Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year… (NCT03687333) | Clinical Trial Compass
CompletedPhase 4
Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment
China10 participantsStarted 2018-12-04
Plain-language summary
Primary Objective:
To evaluate effect of 52-week treatment with Alglucosidase Alfa in the extension of survival and improvement of cardiomyopathy measured by Left Ventricular Mass Index in Chinese patients with infantile-onset Pompe Disease.
Secondary Objectives:
* To observe the improvement of physical growth, motor and cognitive development of 52-week treatment with Alglucosidase Alfa in infantile-onset Pompe Disease from the baseline.
* To observe the efficacy on survival free of invasive ventilation, use of any ventilation support of 52- week treatment with Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease.
* To evaluate the safety and tolerability of Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease.
Who can participate
Age range
0 Months – 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:
* Subject's parents or legal guardians must provide written informed consent prior to any study-related procedures.
* Documented onset of Pompe disease symptoms up to 12 months of age (corrected for gestation if born before 40 weeks); diagnosis of Pompe disease confirmed by acid alpha-glucosidase enzyme deficiency from any tissue source and acid alpha-glucosidase gene mutations.
* Age 0-12 months at enrollment, defined as at the time of providing written informed consent.
* Cardiomyopathy (abnormal left ventricular mass indices \[LVMIs\], measured by echocardiography, abnormal value is defined as ≥65 g/m2 for patients up to 12 months old) confirmed by cardiologist at study site.
Exclusion criteria:
* Patient who has previously been treated with acid alpha-glucosidase.
* Patient who is participating in another clinical study using any investigational therapy.
* Conditions/situations such as:
* Clinical signs of cardiac failure with ejection fraction \< 40%.
* Respiratory insufficiency (oxygen saturation \< 90% or carbon dioxide partial pressure \> 55 mm Hg \[venous\] or \> 40 mm hydrargyrum \[arterial\] in room air or any ventilator use).
* Patients who are dependent on invasive or non-invasive ventilator support.
* Patients with major congenital anomaly or clinically significant intercurrent organic disease unrelated to Pompe disease.
* Patients not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clin…
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.