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

NCT ID: NCT03687333

Last Updated: 2022-04-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-12-04
• Study Completion Date: 2020-12-30

Brief 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.

Detailed 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.

Conditions

Glycogen Storage Disease Type II

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Alglucosidase Alfa therapy

Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.

Group Type: EXPERIMENTAL

ALGLUCOSIDASE ALFA (MYOZYME)

Intervention Type: DRUG

Pharmaceutical form: cake or powder for injection

Route of administration: intravenous infusion

Interventions

ALGLUCOSIDASE ALFA (MYOZYME)

Pharmaceutical form: cake or powder for injection

Route of administration: intravenous infusion

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 0 Months
• Maximum Eligible Age: 12 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Investigational site number

Shanghai, , China

Countries

China

References

Zhu D, Zhu J, Qiu W, Wang B, Liu L, Yu X, Ou Z, Shan G, Wang J, Li B, Chen X, Liu C, Li Z, Fu L. A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease. Front Pharmacol. 2022 Jun 27;13:903488. doi: 10.3389/fphar.2022.903488. eCollection 2022.

Reference Type: DERIVED

PMID: 35833019 (View on PubMed)

Other Identifiers

U1111-1203-8484

Identifier Type: OTHER

Identifier Source: secondary_id

ALGMYL08718

Identifier Type: -

Identifier Source: org_study_id