Clinical trial NCT04676373: Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Pompe's Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2020-12-21
Enrollment: 41
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Detailed description: Study duration per participants is approximatively 56 weeks including a 52-week treatment period.

Eligibility criteria: Inclusion criteria :

* 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.

  1. For patient ≥ 3-year and \< 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
  2. 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,

  1. GAA enzyme deficiency from any tissue source.
  2. 2 confirmed GAA gene mutations.
  3. 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.

Exclusion criteria:

* 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.

Primary outcomes:
measure: Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
timeFrame: Baseline to 12 months
measure: Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old
timeFrame: Baseline to 12 months
measure: Number of participants with adverse events Number of AEs
timeFrame: From signing the ICF to the 30th day after the last dosage of the study medications
Secondary outcomes:
measure: Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old
timeFrame: Week 52
measure: Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old
timeFrame: Week 52
measure: Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old
timeFrame: Week 52
measure: Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
timeFrame: Week 52
measure: Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
timeFrame: Week 52
Other outcomes:
Locations:
city: China
country: China
facility: Investigational Site
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: