Clinical Trials Logo

Pompe Disease (Late-onset) clinical trials

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

Filter by:

NCT ID: NCT04120168 Completed - Clinical trials for Duchenne Muscular Dystrophy

Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease

VICTORIA
Start date: April 1, 2019
Phase:
Study type: Observational [Patient Registry]

This is a multicenter prospective non-drug screening study. The working period is 12 months. There is no research product to be followed or used in the study. Demographic data, medical and family histories of the patients included in the study will be collected at the first admission. The following laboratory values of the patients will be collected: - Alanine Transaminase (ALT) - Aspartate Transaminase (AST) - Gamma Glutamyl Transferase (GGT) - Creatine Phosphokinase (CPK) - In addition, physical examination information and Abdominal USG and Liver Biopsy Results, if any, will be collected. Following the above scans, enzyme analysis for late-onset Pompe disease in boys and girls and adolescents with high CPK levels and molecular genetic tests for Duchenne muscular dystrophy in boys and adolescents with high CPK levels will be performed.

NCT ID: NCT04094948 Withdrawn - Clinical trials for Pompe Disease (Late-onset)

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Start date: January 1, 2023
Phase: Phase 2
Study type: Interventional

The goals of this study are to determine safety and efficacy with regard to motor function of oral clenbuterol in combination with ERT in subjects with LOPD

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: NCT03911505 Recruiting - Clinical trials for Pompe Disease (Late-onset)

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

Start date: February 13, 2020
Phase: Phase 3
Study type: Interventional

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years

NCT ID: NCT03893240 Completed - Pompe Disease Clinical Trials

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Start date: June 12, 2019
Phase: N/A
Study type: Interventional

The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.

NCT ID: NCT03729362 Completed - Clinical trials for Pompe Disease (Late-onset)

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

PROPEL
Start date: December 4, 2018
Phase: Phase 3
Study type: Interventional

This is a phase 3 double-blind randomized study to study the efficacy and safety of intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe Disease compared with Alglucosidase Alfa/placebo.

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: NCT03285126 Completed - Clinical trials for Pompe Disease (Late-onset)

Pompe Gene Therapy- Screening for Eligibility

Start date: November 1, 2017
Phase:
Study type: Observational

The purpose of this study is to determine eligibility for the future clinical trial of gene therapy in adults with late-onset Pompe disease. This screening protocol will enroll up to 20 adults patients (>=18 yo) with late-onset Pompe disease. Study assessments include review of medical history, vital signs, physical examination, muscle function testing, lung function testing, blood and urine collection, serum pregnancy test and ECG. The study results will be collected to perform descriptive statistical analysis and used to determine eligibility for the proposed clinical trial of gene therapy in the future.

NCT ID: NCT01288027 Completed - Clinical trials for Pompe Disease (Late-Onset)

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Start date: June 2011
Phase: Phase 4
Study type: Interventional

This is an open-label, multicenter study of participants with late-onset Pompe disease naive to treatment with enzyme replacement therapy (ERT). The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in participants with Late-Onset Pompe disease. The secondary objectives are to characterize the disease burden in participants with late-onset Pompe disease and explore imaging, histologic, and functional assessments in these participants and to explore potential plasma or urine biomarkers relative to late-onset Pompe disease and participant's response to treatment with alglucosidase alfa (Myozyme®/Lumizyme®/GZ419829).

NCT ID: NCT00765414 Completed - Clinical trials for Pompe Disease Late-Onset

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Start date: April 2003
Phase: Phase 2
Study type: Interventional

This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.