NCT07834320

Brief Summary

Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P25-P50 for all trials

Timeline
121mo left

Started Oct 2023

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress23%
Oct 2023Oct 2036

Study Start

First participant enrolled

October 1, 2023

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

April 19, 2024

Completed
2.4 years until next milestone

First Posted

Study publicly available on registry

September 22, 2026

Completed
10 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2036

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2036

Last Updated

September 22, 2026

Status Verified

January 1, 2026

Enrollment Period

13 years

First QC Date

April 19, 2024

Last Update Submit

September 16, 2026

Conditions

Pompe Disease

Keywords

Pompe Disease

Outcome Measures

Primary Outcomes (1)

  • Urinary Glc4/Hex4

    the primary endpoints for effectiveness analysis of avalglucosidase alfa. Urinary Glc4/Hex4 is usually elevated in Pompe patients and are sensitive though nonspecific biomarkers for diagnosis and monitoring of IOPD or LOPD

    3years

Secondary Outcomes (3)

  • Creatine kinase

    3years

  • motor function (Quick Motor Function Test)

    3 years

  • respiratory function (forced vital capacity).

    3 years

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Pompe Patients receiving Avalglucosidase alfa

You may qualify if:

  • Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient

You may not qualify if:

  • Having severe allergic reaction to avalglucosidase alfa.
  • Patient/parent/legal guardian is not agreeing to provide signed informed consent, and the patient, if \<18 years of age

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Taiwan University Hospital

Taipei, Taiwan, 100, Taiwan

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Yin-Hsiu Chien, MD, PhD

    National Taiwan University Hospital Taipei, Taiwan, 100

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yin-Hsiu Chien, MD, PhD

CONTACT

+886223123456chienyh@ntu.edu.tw

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Attending Physician

Study Record Dates

First Submitted

April 19, 2024

First Posted

September 22, 2026

Study Start

October 1, 2023

Primary Completion (Estimated)

October 1, 2036

Study Completion (Estimated)

October 1, 2036

Last Updated

September 22, 2026

Record last verified: 2026-01

Locations

TW(1)