Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment (APOLLO-IOPD)

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

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.

Study Overview

• Status: Completed
• Conditions: Glycogen Storage Disease Type II
• Intervention / Treatment: Drug: ALGLUCOSIDASE ALFA (MYOZYME)

Detailed Description

Total of 56 weeks in the study period, including an up to 28-day screening period and 52-week treatment period, followed by 30-day post-treatment observation period.

After the end of 52-week treatment, patients' guardians could choose to participate in a patient assistance program (PAP) sponsored by Sanofi and launched before first patient out (FPO) or reimbursement from social insurance for continued treatment.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Shanghai, China
    • Investigational Site Number

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second to 1 year (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 clinical conditions, or patients 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Alglucosidase Alfa therapy; Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.	Drug: ALGLUCOSIDASE ALFA (MYOZYME); Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Survival	The proportion of patients alive at the end of study	at week 52
Left Ventricular Mass Index (LVMI)	Change from baseline in LVMI	at week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Invasive ventilation-free survival	Survival free of invasive ventilator use at 52-week treatment	at week 52
Any ventilation-free survival	Survival free of any ventilator use at 52-week treatment	at week 52
Growth in body weight and length	Physical growth: Change from baseline at Week 52 with regards to length and weight	at week 52
Motor development milestones	Number of motor development milestones achieved at Week 52 and change from baseline	at week 52
GESELL Development Scale	Change from baseline at Week 52 on GESELL Developmental Scale	at week 52
Cardiac failure	Proportion of patients with signs and/or symptoms of cardiac failure at Week 52	at week 52

Collaborators and Investigators

• Sponsor: Genzyme, a Sanofi Company

Publications and helpful links

General Publications

• Zhu D, Zhu J, Qiu W, Wang B, Liu L, Yu X, Ou Z, Shan G, Wang J, Li B, Chen X, Liu C, Li Z, Fu L. A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease. Front Pharmacol. 2022 Jun 27;13:903488. doi: 10.3389/fphar.2022.903488. eCollection 2022.

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 4, 2018
• Primary Completion (ACTUAL): December 30, 2020
• Study Completion (ACTUAL): December 30, 2020

Study Registration Dates

• First Submitted: September 24, 2018
• First Submitted That Met QC Criteria: September 25, 2018
• First Posted (ACTUAL): September 27, 2018

Study Record Updates

• Last Update Posted (ACTUAL): April 13, 2022
• Last Update Submitted That Met QC Criteria: April 5, 2022
• Last Verified: April 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Lysosomal Storage Diseases, Nervous System; Glycogen Storage Disease Type II; Glycogen Storage Disease
• Other Study ID Numbers: ALGMYL08718; U1111-1203-8484 (OTHER: UTN)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No