NCT04676373CompletedPhase 4
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment
Source: ClinicalTrials.gov · Last updated 10 Feb 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 41 (actual)
- First submitted
- 15 Dec 2020
- Start date
- 10 Mar 2021
- Primary completion date
- 25 Jul 2024
- Lead sponsor
- Genzyme, a Sanofi Company
- Diseases
- Pompe disease
- NCT number
- NCT04676373
- EudraCT number
- 2024-000461-24
What you commit to
- Study type: Interventional · Phase 4
- Participants enrolled: 41
- From 10 Mar 2021 to the primary completion date, 25 Jul 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previously treated with Enzyme Replacement Treatment.
Other illness
- 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.
Other
- 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.)
- A Female patient of childbearing potential with a positive pregnancy test.
- Wheelchair dependent.
- The patient has a major congenital anomaly.
- 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.
Who the study is looking for
- 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
- 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.
- 2 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.
Age in the registry: 3 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | China | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.