Infantile-onset Pompe DiseaseNCT05567627
Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease
A study testing Genetic: GC301 in people with Infantile-onset Pompe Disease, run by Seventh Medical Center of PLA General Hospital at 1 site in China.
Trial summary
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Read more about this study
- Who is it for?
- Ages Up to 6 months
- Conditions the study is for
- Infantile-onset Pompe Disease
- What is being tested?
- Genetic: GC301
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2022to Sep 2025
- Phase
- Interventional
- 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 Up to 6 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed;
- The patient must be no older than 6 months;
- The patient must be diagnosed with infantile-onset Pompe disease.
Who cannot join
- Class IV patient based on Modified Ross Heart Failure Classification for Children;
- Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 3x upper limit of normal (ULN), alkaline phosphatase (ALP) > 2x ULN (with the exception of liver abnormalities related to Pompe disease);
- Patient has severe organ dysfunction, such as liver and kidney failure (Liver failure: patients may have liver failure syndrome, including fatigue, severe gastrointestinal symptoms; clinical examination found prolonged prothrombin time, prothrombin activity less than 40%; Neuropsychiatric symptoms, such as restlessness, changes in personality and behavior, lethargy, coma, etc.; Toxic tympanic bowel, ascites, multiple organ dysfunction, etc.; hyperalbuminemia exceeding 171 μmol/L, hypoalbuminemia. Renal failure: creatinine exceeding 110 μmol/L, or glomerular filtration rate less than 100 mL/min), congenital/acquired encephalopathy, etc.;
- Patient with congenital organ absence;
- Patient with primary immunodeficiency;
- Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
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- Patient with a history of glucocorticoid allergy；
- Patient who has participated in a previous gene therapy research trial;
- Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Bayi Children's Hospital, Seventh Medical Center, PLA general hospital
Beijing, Beijing Municipality, 100700
Zhichun Feng, Prof, +86(10) 66721786, zhichunfeng81@163.com
Xiaodong Wang, PhD, +86 17801060980, donnawang@bj-genecradle.com
About this study
Official title: Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
- Conditions
- Infantile-onset Pompe Disease
- Being tested
- Genetic: GC301
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genetic: GC301Biological
GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene
| Study group | Role | What they receive |
|---|---|---|
| Initial dose cohort | Experimental | 1.2x10^14 vg/kg of GC301 administered via intravenous infusion Receives: Biological: Genetic: GC301 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability over time
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Measured: Infusion to the end of study, average 1 year
Secondary outcomes (3)
Proportion of patients treated w/ GC301 who were alive and free of ventilator support at 12 months of age;
Measured: 52 weeks
Changes from baseline Left Ventricular Mass (LVM)
Measured: 26 and 52 weeks
Changes from baseline creatine kinase (CK)
Measured: 26 and 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2022
- Study start1 Aug 2022
- Primary completionSep 2024 (estimated)
- Study completionSep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile-onset Pompe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Seventh Medical Center of PLA General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- GeneCradle Therapeutics, Inc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05567627
- Sponsor's protocol ID
- JLJY-GC301-IOPD-003
- Status last verified
- Oct 2023
- First submitted
- 25 Sep 2022
- Last updated
- 1 Nov 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease
- ClinicalTrials.gov ID
- NCT05567627
- Acronym / sponsor ID
- JLJY-GC301-IOPD-003
- Conditions
- Infantile-onset Pompe Disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Seventh Medical Center of PLA General HospitalUniversity, hospital or other
- Collaborators
- GeneCradle Therapeutics, Inc
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- Sep 2024 (estimated)
- Study completion
- Sep 2025 (estimated)
- First posted
- 5 Oct 2022 (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.
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Initial dose cohort | Experimental | Biological: Genetic: GC3011.2x10^14 vg/kg of GC301 administered via intravenous infusion |
Who may qualify
- Ages
- Up to 6 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed;
- The patient must be no older than 6 months;
- The patient must be diagnosed with infantile-onset Pompe disease.
Key exclusion criteria (9 listed)
- Class IV patient based on Modified Ross Heart Failure Classification for Children;
- Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 3x upper limit of normal (ULN), alkaline phosphatase (ALP) > 2x ULN (with the exception of liver abnormalities related to Pompe disease);
- Patient has severe organ dysfunction, such as liver and kidney failure (Liver failure: patients may have liver failure syndrome, including fatigue, severe gastrointestinal symptoms; clinical examination found prolonged prothrombin time, prothrombin activity less than 40%; Neuropsychiatric symptoms, such as restlessness, changes in personality and behavior, lethargy, coma, etc.; Toxic tympanic bowel, ascites, multiple organ dysfunction, etc.; hyperalbuminemia exceeding 171 μmol/L, hypoalbuminemia. Renal failure: creatinine exceeding 110 μmol/L, or glomerular filtration rate less than 100 mL/min), congenital/acquired encephalopathy, etc.;
- Patient with congenital organ absence;
- Patient with primary immunodeficiency;
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 |
|---|---|---|
| Genetic: GC301GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene | Biological | Initial dose cohort |
Primary outcome
Safety and tolerability over time
Time frame: Infusion to the end of study, average 1 year
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
3 secondary outcomes
Proportion of patients treated w/ GC301 who were alive and free of ventilator support at 12 months of age;
Time frame: 52 weeks
Changes from baseline Left Ventricular Mass (LVM)
Time frame: 26 and 52 weeks
Changes from baseline creatine kinase (CK)
Time frame: 26 and 52 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2023.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Genetic: GC301 in people with Infantile-onset Pompe Disease, run by Seventh Medical Center of PLA General Hospital 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 3 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: status not confirmed, as of the sponsor's last update on 1 Nov 2023. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Zhichun Feng (+86(10) 66721786, zhichunfeng81@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability over time (time frame: Infusion to the end of study, average 1 year). 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 1 Nov 2023.