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

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ClinicalTrials.gov Identifier: NCT03687333
Recruitment Status : Recruiting
First Posted : September 27, 2018
Last Update Posted : December 12, 2018
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

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.

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II Drug: ALGLUCOSIDASE ALFA (MYOZYME) Phase 4

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : December 4, 2018
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021


Arm Intervention/treatment
Experimental: Alglucosidase Alfa therapy
Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.
Drug: ALGLUCOSIDASE ALFA (MYOZYME)

Pharmaceutical form: cake or powder for injection

Route of administration: intravenous infusion





Primary Outcome Measures :
  1. Survival [ Time Frame: at week 52 ]
    Survival at week 52 or last available follow-up (the last time point at which a given outcome is measured) will be analyzed using Kaplan-Meier time-to-event analysis; the binomial proportion of patients alive at the end of study (at week 52) will be also calculated.

  2. Left Ventricular Mass Index (LVMI) [ Time Frame: at week 52 ]
    Change from baseline in LVMI


Secondary Outcome Measures :
  1. Invasive ventilation-free survival [ Time Frame: at week 52 ]
    Survival free of invasive ventilator use at 52-week treatment (using Kaplan-Meier methodology)

  2. Any ventilation-free survival [ Time Frame: at week 52 ]
    Survival free of any ventilator use at 52-week treatment (using Kaplan-Meier methodology)

  3. Growth in body weight and length [ Time Frame: at week 52 ]
    Physical growth: Change from baseline at Week 52 with regards to length and weight

  4. Motor development milestones [ Time Frame: at week 52 ]
    Number of motor development milestones achieved at Week 52 and change from baseline

  5. GESELL Development Scale [ Time Frame: at week 52 ]
    Change from baseline at Week 52 on GESELL Developmental Scale

  6. Hours of ventilation [ Time Frame: at week 52 ]
    Change from baseline at Week 52 with respect to hours of ventilation (both invasive and non-invasive) per day

  7. Cardiac failure [ Time Frame: at week 52 ]
    Proportion of patients with signs and/or symptoms of cardiac failure at Week 52



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03687333


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-US@sanofi.com

Locations
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China
Investigational site number Recruiting
Shanghai, China
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Clinical Sciences & Operations Sanofi

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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT03687333     History of Changes
Other Study ID Numbers: ALGMYL08718
U1111-1203-8484 ( Other Identifier: UTN )
First Posted: September 27, 2018    Key Record Dates
Last Update Posted: December 12, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors