Pompe's DiseasePhase 4APOLLO-LOPDNCT04676373
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
A Phase 4 study testing ALGLUCOSIDASE ALFA in people with Pompe's Disease, run by Genzyme, a Sanofi Company at 1 site in China.
Trial summary
Primary Objective: To evaluate the effect of one-year Alglucosidase alfa treatment on motor function [Six-minute walk test (6MWT) and lung function predicted Forced vital capacity (FVC)] among Chinese Late Onset Pompe Disease patients above 5 years old. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Pompe's Disease
- What is being tested?
- ALGLUCOSIDASE ALFA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 1 siteChina
- Study dates
- 10 Mar 2021to 25 Jul 2024
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures
- The patient must be ≥ 3 years of age at the time of enrollment.
- For patient ≥ 3-year and < 5-year old: must be able to walk 10 meters or climb 4-step stairs independently.
- For patients ≥5-year old
- Must be able to ambulate 40 meters in 6 minutes without assistance ii. Must be able to successfully perform repeated forced vital capacity (VC) measurements in upright position of ≥ 30% predicted and ≤85% predicted.
- The patient has confirmed Pompe's Disease with at least 2 of the following condition,
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- GAA enzyme deficiency from any tissue source.
- 2 confirmed GAA gene mutations.
- muscle pathology meet the diagnosis of Pompe disease.
- The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
- The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline.
Who cannot join
- Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.)
- Use of non-invasive ventilatory support while awake and in an upright position. (Non-invasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube.)
- Previously treated with Enzyme Replacement Treatment.
- A Female patient of childbearing potential with a positive pregnancy test.
- Wheelchair dependent.
- The patient has a major congenital anomaly.
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- The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
- The patients with ≥5-year old are unable to ambulate 40 meters without assistance or unable to successfully perform repeated FVC of >30% and <85% predicted (upright).
- 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 Late Onset Pompe Disease With Alglucosidase Alfa Treatment
Primary Objective:
To evaluate the effect of one-year Alglucosidase alfa treatment on motor function [Six-minute walk test (6MWT) and lung function predicted Forced vital capacity (FVC)] among Chinese Late Onset Pompe Disease patients above 5 years old.
To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old.
Secondary Objective:
To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test (MMT), Maximal inspiratory and expiratory pressure (MIP and MEP)], Quick Motor Function Test scores, and health-related quality of life (SF-12) among LOPD patients over 5 years old.
Read the detailed description
Study duration per participants is approximatively 56 weeks including a 52-week treatment period.
- Conditions
- Pompe's Disease
- Being tested
- ALGLUCOSIDASE ALFA
- 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 ALFADrug
Also called MYOZYME
Pharmaceutical form:solution for infusion
Route of administration: intravenous
| Study group | Role | What they receive |
|---|---|---|
| Alglucosidase Alfa | Experimental | A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks Receives: Drug: ALGLUCOSIDASE ALFA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.
Measured: Baseline to 12 months
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Measured: Baseline to 12 months
Number of participants with adverse events Number of AEs
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Measured: From signing the ICF to the 30th day after the last dosage of the study medications
Secondary outcomes (5)
Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Measured: Week 52
Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Measured: Week 52
Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old
MMT has been reported most often as a summary score of a total number of proximal, distal, and axial muscle groups tested bilaterally or as a proximal score that sums a number of proximal muscle groups from the upper and lower extremities.
Measured: Week 52
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
The Quick Motor Function Test is a reliable and valid test for assessing motor function in patients with Pompe's disease.
Measured: Week 52
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
The 12-Item Short Form Health Survey (SF-12) was developed for the Medical Outcomes evaluation of patients with chronic conditions.
Measured: Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2020
- Study start10 Mar 2021
- Primary completion25 Jul 2024
- Study completion25 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe's DiseaseStudies 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
- 41 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
- NCT04676373
- Sponsor's protocol ID
- ALGMYL09010
- Other IDs
- U1111-1238-1267, LPS15677, 2024-000461-24
- Acronym
- APOLLO-LOPD
- Status last verified
- Feb 2025
- First submitted
- 15 Dec 2020
- Last updated
- 10 Feb 2025
- 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
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2025. 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 Late Onset Pompe Disease With Alglucosidase Alfa Treatment
- ClinicalTrials.gov ID
- NCT04676373
- Acronym / sponsor ID
- APOLLO-LOPD / ALGMYL09010
- Conditions
- Pompe's Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 10 Mar 2021 (actual)
- Primary completion
- 25 Jul 2024 (actual)
- Study completion
- 25 Jul 2024 (actual)
- First posted
- 21 Dec 2020 (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 the effect of one-year Alglucosidase alfa treatment on motor function [Six-minute walk test (6MWT) and lung function predicted Forced vital capacity (FVC)] among Chinese Late Onset Pompe Disease patients above 5 years old.
To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old.
Secondary Objective:
To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test (MMT), Maximal inspiratory and expiratory pressure (MIP and MEP)], Quick Motor Function Test scores, and health-related quality of life (SF-12) among LOPD patients over 5 years old.
Detailed description
Study duration per participants is approximatively 56 weeks including a 52-week treatment period.
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 | Experimental | Drug: ALGLUCOSIDASE ALFAA dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures
- The patient must be ≥ 3 years of age at the time of enrollment.
- The patient has confirmed Pompe's Disease with at least 2 of the following condition,
- The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
- The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline.
Key exclusion criteria (9 listed)
- Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.)
- Use of non-invasive ventilatory support while awake and in an upright position. (Non-invasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube.)
- Previously treated with Enzyme Replacement Treatment.
- A Female patient of childbearing potential with a positive pregnancy test.
- Wheelchair dependent.
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 ALFAAlso: MYOZYMEPharmaceutical form:solution for infusion Route of administration: intravenous | Drug | Alglucosidase Alfa |
Primary outcome
Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
Time frame: Baseline to 12 months
This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old
Time frame: Baseline to 12 months
The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Number of participants with adverse events Number of AEs
Time frame: From signing the ICF to the 30th day after the last dosage of the study medications
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
5 secondary outcomes
Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old
Time frame: Week 52
Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old
Time frame: Week 52
Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old
Time frame: Week 52
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
Time frame: Week 52
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
Time frame: 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 10 Feb 2025.
- ALGMYL09010 Plain language Results SummaryLink provided by the sponsor.
- 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 in people with Pompe's Disease, 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 11 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 10 Feb 2025. 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?
Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old (time frame: Baseline to 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Feb 2025.