Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

NCT04676373 | Completed Phase 4

• 4 other identifiers: ALGMYL09010U1111-1238-1267LPS156772024-000461-24
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

Brief Summary

Primary Objective: To evaluate the effect of one-year Alglucosidase alfa treatment on motor function \[Six-minute walk test (6MWT) and lung function predicted Forced vital capacity (FVC)\] among Chinese Late Onset Pompe Disease patients above 5 years old. To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old. Secondary Objective: To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test (MMT), Maximal inspiratory and expiratory pressure (MIP and MEP)\], Quick Motor Function Test scores, and health-related quality of life (SF-12) among LOPD patients over 5 years old.

Conditions

Pompe's Disease

Outcome Measures

Primary Outcomes (3)

• Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old

  This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.

  Baseline to 12 months

• Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old

  The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.

  Baseline to 12 months

• Number of participants with adverse events Number of AEs

  An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.

  From signing the ICF to the 30th day after the last dosage of the study medications

Secondary Outcomes (5)

• Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old

  Week 52

• Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old

  Week 52

• Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old

  Week 52

• Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old

  Week 52

• Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old

  Week 52

Study Arms (1)

Alglucosidase Alfa

EXPERIMENTAL

A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks

Drug: ALGLUCOSIDASE ALFA

Interventions

ALGLUCOSIDASE ALFA DRUG

Pharmaceutical form:solution for infusion Route of administration: intravenous

Also known as: MYOZYME

Alglucosidase Alfa

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures
• The patient must be ≥ 3 years of age at the time of enrollment.
• For patient ≥ 3-year and \< 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
• For patients ≥5-year old
• i. Must be able to ambulate 40 meters in 6 minutes without assistance ii. Must be able to successfully perform repeated forced vital capacity (VC) measurements in upright position of ≥ 30% predicted and ≤85% predicted.
• The patient has confirmed Pompe's Disease with at least 2 of the following condition,
• GAA enzyme deficiency from any tissue source.
• confirmed GAA gene mutations.
• muscle pathology meet the diagnosis of Pompe disease.
• The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
• The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline.

You may not qualify if:

• Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.)
• Use of non-invasive ventilatory support while awake and in an upright position. (Non-invasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube.)
• Previously treated with Enzyme Replacement Treatment.
• A Female patient of childbearing potential with a positive pregnancy test.
• Wheelchair dependent.
• The patient has a major congenital anomaly.
• The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
• The patients with ≥5-year old are unable to ambulate 40 meters without assistance or unable to successfully perform repeated FVC of \>30% and \<85% predicted (upright).
• 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

China, China

Location

Related Links

• ALGMYL09010 Plain language Results Summary

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: December 15, 2020
• First Posted: December 21, 2020
• Study Start: March 10, 2021
• Primary Completion: July 25, 2024
• Study Completion: July 25, 2024
• Last Updated: February 10, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)