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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 April 2022
• Main ID: NCT03687333
• Date of registration: 24/09/2018
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment APOLLO-IOPD
• Scientific title: 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
• Date of first enrolment: December 4, 2018
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03687333
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

China

Contacts

• Name: Clinical Sciences & Operations
• Affiliation: Sanofi

Key inclusion & exclusion criteria

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.



Age minimum: N/A
Age maximum: 12 Months
Gender: All

Health Condition(s) or Problem(s) studied

Glycogen Storage Disease Type II

Intervention(s)

Drug: ALGLUCOSIDASE ALFA (MYOZYME)

Primary Outcome(s)

Left Ventricular Mass Index (LVMI) [Time Frame: at week 52]

Survival [Time Frame: at week 52]

Secondary Outcome(s)

Motor development milestones [Time Frame: at week 52]

Any ventilation-free survival [Time Frame: at week 52]

GESELL Development Scale [Time Frame: at week 52]

Growth in body weight and length [Time Frame: at week 52]

Invasive ventilation-free survival [Time Frame: at week 52]

Cardiac failure [Time Frame: at week 52]

Secondary ID(s)

ALGMYL08718

U1111-1203-8484

Source(s) of Monetary Support

Please refer to primary and secondary sponsors