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New treatment option for Glycogen Storage Disease Type II
Official title China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
ClinicalTrials.gov ID: NCT06666413
What this study is testing
What is Avalglucosidase alfa?
Avalglucosidase alfa is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for glycogen storage disease type ii.
Also referred to as GZ402666, Nexviazyme.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT. Study details include: The study duration: total study duration is approximately 64 weeks.
- Phase 4: studies an already-approved treatment
- Time commitment: about 52 weeks
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 17
You may be able to join if
- 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...
- 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...
You likely can't join if
- 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...
- 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...
- Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as...
- Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug...
See the full eligibility 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...
- 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.
- 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...
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 52 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, up to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.