Miglustat

DRUG16 trials

Sponsors

Actelion, The Hospital for Sick Children, Cliniques universitaires Saint-Luc- Université Catholique de Louvain, University of Florida, National Taiwan University Hospital

Conditions

Cystic FibrosisGM1 GangliosidosesGM2 GangliosidosesGM2 GangliosidosisGangliosidoses GM2Gaucher DiseaseGaucher Disease Type 1Glycogen Storage Disease Type II Infantile Onset

Phase 2

Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

CompletedNCT00418847

The Hospital for Sick ChildrenGangliosidoses GM2

Start: 2004-07-31End: 2009-04-30Updated: 2016-05-19

Miglustat in Niemann-Pick Type C Disease

CompletedNCT00517153

ActelionNiemann-Pick Type C Disease

Start: 2002-01-31End: 2008-01-31Updated: 2025-02-04

Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

TerminatedNCT00537602

ActelionCystic Fibrosis

Start: 2007-11-30End: 2008-03-31Updated: 2010-02-12

Miglustat in Cystic Fibrosis

CompletedNCT00742092

ActelionCystic Fibrosis

Start: 2008-08-31End: 2008-12-31Updated: 2014-03-07

Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?

CompletedNCT00945347

Cliniques universitaires Saint-Luc- Université Catholique de LouvainCystic Fibrosis

Start: 2009-07-31End: 2011-06-30Target: 10Updated: 2011-08-10

Phase 3

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

CompletedNCT00319046

ActelionGaucher Disease Type 1

Start: 2006-02-01End: 2010-07-01Updated: 2025-02-04

Application of Miglustat in Patients With Niemann-Pick Type C

CompletedNCT01760564

National Taiwan University HospitalNiemann-Pick Disease Type C

Start: 2008-01-31End: 2010-12-31Updated: 2013-01-04

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

CompletedNCT03729362

Amicus TherapeuticsPompe Disease (Late-onset)

Start: 2018-12-04End: 2021-01-15Updated: 2025-09-11

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

TerminatedNCT03822013

Tehran University of Medical SciencesGM2 Gangliosidosis, Supportive Care

Start: 2019-01-14End: 2025-09-10Updated: 2025-09-17

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Active, not recruitingNCT03911505

Amicus TherapeuticsPompe Disease (Late-onset)

Start: 2020-02-13End: 2026-06-30Updated: 2025-10-27

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

RecruitingNCT04808505

Amicus TherapeuticsGlycogen Storage Disease Type II Infantile Onset

Start: 2023-07-18End: 2027-07-01Target: 36Updated: 2026-01-22

Phase 4

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

TerminatedNCT02030015

University of MinnesotaGM1 Gangliosidoses, GM2 Gangliosidoses, Sandhoff Disease +1

Start: 2015-12-22End: 2019-07-31Updated: 2021-04-14

Safety and Efficacy of Miglustat in Chinese NPC Patients

CompletedNCT03910621

ActelionNiemann-Pick Disease, Type C

Start: 2020-04-02End: 2022-03-25Updated: 2025-03-30

Unknown Phase

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

CompletedNCT01451879

University of FloridaPompe Disease

Start: 2008-10-31End: 2017-10-31Updated: 2017-12-08

Miglustat on Gaucher Disease Type IIIB

National Taiwan University HospitalGaucher Disease

Start: 2015-07-31End: 2019-12-31Updated: 2019-03-29

A Global Prospective Observational Registry of Patients With Pompe Disease

RecruitingNCT06121011

Amicus TherapeuticsPompe Disease

Start: 2024-02-16End: 2034-12-20Target: 500Updated: 2026-03-10

Related Papers

Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials

Therapeutic Advances in Rare Disease2026-01-01

An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.

2025-09-08

Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of <scp>PROPEL</scp>

Muscle & Nerve2025-05-072 citations

Challenges in multinational rare disease clinical studies during COVID-19: regulatory assessment of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease

Journal of Neurology2025-01-012 citations

Evaluation of the safety and efficacy of miglustat for the treatment of Chinese patients with Niemann-Pick disease type C: A prospective, open-label, single-arm, phase IV trial

Intractable & Rare Diseases Research2024-11-292 citations

Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.

2024-11-134 citations

Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial

Journal of health economics and outcomes research2024-09-181 citations

Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial

Journal of health economics and outcomes research2024-09-18

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).

2024-02-2811 citations

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS<sup>®</sup>) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data.

2024-01-314 citations

Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom

Journal of health economics and outcomes research2023-03-037 citations

Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom

Journal of health economics and outcomes research2023-01-017 citations

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.

2021-12-0185 citations

GM1 Gangliosidosis-A Mini-Review.

2021-09-0364 citations

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.

2012-12-2731 citations