Netherlands
🇬🇧Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom
🇧🇷Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
- Conditions
- Mucopolysaccharidosis Type III ASanfilippo Disease Type A
- Interventions
- Genetic: SAF-301
- First Posted Date
- 2014-02-03
- Last Posted Date
- 2017-06-20
- Lead Sponsor
- LYSOGENE
- Target Recruit Count
- 4
- Registration Number
- NCT02053064
- Locations
- 🇫🇷
Hôpitaux Universitaires Paris Sud (Bicêtre), Le Kremlin-Bicêtre, France
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
- Conditions
- Sanfilippo Disease Type AMucopolysaccharidosis Type III A
- Interventions
- Genetic: SAF-301
- First Posted Date
- 2011-11-18
- Last Posted Date
- 2014-05-06
- Lead Sponsor
- LYSOGENE
- Target Recruit Count
- 4
- Registration Number
- NCT01474343
- Locations
- 🇫🇷
Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris, Le Kremlin Bicêtre, France
🇫🇷Hôpital Necker, Assistance Publique des Hôpitaux de Paris, Paris, France