CompletedPhase 4

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

China41 participantsStarted 2021-03-10

Plain-language summary

Primary Objective: To evaluate the effect of one-year Alglucosidase alfa treatment on motor function \[Six-minute walk test (6MWT) and lung function predicted Forced vital capacity (FVC)\] among Chinese Late Onset Pompe Disease patients above 5 years old. To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old. Secondary Objective: To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test (MMT), Maximal inspiratory and expiratory pressure (MIP and MEP)\], Quick Motor Function Test scores, and health-related quality of life (SF-12) among LOPD patients over 5 years old.

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. For patient ≥ 3-year and \< 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
. For patients ≥5-year old
. GAA enzyme deficiency from any tissue source.
. 2 confirmed GAA gene mutations.
. muscle pathology meet the diagnosis of Pompe disease.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old

Timeframe: Baseline to 12 months

Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old

Timeframe: Baseline to 12 months

Number of participants with adverse events Number of AEs

Timeframe: From signing the ICF to the 30th day after the last dosage of the study medications

Trial details

NCT IDNCT04676373

SponsorGenzyme, a Sanofi Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-07-25

View on ClinicalTrials.gov More Pompe's Disease trials

Plain-language summary

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. For patient ≥ 3-year and \< 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
. For patients ≥5-year old
. GAA enzyme deficiency from any tissue source.
. 2 confirmed GAA gene mutations.
. muscle pathology meet the diagnosis of Pompe disease.

What they're measuring

Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old

Timeframe: Baseline to 12 months

Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old

Timeframe: Baseline to 12 months

Number of participants with adverse events Number of AEs

Timeframe: From signing the ICF to the 30th day after the last dosage of the study medications