A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

NCT03019406 · tracked via the Priya Life Science UK tracker

Sponsor

Genzyme, a Sanofi Company

Phase

Phase 2

Started

2017-10-12

Last updated

2026-01-30

Condition(s) studied

Glycogen Storage Disease Type II-Pompe's Disease

Investigational drug(s) / intervention(s)

Avalglucosidase alfa (GZ402666)Alglucosidase alfa (GZ419829)

Avalglucosidase alfa (GZ402666): Pharmaceutical form: powder for concentrate for solution for infusion, Route of administration: IV

Alglucosidase alfa (GZ419829): Pharmaceutical form: powder for concentrate for solution for infusion, Route of administration: IV

Study summary

This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.

Eligibility

Sex

ALL

Min age

6 Months

Max age

17 Years

Healthy volunteers

Accepted

Inclusion criteria: * The participants has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source. * The participants who has reached legal age of majority as defined by local regulation, or the participant's legal guardian(s) must provide signed informed consent prior to performing any study-related procedures. If the participant is legally minor per local regulations, assent shall be obtained from participants, if applicable. * The participants (and participant's legal guardian if participant is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol. * The participants is less than 18 years old. * The participants, if female and of childbearing potential, must have a negative serum pregnancy test (beta-human chorionic gonadotropin) and must not breastfeeding at screening/Baseline. * The participant has cardiomyopathy at the time of diagnosis: i.e., left ventricular mass index (LVMI) equivalent to mean age specific LVMI plus 2 standard deviations. * The participant has been receiving a stable dose of alglucosidase alfa regularly for a minimum of 6 months immediately prior to study entry. * For participants in Stage 1: The participant has documented evidence of clinical decline in at least 1 of the following parameters related to Pompe Disease and not related to intercurrent illness as assessed by the Investigator: respiratory function, motor skills, and/or cardiac parameters. * For participants in Stage 2: The participant has documented evidence of suboptimal clinical response in at least 1 of the following parameters related to Pompe Disease and not related to intercurrent illness as assessed by the Investigator: respiratory function, motor skills, and/or new onset of ptosis. Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: * The participant has high antibody titer to alglucosidase alfa. * The participant has a high risk for a severe allergic reaction to neoGAA (avalglucosidase alfa). * The participant requires any prohibited concomitant medications (e.g., immune modulatory treatment) for the duration of the study. * The participant has previously participated in any ACT14132 study cohort. * Female participant of childbearing potential not protected by highly effective contraceptive method of birth control and/or who is unwilling or unable to tested for pregnancy. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Primary outcome measure(s)

PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) — From Baseline to Week 25
Adverse event (AE): any untoward medical occurrence in participant received study drug \& did not necessarily had to have causal relationship with treatment. TEAEs: AEs developed/worsened in grade/become serious during PAP period (from the time of 1st study drug dose up to Week 25). Serious AE(SAE): any untoward medical occurrence at any dose resulted in death, was life-threatening, required inpatient hospitalization, prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect or was medically important event. TEAEs included SAEs \& non-SAEs. AESI:AE (serious/non-serious) of scientific \& medical concern specific to Sponsor's product/program, for which ongoing monitoring \& immediate notification by Investigator to Sponsor required.
PAP: Number of Participants With Infusion-associated Reactions (IARs) — From Baseline to Week 25
IARs were defined as AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to the study drug. Protocol-defined IARs: An AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 or 2 criteria: 1) event occurred from the start of infusion to the end of infusion plus 24 hours, and considered related to study drug, 2) If an AE time component was missing, compared AE Start date with infusion start date and infusion end date. If an AE Start date was between infusion start date and infusion end date plus one day, and it was related to study drug.

Trial sites (12)

Facility	City	Region
Regional Medical Genetics Center of New York Site Number : 8400002	Valhalla	New York
Duke University Medical Center Site Number : 8400001	Durham	North Carolina
Seattle Childrens Hospital and Regional Medical Center- Site Number : 8400005	Seattle	Washington
Investigational Site Number : 2500004	Nantes	France
Investigational Site Number : 2500003	Paris	France
Investigational Site Number : 2500002	Paris	France
Investigational Site Number : 2500001	Tours	France
Investigational Site Number : 3920001	Fuchu-shi	Tokyo
Investigational Site Number : 3920002	Fuchu-shi	Tokyo
Investigational Site Number : 1580001	Hsinchu	Taiwan
Investigational Site Number : 8260001	London	London, City of
Investigational Site Number : 8260002	Manchester	United Kingdom

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03019406 on ClinicalTrials.gov ↗ ← All trials in the UK