Miglustat

5 trials 3 recruiting AGastroenterology Orphan designation

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5 Registered trials

3 Recruiting

2 Completed trials

3 Sponsors

11 Countries

Phase III

Phase IV

Phase III: 4 Phase IV: 1

Indications tested

Niemann-Pick disease 1

Sponsors using this IMP

Amicus Therapeutics Inc. 3 Cyclo Therapeutics Inc. 1 Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC) 1

All trials

5 total

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of cipaglucosidase alfa/miglustat in both E… Recruiting

2022-501095-25-01 Phase III 5 countries Amicus Therapeutics Inc.
Study to evaluate the safety and efficacy of Cipaglucosidase Alfa and Miglustat in pediatric subjects with late-onset Pompe disease Recruiting

2022-502547-36-00 Phase III 2 countries Amicus Therapeutics Inc.
Treatment frequency Reduction In POmpe disease (TRIPO-Study) Recruiting

2024-514255-15-01 Phase IV 1 country Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Standard of Care Compared to Placebo and Stan… Ended

2024-518266-27-00 Phase III 4 countries Cyclo Therapeutics Inc.
A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects … Ended

2023-505170-15-00 Phase III 9 countries Amicus Therapeutics Inc.

Showing 1–5 of 5 trials

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