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About this data Sourced primarily from ClinicalTrials.gov — a listing isn't proof of safety or FDA approval.

Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.

Not yet reported

AT2221

Company
Amicus Therapeutics
Mechanism
Mechanism not yet reported
Route
Route not yet reported
Recruiting trials
3 recruiting

Efficacy results: Not yet reported

Find a Clinical Trial Near Me

3 recruiting studies currently listed on ClinicalTrials.gov.

Data source: ClinicalTrials.gov · trial data as of Oct 1, 2026

Find a Clinical Trial Near Me

See recruiting studies for AT2221 and check eligibility with the study team.

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Match
Treatment type

"Added to GLP-1" means the investigational treatment is being studied on top of an existing GLP-1-based therapy.

Your ZIP code is only used to calculate distance to trial sites — it isn't stored.

Trial details
  • Recruiting NCT NCT04808505

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

    Not yet reported Amicus Therapeutics 36 participants Est. completion 2027-07 14 sites Stand-alone
    Other conditions: Glycogen Storage Disease Type II Infantile Onset
    View official trial listing →
  • Recruiting NCT NCT06121011

    A Global Prospective Observational Registry of Patients With Pompe Disease

    Amicus Therapeutics 500 participants Est. completion 2034-12-20 41 sites
    Other conditions: Pompe Disease
    View official trial listing →
  • Recruiting NCT NCT07399704

    A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

    Not yet reported Azafaros B.V. 21 participants Est. completion 2030-08-07 3 sites
    Other conditions: GM2 Gangliosidosis, Niemann-Pick Type C Disease
    View official trial listing →
  • Completed NCT NCT00000692

    Phase I Rising Dose Tolerability Study of SC-48334 in Patients With Acquired Immunodeficiency Syndrome (AIDS) and Advanced AIDS Related Complex

    Not yet reported G D Searle 48 participants 6 sites
    Other conditions: HIV Infections
    View official trial listing →
  • Completed NCT NCT00001993

    Initial Phase II Efficacy and Safety Study of SC-48334 Administered in Combination With Low-Dose Zidovudine (AZT) to Symptomatic HIV-1 Infected Patients With = or > 200 to = or < 500 CD4+ Cells/mm3

    Not yet reported G D Searle 120 participants 6 sites
    Other conditions: HIV Infections
    View official trial listing →
  • Completed NCT NCT00002079

    Phase II Study of the Safety and Surrogate Marker Efficacy of Butyldeoxynojirimycin (SC-48334) and AZT in Symptomatic HIV-1 Infected Patients With 200 - 500 CD4+ Cells/mm3. (NOTE: Asymptomatic HIV-1 Infected Patients Also Eligible)

    Not yet reported G D Searle 9 sites
    Other conditions: HIV Infections
    View official trial listing →
  • Completed NCT NCT00041535

    OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

    Not yet reported National Institute of Neurological Disorders and Stroke (NINDS) 30 participants Est. completion 2007-03-21 2 sites
    Other conditions: Gaucher Disease
    View official trial listing →
  • Completed NCT NCT00194649

    Glycosphingolipid Inhibition and Spermatogenesis in Man: A Pilot Study (MIG 2)

    Not yet reported University of Washington 8 participants Est. completion 2006-01 1 site Stand-alone
    Other conditions: Contraception
    View official trial listing →
  • Completed NCT NCT00316498

    Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

    Not yet reported National Eye Institute (NEI) 30 participants Est. completion 2007-08-15 3 sites
    Other conditions: Niemann Pick Diseases
    View official trial listing →
  • Completed NCT NCT00319046

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

    Not yet reported Actelion 42 participants Est. completion 2010-07-01 Stand-alone
    Other conditions: Gaucher Disease Type 1
    View official trial listing →
  • Completed NCT NCT00418847

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

    Not yet reported The Hospital for Sick Children 5 participants Est. completion 2009-04 1 site Stand-alone
    Other conditions: Gangliosidoses GM2
    View official trial listing →
  • Completed NCT NCT00517153

    Miglustat in Niemann-Pick Type C Disease

    Not yet reported Actelion 29 participants Est. completion 2008-01 Stand-alone
    Other conditions: Niemann-Pick Type C Disease
    View official trial listing →
  • Terminated NCT NCT00537602

    Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

    Not yet reported Actelion 6 participants Est. completion 2008-03 1 site Stand-alone
    Other conditions: Cystic Fibrosis
    View official trial listing →
  • Completed NCT NCT00672022

    Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

    Not yet reported Children's National Research Institute 10 participants Est. completion 2007-08 1 site
    Other conditions: GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
    View official trial listing →
  • Completed NCT NCT00742092

    Miglustat in Cystic Fibrosis

    Not yet reported Actelion 11 participants Est. completion 2008-12 1 site Stand-alone
    Other conditions: Cystic Fibrosis
    View official trial listing →
  • Completed NCT NCT00945347

