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
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.
- 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|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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
- 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.
- Left Ventricular Mass Index (LVMI) [ Time Frame: at week 52 ]Change from baseline in LVMI
- Invasive ventilation-free survival [ Time Frame: at week 52 ]Survival free of invasive ventilator use at 52-week treatment (using Kaplan-Meier methodology)
- Any ventilation-free survival [ Time Frame: at week 52 ]Survival free of any ventilator use at 52-week treatment (using Kaplan-Meier methodology)
- 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
- Motor development milestones [ Time Frame: at week 52 ]Number of motor development milestones achieved at Week 52 and change from baseline
- GESELL Development Scale [ Time Frame: at week 52 ]Change from baseline at Week 52 on GESELL Developmental Scale
- 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
- Cardiac failure [ Time Frame: at week 52 ]Proportion of patients with signs and/or symptoms of cardiac failure at Week 52
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
|Contact: Trial Transparency email recommended (Toll free number for US & Canada)||800-633-1610 ext 1 then #||Contact-US@sanofi.com|
|Investigational site number||Recruiting|
|Study Director:||Clinical Sciences & Operations||Sanofi|