China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
AgeUp to 17
SponsorGenzyme, a Sanofi Company
About this trial
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.
* Screening period of up to 8 weeks * Treatment period of 52 weeks * Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks) * The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Avalglucosidase alfa
DrugPharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion
Treatment groups
13 Participants
are divided into 1 treatment groupGroup A: avalglucosidase alfaExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of AEs, SAEs, AESIs including IARs during the TE period
Time frame
From baseline to Week 562
Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period
Time frame
From baseline to Week 56Secondary outcomes
1
Proportion of participants who are alive and free of invasive ventilation at Week 52
Time frame
At Week 522
Change from baseline to Week 52 in LVM Z score evaluated by echocardiography
Time frame
From baseline to Week 523
The absolute change in body growth Z scores from baseline to Week 52
Time frame
From baseline to Week 524
The percentiles of the body growth Z scores changes from baseline to Week 52
Time frame
From baseline to Week 52Sponsors and contacts
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Source ClinicalTrials.gov