Pompe Disease clinical trials

Explore recruiting pompe disease clinical trials.

Recruiting pompe disease studies right now

Study details & eligibility ClinicalTrials.gov record

Also recruiting in Europe

Pompe Disease trials registered in the EU Clinical Trials Information System (CTIS). These are outside the "near me" matcher above - open a study for full details.

A French multicenter Phase 4 open label extension study of long-term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France

France

Therapeutic use (Phase IV) EU CTIS Study details

GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE

Denmark

Therapeutic use (Phase IV) EU CTIS Study details

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

Spain, Netherlands, Germany, Belgium, Italy

Therapeutic confirmatory (Phase III) EU CTIS Study details

Open label, single-center pilot study to investigate enzyme replacement therapy (ERT) frequency reduction from 2 to 4 weeks in a subgroup of elderly patients with late-onset Pompe disease

Netherlands

Therapeutic use (Phase IV) EU CTIS Study details

An open-label, single-center, exploratory study of the safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged ≥ 5 years.

Netherlands

Therapeutic use (Phase IV) EU CTIS Study details

A Phase 1b/2a, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants with Late-Onset Pompe Disease

Germany, Netherlands

Phase I and Phase II (Integrated)- First administration to humans EU CTIS Study details

Pompe Disease trials by city

Pompe Disease in Toronto Pompe Disease in Vancouver Pompe Disease in Montreal Pompe Disease in Calgary Pompe Disease in Edmonton Pompe Disease in Ottawa Pompe Disease in Winnipeg Pompe Disease in Quebec City Pompe Disease in Hamilton Pompe Disease in Kitchener Pompe Disease in London Pompe Disease in Halifax Pompe Disease in New York Pompe Disease in Los Angeles Pompe Disease in Chicago Pompe Disease in Houston

Pompe Disease clinical trials

Recruiting pompe disease studies right now

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Comparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment Intervals

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD

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

Also recruiting in Europe

A French multicenter Phase 4 open label extension study of long-term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France

GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

Open label, single-center pilot study to investigate enzyme replacement therapy (ERT) frequency reduction from 2 to 4 weeks in a subgroup of elderly patients with late-onset Pompe disease

An open-label, single-center, exploratory study of the safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged ≥ 5 years.

A Phase 1b/2a, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants with Late-Onset Pompe Disease

Pompe Disease trials by city

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