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

    Not yet reported Cliniques universitaires Saint-Luc- Université Catholique de Louvain 10 participants Est. completion 2011-06 1 site
    Other conditions: Cystic Fibrosis
    View official trial listing →
  • Completed NCT NCT01451879

    Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

    University of Florida 11 participants Est. completion 2017-10 1 site
    Other conditions: Pompe Disease
    View official trial listing →
  • Completed NCT NCT01760564

    Application of Miglustat in Patients With Niemann-Pick Type C

    Not yet reported National Taiwan University Hospital 5 participants Est. completion 2010-12 1 site Stand-alone
    Other conditions: Niemann-Pick Disease Type C
    View official trial listing →
  • Completed NCT NCT01822028

    Two Period / Two Treatment Cross-over to Assess the Effect of Florastor® on Gastrointestinal Tolerability, Safety, and PK in Healthy Subjects Receiving Zavesca®

    Not yet reported Actelion 42 participants Est. completion 2013-08 1 site
    Other conditions: Diarrhea
    View official trial listing →
  • Terminated NCT NCT02030015

    Synergistic Enteral Regimen for Treatment of the Gangliosidoses

    Not yet reported University of Minnesota 16 participants Est. completion 2019-07-31 1 site Stand-alone
    Other conditions: GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
    View official trial listing →
  • Terminated NCT NCT02185651

    A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction

    Not yet reported University of Florida 2 participants Est. completion 2018-07
    Other conditions: Pompe Disease, Hypersensitivity Reaction
    View official trial listing →
  • Completed NCT NCT02325362

    Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation

    Not yet reported, Not yet reported Assistance Publique - Hôpitaux de Paris 16 participants Est. completion 2017-04-03 1 site Stand-alone
    Other conditions: Cystic Fibrosis
    View official trial listing →
  • Status unknown NCT NCT02520934

    Miglustat on Gaucher Disease Type IIIB

    Not yet reported National Taiwan University Hospital 19 participants Est. completion 2019-12 1 site Stand-alone
    Other conditions: Gaucher Disease
    View official trial listing →
  • Completed NCT NCT02675465

    First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

    Not yet reported, Not yet reported Amicus Therapeutics 29 participants Est. completion 2024-08-22 19 sites Stand-alone
    Other conditions: Pompe Disease
    View official trial listing →
  • Completed NCT NCT03729362

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

    Not yet reported Amicus Therapeutics 125 participants Est. completion 2021-01-15 73 sites Stand-alone
    Other conditions: Pompe Disease (Late-onset)
    View official trial listing →
  • Terminated NCT NCT03822013

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

    Not yet reported Tehran University of Medical Sciences 30 participants Est. completion 2025-09-10 3 sites Stand-alone
    Other conditions: GM2 Gangliosidosis, Supportive Care
    View official trial listing →
  • Available NCT NCT03865836

    Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

    Amicus Therapeutics
    Other conditions: Pompe Disease
    View official trial listing →
  • Completed NCT NCT03910621

    Safety and Efficacy of Miglustat in Chinese NPC Patients

    Not yet reported Actelion 17 participants Est. completion 2022-03-25 2 sites Stand-alone
    Other conditions: Niemann-Pick Disease, Type C
    View official trial listing →
  • Active, not recruiting NCT NCT03911505

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

    Not yet reported Amicus Therapeutics 21 participants Est. completion 2027-09 17 sites Stand-alone
    Other conditions: Pompe Disease (Late-onset)
    View official trial listing →
  • Completed NCT NCT04138277

    A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

    Not yet reported Amicus Therapeutics 119 participants Est. completion 2024-12-31 60 sites Stand-alone
    Other conditions: Pompe Disease (Late-onset)
    View official trial listing →
  • Available NCT NCT04327973

    Expanded Access for ATB200/AT2221 for the Treatment of IOPD

    Amicus Therapeutics 4 sites
    Other conditions: Pompe Disease Infantile-Onset
    View official trial listing →
  • Completed NCT NCT04768166

    Testing Miglustat Administration in Subjects With Spastic Paraplegia 11

    Not yet reported IRCCS Fondazione Stella Maris 10 participants Est. completion 2021-09-15 1 site Stand-alone
    Other conditions: Hereditary Spastic Paraparesis
    View official trial listing →
  • Completed NCT NCT05174039

    An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

    Not yet reported, Not yet reported Beyond Batten Disease Foundation 6 participants Est. completion 2024-05-30 1 site Stand-alone
    Other conditions: Batten Disease
    View official trial listing →
Efficacy history

Not yet reported.

Safety

Not yet reported.

Sources

Also known as: AT2221, Miglustat

Related On The Pen coverage

No related coverage yet.

This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.