Pompe Disease Pipeline — VivoSeq

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

Recruiting·DDenali Therapeutics Inc.·Phase 1·NCT07354724·32 enrolled

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 Participants With Late-Onset Pompe Disease (LOPD)

Recruiting·AAskBio Inc·Preclinical·NCT07282847·12 enrolled

Electrophysiological Study of the Phrenic Nerve and Diaphragm in Pompe Disease: Retrospective and Prospective Cohort Analysis

Recruiting·IIRCCS National Neurological Institute "C. Mondino" Foundation·N/A·NCT07664930·20 enrolled

A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease

Recruiting· Shionogi logo

Shionogi·Phase 2·NCT07123155·45 enrolled

A Global Prospective Observational Registry of Patients With Pompe Disease

Recruiting·AAmicus Therapeutics·N/A·NCT06121011·500 enrolled

A Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients With Late-Onset Pompe Disease

Active, not recruiting· Astellas logo

Astellas·Preclinical·NCT06150820·119 enrolled

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

Active, not recruiting·AAmicus Therapeutics·Phase 3·NCT03911505·21 enrolled

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease GAA Variant Identified by Newborn Screening

Active, not recruiting·DDuke University·N/A·NCT03694561·20 enrolled

A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease

Active, not recruiting· Astellas logo

Astellas·Preclinical·NCT04174105·11 enrolled

PROPEL-2: Cipaglucosidase + Miglustat in Late-Onset Pompe

Active, not recruiting· Amgen logo

Amgen·Phase 3·NCT05700001·175 enrolled

PROPEL: Cipaglucosidase alfa + Miglustat in Pompe

Active, not recruiting· Amgen logo

Amgen·Phase 3·NCT04681717·123 enrolled

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

Active, not recruiting·AAskBio Inc·Phase 1·NCT03533673·7 enrolled

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Active, not recruiting· Sanofi logo

Sanofi·Phase 2·NCT03019406·22 enrolled

A Phase 1b, Multicenter, Single-arm, Open-label Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of S-606001 as a Monotherapy in Adult Patients With Late-onset Pompe Disease

Not yet recruiting· Shionogi logo

Shionogi·Phase 1·NCT07787650·15 enrolled

A Phase 2, Open-label, Multicenter, Extension Study to Investigate the Long-term Safety and Efficacy of S-606001 in Patients With Late-onset Pompe Disease

Not yet recruiting· Shionogi logo

Shionogi·Phase 2·NCT07750990·60 enrolled

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