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Pompe Disease clinical trials

View clinical trials related to Pompe Disease.

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NCT ID: NCT04093349 Active, not recruiting - Pompe Disease Clinical Trials

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Start date: October 1, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

NCT ID: NCT03694561 Active, not recruiting - Pompe Disease Clinical Trials

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

Start date: March 25, 2019
Phase:
Study type: Observational

This is an observational study with no study related treatment of interventions. The purpose of the study is to investigate and document disease specific clinical symptoms in newborns, infants and children with Pompe disease without cardiomyopathy identified in newborn screening(NBS). There will be baseline, months 6 and months 12 visits for infants and newborns (infants study). For children of ages 24 months to 54 months, there will be baseline, year 1 and year 2 visits (children study). The study has four goals: 1. To study and record disease specific clinical symptoms in newborns, infants and children with Pompe disease without cardiomyopathy (disease of the heart muscle) in the first year of life identified through newborn screening (NBS) 2. To devise an approach to characterize early musculoskeletal (muscles and joints) involvement in subjects with the "late-onset" GAA variant identified by NBS including ability to collect research information via virtual health platforms. 3. To determine criteria to start preventative therapies including enzyme replacement therapy (ERT) in patients with clinical features of Pompe disease identified via NBS 4. To document parental coping and anxiety/emotional distress overtime using quality of life questionnaires after a child is diagnosed with late onset Pompe disease via NBS

NCT ID: NCT03533673 Active, not recruiting - Pompe Disease Clinical Trials

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

Start date: December 17, 2018
Phase: Phase 1/Phase 2
Study type: Interventional

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

NCT ID: NCT02675465 Active, not recruiting - Pompe Disease Clinical Trials

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

Start date: January 2016
Phase: Phase 1/Phase 2
Study type: Interventional

This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.