21 completed, 4 terminated, 2 recruiting, 1 active not recruiting. 11 Phase 2, 9 Phase 3, 5 Phase 1, 3 Phase 4, 1 Not applicable.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Global Prospective Observational Registry of Patients With Pompe Disease Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa - Pompe Disease | Recruiting | Amicus Therapeutics | 500 | 2024-02-16 | 2026-03-10 | |
| A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 Cipaglucosidase alfa, Miglustat - Glycogen Storage Disease Type II Infantile Onset | Phase 3 | Recruiting | Amicus Therapeutics | 36 | 2023-07-18 | 2026-01-22 |
| Testing Miglustat Administration in Subjects With Spastic Paraplegia 11 Miglustat 100 MG - Hereditary Spastic Paraparesis | Phase 2 | Completed | IRCCS Fondazione Stella Maris | 10 | 2021-06-15 | 2022-04-11 |
| Safety and Efficacy of Miglustat in Chinese NPC Patients Miglustat - Niemann-Pick Disease, Type C | Phase 4 | Completed | Actelion | 17 | 2020-04-02 | 2025-03-30 |
| ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Cipaglucosidase Alfa, Miglustat - Pompe Disease (Late-onset) | Phase 3 | Active not recruiting | Amicus Therapeutics | 21 | 2020-02-13 | 2026-09-17 |
| A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) AT2221, ATB200 - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 119 | 2019-12-18 | 2026-03-05 |
| Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) Miglustat - GM2 Gangliosidosis, Supportive Care | Phase 3 | Terminated | Tehran University of Medical Sciences | 30 | 2019-01-14 | 2025-09-17 |
| A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 125 | 2018-12-04 | 2025-09-11 |
| A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction Zavesca® Prescription - Pompe Disease, Hypersensitivity Reaction | Phase 1 | Terminated | University of Florida | 2 | 2016-10-01 | 2018-12-05 |
| First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 ATB200, AT2221 - Pompe Disease | Phase 1 / Phase 2 | Completed | Amicus Therapeutics | 29 | 2016-04-01 | 2025-10-23 |
| Synergistic Enteral Regimen for Treatment of the Gangliosidoses miglustat, Ketogenic Diet - GM1 Gangliosidoses, GM2 Gangliosidoses | Phase 4 | Terminated | University of Minnesota | 16 | 2015-12-22 | 2021-04-14 |
| Miglustat on Gaucher Disease Type IIIB Miglustat, ERT - Gaucher Disease | Not applicable | Unknown | National Taiwan University Hospital | 19 | 2015-07-01 | 2019-03-29 |
| Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation Miglustat ; placebo, Placebo ; Miglustat - Cystic Fibrosis | Phase 2 / Phase 3 | Completed | Assistance Publique - Hôpitaux de Paris | 16 | 2015-03-17 | 2025-11-20 |
| Two Period / Two Treatment Cross-over to Assess the Effect of Florastor® on Gastrointestinal Tolerability, Safety, and PK in Healthy Subjects Receiving Zavesca® Treatment A, Treatment B - Diarrhea | Phase 1 | Completed | Actelion | 42 | 2013-03-01 | 2015-03-10 |
| Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ? Miglustat, Placebo - Cystic Fibrosis | Phase 2 | Completed | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | 10 | 2009-07-01 | 2011-08-10 |
| Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies Rituximab, Miglustat - Pompe Disease | Completed | University of Florida | 11 | 2008-10-01 | 2017-12-08 | |
| Miglustat in Cystic Fibrosis miglustat, placebo - Cystic Fibrosis | Phase 2 | Completed | Actelion | 11 | 2008-08-01 | 2014-03-07 |
| Application of Miglustat in Patients With Niemann-Pick Type C Miglustat - Niemann-Pick Disease Type C | Phase 3 | Completed | National Taiwan University Hospital | 5 | 2008-01-01 | 2013-01-04 |
| Miglustat / OGT 918 in the Treatment of Cystic Fibrosis miglustat, placebo - Cystic Fibrosis | Phase 2 | Terminated | Actelion | 6 | 2007-11-01 | 2010-02-12 |
| Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease Miglustat - Gaucher Disease Type 1 | Phase 3 | Completed | Actelion | 42 | 2006-02-01 | 2025-02-04 |
Showing 20 of 29. Sign in to see all of them.
Glycogen Storage Disease Type II (7) · Cystic Fibrosis (4) · Gangliosidoses, GM2 (4) · Sandhoff Disease (4) · Gaucher Disease (3) · HIV Infections (3) · Niemann-Pick Disease, Type C (3) · Tay-Sachs Disease (3) · AIDS-Related Complex (1) · Acquired Immunodeficiency Syndrome (1) · Diarrhea (1) · Gangliosidoses (1) · Gangliosidosis, GM1 (1) · Hypersensitivity (1) · Lysosomal Storage Diseases (1)
Actelion (6) · Amicus Therapeutics (6) · G D Searle (3) · National Taiwan University Hospital (2) · University of Florida (2) · Assistance Publique - Hôpitaux de Paris (1) · Children's National Research Institute (1) · Cliniques universitaires Saint-Luc- Université Catholique de Louvain (1) · IRCCS Fondazione Stella Maris (1) · National Eye Institute (NEI) (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · Tehran University of Medical Sciences (1) · The Hospital for Sick Children (1) · University of Minnesota (1) · University of Washington (1)
United States (15) · United Kingdom (7) · Germany (6) · Italy (6) · Belgium (5) · Canada (5) · Netherlands (5) · Taiwan (5) · Australia (4) · Austria (3) · Denmark (3) · France (3) · Greece (3) · Hungary (3) · Japan (3)
Barry J Byrne, MD, PhD (1) · Barry J. Byrne, MD, PhD (1) · Chad McQueen, PharmD (1) · Christian Domingo-Ribas, MD (1) · Claudia Chiriboga, Assoc. Prof. MD (1) · Cynthia J TIfft, MD, PhD (1) · Filippo M Santorelli, MD PhD (1) · Jeanine R. Jarnes, PharmD (1) · Joe TR Clarke, MD (1) · John Amory (1) · Patrick Lebecque, MD, PhD (1) · Paul van Giersbergen, PhD (1)
PMID 41631150, PMID 33331662, PMID 37983082, PMID 17067996, PMID 19595619, PMID 22476655, PMID 23270487, PMID 23601496, PMID 28476546, PMID 32671834, PMID 34800400, PMID 38057636, PMID 38294575, PMID 38418563, PMID 39318718, PMID 39535661, PMID 39628618, PMID 40342075, PMID 40920287, PMID 41769220
2019 (1), U54HD042454 (1), U54NS065768 (1)