Glycogen Storage Disease Type IIPhase 4APOLLO-IOPDNCT03687333
Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment
A Phase 4 study testing ALGLUCOSIDASE ALFA (MYOZYME) in people with Glycogen Storage Disease Type II, run by Genzyme, a Sanofi Company at 1 site in China.
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. Read more about this study
- Who is it for?
- Ages 0 to 12 months
- Conditions the study is for
- Glycogen Storage Disease Type II
- What is being tested?
- ALGLUCOSIDASE ALFA (MYOZYME)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 4 Dec 2018to 30 Dec 2020
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read more
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.
Read the 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
- Being tested
- ALGLUCOSIDASE ALFA (MYOZYME)
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALGLUCOSIDASE ALFA (MYOZYME)Drug
Pharmaceutical form: cake or powder for injection
Route of administration: intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Alglucosidase Alfa therapy | Experimental | Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks. Receives: Drug: ALGLUCOSIDASE ALFA (MYOZYME) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival
The proportion of patients alive at the end of study
Measured: at week 52
Left Ventricular Mass Index (LVMI)
Change from baseline in LVMI
Measured: at week 52
Secondary outcomes (6)
Invasive ventilation-free survival
Survival free of invasive ventilator use at 52-week treatment
Measured: at week 52
Any ventilation-free survival
Survival free of any ventilator use at 52-week treatment
Measured: at week 52
Growth in body weight and length
Physical growth: Change from baseline at Week 52 with regards to length and weight
Measured: at week 52
Motor development milestones
Number of motor development milestones achieved at Week 52 and change from baseline
Measured: at week 52
GESELL Development Scale
Change from baseline at Week 52 on GESELL Developmental Scale
Measured: at week 52
Cardiac failure
Proportion of patients with signs and/or symptoms of cardiac failure at Week 52
Measured: at week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2018
- Study start4 Dec 2018
- Primary completion30 Dec 2020
- Study completion30 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Glycogen Storage Disease Type IIStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03687333
- Sponsor's protocol ID
- ALGMYL08718
- Other IDs
- U1111-1203-8484
- Acronym
- APOLLO-IOPD
- Status last verified
- Apr 2022
- First submitted
- 24 Sep 2018
- Last updated
- 13 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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://vivli.org
Publications
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.
PubMed 35833019
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03687333
- Acronym / sponsor ID
- APOLLO-IOPD / ALGMYL08718
- Conditions
- Glycogen Storage Disease Type II
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 4 Dec 2018 (actual)
- Primary completion
- 30 Dec 2020 (actual)
- Study completion
- 30 Dec 2020 (actual)
- First posted
- 27 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Alglucosidase Alfa therapy | Experimental | Drug: ALGLUCOSIDASE ALFA (MYOZYME)Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks. |
Who may qualify
- Ages
- 0 to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (9 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALGLUCOSIDASE ALFA (MYOZYME)Pharmaceutical form: cake or powder for injection Route of administration: intravenous infusion | Drug | Alglucosidase Alfa therapy |
Primary outcome
Survival
Time frame: at week 52
The proportion of patients alive at the end of study
Left Ventricular Mass Index (LVMI)
Time frame: at week 52
Change from baseline in LVMI
6 secondary outcomes
Invasive ventilation-free survival
Time frame: at week 52
Any ventilation-free survival
Time frame: at week 52
Growth in body weight and length
Time frame: at week 52
Motor development milestones
Time frame: at week 52
GESELL Development Scale
Time frame: at week 52
Cardiac failure
Time frame: at week 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2022.
- Derived from this study: PubMed 35833019Zhu 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.
- Individual participant dataSharing planned: Yes. 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://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ALGLUCOSIDASE ALFA (MYOZYME) in people with Glycogen Storage Disease Type II, run by Genzyme, a Sanofi Company at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival (time frame: at week 52). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03687333, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2022.