Gaucher's disease type I

2 trials MedDRA PT
2 Registered trials
0 Recruiting
2 Unique sponsors
5 Unique IMPs
1 Commercial trials
Drug classes (ATC)
— ATC code not specified 1
H Endocrinology (ATC H) 1
L Oncology (ATC L) 1
Mechanism of action (INN-stem analysis)
Unknown / small molecule 2 substances
Corticosteroid 2 substances
mTOR inhibitor 1 substance
Phase II: 1 Phase I/II: 1

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Show: 2 trials

Phase II

1 trial
  • Genzyme Corp. Commercial
    VENGLUSTAT
    2023-508646-18-00 1 country 2017 n=3 Orphan
    Ended

Phase I/II

1 trial
  • Prevail Therapeutics Inc. Academic
    ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 9 EXPRESSING CODON-OPTIMIZED HUMAN GBA GENE; METHYLPREDNISOLONE; PREDNISONE; SIROLIMUS
    2022-500281-10-02 2 countries 2026 n=6 Orphan
    Halted
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Genzyme Corp. Commercial 1 Phase II 0
Prevail Therapeutics Inc. Academic 1 Phase I/II 0
New trials by year
2026
1
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