TerminatedPhase 4

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Stopped: Study was stopped due to approved label expansion of alglucosidase alfa.

United States, Germany, Taiwan4 participantsStarted 2012-08

Plain-language summary

A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.

Who can participate

Age range

12 Months

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: * The participant's parent/legal guardian was willing and able to provide signed informed consent. * The participant might be less than or equal to 12 months of age. * The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue. * The participant might be naïve to treatment with alglucosidase alfa. Exclusion Criteria: * The participant was cross-reactive immunologic material negative. * The participant required invasive ventilator support at the time of enrollment. * The participant had decompensated clinical heart failure. * The participant had a major congenital abnormality, excluding cardiac hypertrophy. * The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease). * The participant was currently receiving any investigational product. * The participant was participating in another clinical study. * The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.

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 Cardiac Function at Week 52

Timeframe: Baseline, Week 52

Trial details

NCT IDNCT01597596

SponsorGenzyme, a Sanofi Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2014-12

Results submitted2015-09-25

View on ClinicalTrials.gov More Pompe Disease (Infantile-Onset) trials