Metachromatic leukodystrophy

5 trials MedDRA PT
5 Registered trials
0 Recruiting
2 Unique sponsors
2 Unique IMPs
5 Commercial trials
Drug classes (ATC)
— ATC code not specified 2
A Gastroenterology (ATC A) 3
Mechanism of action (INN-stem analysis)
Unknown / small molecule 2 substances
Phase III: 2 Phase II: 1 Phase I/II: 2

Filter these 5 trials by country, sponsor, date range and more.

Open in Search → Gap analysis →
Show: 5 trials

Phase III

2 trials
  • Orchard Therapeutics (Europe) Limited Commercial
    ATIDARSAGENE AUTOTEMCEL
    2024-511970-66-00 1 country 2017 n=10 Orphan
    Ended
  • Orchard Therapeutics (Europe) Limited Commercial
    ATIDARSAGENE AUTOTEMCEL
    2024-511971-13-00 1 country 2020 n=6 Orphan
    Ended

Phase II

1 trial
  • Shire Human Genetic Therapies Inc. Commercial
    CEBSULFASE ALFA
    2024-514402-31-00 6 countries 2019 n=10 Orphan
    Ended

Phase I/II

2 trials
  • Orchard Therapeutics (Europe) Limited Commercial
    ATIDARSAGENE AUTOTEMCEL
    2024-515253-25-00 1 country 2010 n=20 Orphan
    Ended
  • Shire Human Genetic Therapies Inc. Commercial
    CEBSULFASE ALFA
    2024-514403-34-00 5 countries 2012 n=10 Orphan
    Ended
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Orchard Therapeutics (Europe) Limited Commercial 3 Phase III, Phase I/II 0
Shire Human Genetic Therapies Inc. Commercial 2 Phase II, Phase I/II 0
New trials by year
2019
1
2020
1
← All indication landscapes