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Pompe Disease (Late-onset) clinical trials

View clinical trials related to Pompe Disease (Late-onset).

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NCT ID: NCT06391736 Recruiting - Clinical trials for Pompe Disease (Late-onset)

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

Start date: April 19, 2024
Phase: Phase 1/Phase 2
Study type: Interventional

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with late-onset Pompe disease (LOPD) who are ≥ 6 years old will be studied.

NCT ID: NCT06178432 Not yet recruiting - Clinical trials for Pompe Disease (Late-onset)

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

Start date: December 2023
Phase: Early Phase 1
Study type: Interventional

This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term follow-up period of 5 years. CRG003 (BBM-G102) injection is an adeno-associated virus (AAV) gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection.

NCT ID: NCT06150820 Recruiting - Clinical trials for Pompe Disease (Late-onset)

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

Start date: February 1, 2024
Phase: N/A
Study type: Interventional
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NCT ID: NCT05951790 Recruiting - Clinical trials for Glycogen Storage Disease Type II

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

LOPD01
Start date: March 1, 2023
Phase: N/A
Study type: Interventional

The goal of this multicentre, randomized and controlled cross-over trial is to evaluate the efficacy of a programme of Inspiratory Muscle Training in subjects with Late On-set Pompe Disease (LOPD). The main question is to: - verify changes in Forced Vital Capacity, Postural Drop, Maximal Inspiratory Pressure, Maximal Expiratory Pressure, Peak expiratory cough pressure, Maximal Inspiratory Capacity, six- minute walk test and or 6-minute pegboard ring test.- - measure changes in some questionnaries investigating dispnoea and quality of life (Short-Form 36, Individualized-Neuromuscular-Quality-of-Life, Maugeri-Respiratory-Failure 28, Borg scale, Dispnoea 12, Mulditimensional Dispnea Profile, modified Medical Research Council, Fatigue Severity Scale, Epsworth Scale, Visual Analogue Scale). Measurement will take place at baseline and after one, three, four, six and twelve months. Participants will undergo a specific treatment consisting of aerobic exercise and Inspiratory Muscle Training with Powerbreathe device or Air-Stacking. Researchers will study if Powerbreathe device is more effective than Air-stacking maneuvres

NCT ID: NCT05272969 Recruiting - Clinical trials for Pompe Disease (Late-onset)

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Start date: March 31, 2022
Phase:
Study type: Observational

The primary aim of this nationwide, explorative, cross-sectional study in Germany is to characterize the prevalence, severity and quality of musculoskeletal pain in adult patients with late-onset Pompe disease (LOPD). The secondary objectives are to evaluate whether muscle pain is associated with muscle function, to assess whether muscle pain is associated with alterations of muscle tissue, and whether vitamin D metabolism and polymorphisms of ACE and ACTN3 genes may contribute to an increased level of perceived musculoskeletal pain. In a second step, exome sequencing of genes associated with musculoskeletal pain will be analyzed. Results of LOPD patients will be compared to patients with neuromuscular disorders with a similar distribution of muscle weakness and/or musculoskeletal pain.

NCT ID: NCT05083806 Recruiting - Pompe Disease Clinical Trials

MSOT in Pompe Disease

SPOT_PD
Start date: May 17, 2022
Phase: N/A
Study type: Interventional
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NCT ID: NCT04929002 Recruiting - Clinical trials for Pompe Disease (Late-onset)

Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases

Start date: December 10, 2021
Phase:
Study type: Observational

The project will use carbon-13 magnetic resonance spectroscopy to assess whether high glycogen levels in skeletal muscle of patients with Glycogen Storage Diseases is a prelude for muscle damage. Patients with Glycogen Storage Diseases will be examined using carbon-13 MR-spectroscopy to quantify the glycogen levels in lumbar, thigh and calf-muscles. The pattern of glycogen concentration will be compared to the pattern of muscle atrophy found in the literature.

NCT ID: NCT04907162 Completed - Healthy Clinical Trials

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Start date: April 15, 2021
Phase:
Study type: Observational

The primary aim is to characterize the prevalence, severity and quality of musculoskeletal nociceptive pain in adult patients with neuromuscular disorders (NMD). The secondary objectives are to evaluate whether severity and distribution of muscle pain is associated with muscle function, and to assess whether muscle pain is associated with alterations of muscle elasticity and muscle stiffness. Results of patients with neuromuscular disorders will be compared to age- and gender-matched healthy volunteers. Approx. 70 patients with neuromuscular disorders and 20 healthy volunteers will be enrolled, including patients with the following neuromuscular disorders: histologically confirmed inclusion body myositis (IBM), genetically confirmed late-onset Pompe disease (LOPD), genetically confirmed spinal muscular atrophy type 3 (SMA3), genetically confirmed facio-scapulo-humeral muscle dystrophy (FSHD), genetically confirmed myotonic dystrophy type 1 or type 2 (DM1, DM2). The duration of patient recruitment will be around 12 months.

NCT ID: NCT04174105 Recruiting - Clinical trials for Pompe Disease (Late-onset)

Gene Transfer Study in Patients With Late Onset Pompe Disease

FORTIS
Start date: October 27, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).

NCT ID: NCT04138277 Active, not recruiting - Clinical trials for Pompe Disease (Late-onset)

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Start date: December 18, 2019
Phase: Phase 3
Study type: Interventional

This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.