Avalglucosidase alfa treatment for children with Pompe disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a 52-week, open-label, single-arm study assessing the safety and efficacy of avalglucosidase alfa IV infusion in Chinese participants diagnosed with infantile-onset Pompe disease (IOPD). The study includes a screening period of up to 8 weeks, followed by a treatment phase lasting 52 weeks, and a 4-week follow-up. Participants will have a total of 30 visits, including 29 site visits and one follow-up phone call. The study aims to evaluate the effects of the treatment on participants who are either treatment-naïve or have previously received enzyme replacement therapy.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Participant must be \<18 years of age, at the time of signing the informed consent.
* Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.
* Participants must have documented cardiomyopathy at the time of diagnosis.
* Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent.

Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:

* Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
* Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
* Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.
* Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.
* Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.
* Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Where it's running

• Investigational Site Number: 1560001
  Shanghai, China Recruiting

Study contacts

• Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
• Email: contact-us@sanofi.com
• Phone: 800-633-1610

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Global registry for tracking Pompe disease outcomes

  This study is tracking the health and progress of people with Pompe disease to learn more about how it affects them and to see how well treatments are working.

  Recruiting at 207 sites Sanofi · Birmingham, Alabama and 281 other locations · NCT00231400

• Pompe disease long-term monitoring with avalglucosidase alfa

  This is an observational program to see how people with Pompe disease who started avalglucosidase alfa fare over time, including biomarkers, muscle and breathing function, and safety.

  Recruiting National Taiwan University Hospital · Taipei, Taiwan · NCT07834320

• Phrenic nerve and diaphragm testing in Pompe disease

  This study tests phrenic nerve conduction and diaphragm activity in adults with Pompe disease to explore respiratory motor-unit involvement.

  Recruiting IRCCS National Neurological Institute "C. Mondino" Foundation · Pavia · NCT07664930

• S-606001 added to enzyme replacement therapy for adults with late-onset Pompe disease

  This trial tests whether adding S-606001 to ongoing enzyme replacement therapy is safe and can improve walking distance and breathing in adults with late-onset Pompe disease.

  Recruiting at 27 sites Phase 2 Shionogi Inc. · Irvine, California and 27 other locations · NCT07123155

• Global registry for patients with Pompe disease

  This study is collecting information from people with Pompe disease to see how well current treatments work and how they affect daily life, including both those who are being treated and those who aren't.

  Recruiting at 29 sites Amicus Therapeutics · Little Rock, Arkansas and 40 other locations · NCT06121011

• Safety study of avalglucosidase alfa during pregnancy

  This study is looking at how safe the drug avalglucosidase alfa is for pregnant women and their babies during pregnancy and breastfeeding.

  Recruiting Sanofi · Bridgewater, New Jersey · NCT05734521

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.