Pompe's Disease

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Pompe's Disease trials you may qualify for

100

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 21 trials

RecruitingPhase 1/2

NCT07282847

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Lat…

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult p…

United States12 participantsStarted 2026-04-15

Treatment: AB-1009 (GAA Gene)

RecruitingPhase 2

NCT07123155

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

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an…

United States45 participantsStarted 2025-10-30

Treatment: S-606001, Placebo

RecruitingNot Applicable

NCT00231400

Pompe Disease Registry Protocol

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to trac…

United States2,000 participantsStarted 2004-09-15

RecruitingNot Applicable

NCT00567073

Pompe Pregnancy Sub-Registry

This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woma…

United States20 participantsStarted 2007-06-18

RecruitingNot Applicable

NCT01665326

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the d…

United States400 participantsStarted 2009-09

Treatment: Observational

RecruitingNot Applicable

NCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…

United States250 participantsStarted 2022-05-31

Treatment: There is no intervention

All recent trials (100 shown)

CompletedEarly Phase 1

NCT06109948

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

Study ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single ascending dose…

United States, Canada46 participantsStarted 2023-10-19

Treatment: ABX1100 injection for IV infusion, Placebo injection for IV infusion

CompletedPhase 1

NCT03533673

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Open-label, ascending dose trial of ACTUS-101 administered intravenously.

United States7 participantsStarted 2018-12-17

Treatment: ACTUS-101

Pompe's Disease

Trial phase breakdown

Trial status

Actively recruiting — 21 trials

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Lat…

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

Pompe Disease Registry Protocol

Pompe Pregnancy Sub-Registry

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Registry of Patients Diagnosed With Lysosomal Storage Diseases

All recent trials (100 shown)

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Trial phase breakdown

Trial status

Actively recruiting — 21 trials

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Lat…

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

Pompe Disease Registry Protocol

Pompe Pregnancy Sub-Registry

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Registry of Patients Diagnosed With Lysosomal Storage Diseases

All recent trials (100 shown)

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

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

Gene Transfer Study in Patients With Late Onset Pompe Disease

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With I…

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

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

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

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

High Dose Inspiratory Muscle Training in LOPD

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

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

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

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

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.

Baby Detect : Genomic Newborn Screening