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

NCT03687333 | Completed Phase 4

• 2 other identifiers: ALGMYL08718U1111-1203-8484
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Glycogen Storage Disease Type II

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (6)

• Invasive ventilation-free survival

  at week 52

• Any ventilation-free survival

  at week 52

• Growth in body weight and length

  at week 52

• Motor development milestones

  at week 52

• GESELL Development Scale

  at week 52

Study Arms (1)

Alglucosidase Alfa therapy

EXPERIMENTAL

Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.

Drug: ALGLUCOSIDASE ALFA (MYOZYME)

Interventions

ALGLUCOSIDASE ALFA (MYOZYME) DRUG

Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion

Alglucosidase Alfa therapy

Eligibility Criteria

• Age: 0 Months - 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (1)

Investigational site number

Shanghai, China

Location

Related Publications (1)

• 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.

  PMID: 35833019 DERIVED

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 24, 2018
• First Posted: September 27, 2018
• Study Start: December 4, 2018
• Primary Completion: December 30, 2020
• Study Completion: December 30, 2020
• Last Updated: April 13, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)