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

Glycogen Storage Disease Type II Clinical Trial

— APOLLO-IOPD  

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03687333
• Other study ID #: ALGMYL08718
• Secondary ID: U1111-1203-8484
• Status: Completed
• Phase: Phase 4
• Start date: December 4, 2018
• Est. completion date: December 30, 2020

Study information

• Verified date: April 2022
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

Total of 56 weeks in the study period, including an up to 28-day screening period and 52-week treatment period, followed by 30-day post-treatment observation period. After the end of 52-week treatment, patients' guardians could choose to participate in a patient assistance program (PAP) sponsored by Sanofi and launched before first patient out (FPO) or reimbursement from social insurance for continued treatment.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: December 30, 2020
• Est. primary completion date: December 30, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 12 Months

Eligibility

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.

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease Type II

Intervention

Drug:
• ALGLUCOSIDASE ALFA (MYOZYME)
Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion

Locations

Country	Name	City	State
China	Investigational site number	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Survival	The proportion of patients alive at the end of study	at week 52
Primary	Left Ventricular Mass Index (LVMI)	Change from baseline in LVMI	at week 52
Secondary	Invasive ventilation-free survival	Survival free of invasive ventilator use at 52-week treatment	at week 52
Secondary	Any ventilation-free survival	Survival free of any ventilator use at 52-week treatment	at week 52
Secondary	Growth in body weight and length	Physical growth: Change from baseline at Week 52 with regards to length and weight	at week 52
Secondary	Motor development milestones	Number of motor development milestones achieved at Week 52 and change from baseline	at week 52
Secondary	GESELL Development Scale	Change from baseline at Week 52 on GESELL Developmental Scale	at week 52
Secondary	Cardiac failure	Proportion of patients with signs and/or symptoms of cardiac failure at Week 52	at week 52