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NCT05017402 Clinical Trial

Higher Dose of Alglucosidase Alpha for Pompe Disease

Glycogen Storage Disease Type II Clinical Trial

Official title:
Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study

Clinical Trial Details — Status: Not yet recruiting

Administrative data
NCT number NCT05017402
Other study ID # 2021-02-019A
Secondary ID
Status Not yet recruiting
Phase
First received
Last updated
Start date September 1, 2021
Est. completion date December 31, 2026

Study information
Verified date August 2021
Source Taipei Veterans General Hospital, Taiwan
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.

Recruitment information / eligibility
Status Not yet recruiting
Enrollment 36
Est. completion date December 31, 2026
Est. primary completion date December 31, 2023
Accepts healthy volunteers No
Gender All
Age group N/A to 60 Years
Eligibility Inclusion Criteria: 1. Diagnosis of Pompe disease 2. Currently receiving ERT with regular clinic visits 3. Agree to sign informed consent Exclusion Criteria: 1. Lost follow-up for clinical visits 2. Allergy to Myozyme 3. Refuse to sign informed consent

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• Alglucosidase Alfa
Standard dose: 20-25 mg/kg every other week; Higher dose: >25 mg/kg every other week.

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Taipei Veterans General Hospital, Taiwan

Outcome
Type Measure Description Time frame Safety issue
Primary Forced vital capacity Pulmonary function test (Units: percentage of predictive value) For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Primary Peak expiratory flow Pulmonary function test (Units: percentage of predictive value) For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Primary Polysomnography Comprehensive test used to diagnose sleep disorders. For patient older than 6-month-old, first test before study, then every six months, up to 2-years.
Secondary uGLC4 Urine glucose tetrasaccharide (Units: mmol/mol Creatinine). uGLC4 will be tested before the treatment, then every three months, up to 2-years.
Secondary CK Blood creatine kinase (Units: units per liter, U/L). CK will be tested before the treatment, then every three months, up to 2-years.
Secondary AST Blood aspartate aminotransferase (Units: units per liter, U/L). AST will be tested before the treatment, then every three months, up to 2-years.
Secondary ALT Blood alanine aminotransferase (Units: units per liter, U/L). ALT will be tested before the treatment, then every three months, up to 2-years.
Secondary Body weight Change of body weight (Unit: kilogram, kg) The body weight will be monitored before the treatment, then every two weeks, up to 2-years.
Secondary Body height Change of body height (Units: centimeter, cm) The body height will be monitored before the treatment, then every two weeks, up to 2-years.
Secondary Antibody titers Alglucosidase alfa IgG antibody titer First test will be one month later after first ERT, then every six months, up to 2-years.
See also
  Status Clinical Trial Phase
Recruiting NCT00231400 - Pompe Disease Registry Protocol
Recruiting NCT04910776 - Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa Phase 3
Withdrawn NCT01656590 - High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease Phase 2
Completed NCT00701129 - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease Phase 4
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT02363153 - Diet and Exercise in Pompe Disease N/A
Completed NCT00074932 - Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease N/A
Completed NCT00025896 - Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease Phase 2
Completed NCT00001331 - Genetic and Family Studies of Inherited Muscle Diseases N/A
Completed NCT00077662 - A Prospective, Observational Study in Patients With Late-Onset Pompe Disease N/A
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT00125879 - Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 Phase 2/Phase 3
Recruiting NCT04848779 - A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Active, not recruiting NCT05164055 - Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) Phase 4
Recruiting NCT02761421 - Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT N/A
Active, not recruiting NCT02635269 - Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy N/A
Completed NCT00051935 - A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II Phase 2
Completed NCT00053573 - rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) Phase 1/Phase 2
Completed NCT02801539 - Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) N/A
Completed NCT03687333 - Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment Phase 4