ClinicalTrials.gov record NCT06178432
Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
A Single-arm, Open-label, Single-dose Study to Evaluate the Safety, Tolerability, and Efficacy of CRG003 Injection in the Treatment of Late Onset Pompe Disease
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This open-label, single-arm study gives one weight-based intravenous dose of CRG003 (BBM-G102) to adults with late-onset Pompe disease and follows participants long-term. The study focuses on safety and tolerability, including monitoring liver function and adverse events through the first year, with follow-up planned for five years. There is no placebo or blinded comparison.
What it is studying. The study is testing whether a single IV, weight-based dose of the gene therapy CRG003 can be given safely and tolerated in adults with late-onset Pompe disease, and it will measure dose-limiting toxicities, adverse events, and changes in liver enzymes and liver function over time. It will also collect data relevant to the therapy's efficacy in this population.
What the researchers hope to learn. To evaluate safety and tolerability (including incidence of dose-limiting toxicities and adverse events) and to assess changes in liver enzymes and liver function after a single IV dose of CRG003; to gather data on the therapy's efficacy in adults with late-onset Pompe disease.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term follow-up period of 5 years. CRG003 (BBM-G102) injection is an adeno-associated virus (AAV) gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Genetic
- CRG003 injection
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Participants voluntarily sign informed consent form;
- Clinically diagnosed with LOPD;
- Males or females aged ≥ 18 years;
- Undergone enzyme replacement treatment (ERT) with recombinant human acid alpha-glucosidase (rhGAA) previously, and has been discontinued for at least four weeks before screening;
- Acceptable Pulmonary test results;
- A 6MWT ≥ 100 meters, and ambulation for 40 meters without stopping and without an assistive device;
- Acceptable laboratory values;
- Acceptable GAA anti-drug antibody titer;
- Acceptable capsid antibody titers;
- Use of reliable contraception methods during the study;
- Participants with good compliance.
Exclusion criteria
- Severe cardiomyopathy was defined as left ventricular ejection fraction (LVEF) < 45% or New York Heart Association (NYHA) functional class 3 or above;
- Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
- Intolerance to ERT, prior experience of serious infusion-associated reactions (IARs), prior experience of serious allergic reactions or investigator-assessed intolerance to ERT;
- Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids or investigator-recommended immunosuppressants 30 days prior to screening, and known intolerance to immunosuppressants such as glucocorticoids;
- Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA). Participants with a history of hepatitis B or C can be regarded as negative if both two samples collected at an interval of at least three months are tested negative for the above parameters; positive for human immunodeficiency virus (HIV) or positive serologic test for syphilis;
- Currently on antiviral therapy for hepatitis B or C;
- Have clinical organic diseases (except symptoms or diseases associated with Pompe disease), including active tuberculosis, cardiovascular and cerebrovascular diseases, hepatobiliary system, respiratory system, nervous system, urinary system, or endocrine system disorders (such as diabetes, etc.), or other serious complications, or other conditions that make the patients not eligible for the study according to the investigator;
- Have underlying liver diseases, e.g., prior diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, severe fatty liver, cirrhosis or liver fibrosis ≥stage 3; or ultrasound-identified liver neoplasms or laboratory tests suggesting elevated alpha-fetoprotein, etc., which are considered by the investigator as clinically significant;
- Have received gene therapy prior to screening or used other investigational drugs or drugs that affect this study as evaluated by the investigator within four weeks prior to screening or within 5 half lives of the investigational drug (whichever is longer);
- Have received or will receive any herbal preparations (herbal supplements or traditional Chinese medicines derived from plants, minerals, or animals, other than topical medications) that may affect liver function or Chinese herbal medicines that may affect the study as judged by the investigator four weeks prior to study medication or during the study follow-up period;
- Alcohol dependence or drug addiction, and inability to stop alcohol intake as ordered by the doctor during the study;
- Have received any live vaccine two months predose or history of vaccination within 30 days prior to screening or planning to receive vaccination during the screening and the main study period;
- Pregnant or lactating female participants;
- Other conditions that make the participants not eligible for the study according to the investigator.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive a single intravenous infusion of the investigational gene therapy CRG003; the dose is calculated by weight. They will have regular visits and safety monitoring, including checks for adverse events and liver function over the first year (with specific safety assessments at 12, 26, and 52 weeks) and then long‑term follow‑up for up to 5 years. Baseline assessments include pulmonary testing and a 6‑minute walk test. Contraception, avoidance of certain vaccines, and restrictions on some medications or herbal products are required as part of study rules.
How the study is structured. Participants receive one intravenous infusion of CRG003 in a single treatment group (no randomization or masking). They will have scheduled safety checks, blood tests (including liver tests), and other assessments during the first year and continued follow-up visits for a total of five years.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Pompe disease
Important dates
- Study start
- December 2023 (estimated)
- Primary completion
- December 2024 (estimated)
- Study completion
- December 2028 (estimated)
- Target enrollment
- 6 participants (estimated)
- Last updated on registry
- Dec 21, 2023
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Huashan Hospital
- Principal investigator
- Chongbo Zhao, MD, PhD
Huashan Hospital
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Wenhua Zhu, MD,PhD 📞 +8621-52889999 ext. 8160 ✉ zhuwenhua@huashan.org.cn
Participating sites (1)
- Huashan Hospital of Fudan University
Shanghai · China
- Wenhua Zhu, MD,PhD · +8621-52889999 ext. 8160 · zhuwenhua@huashan.org.cn
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- long-term follow-up
- Observation that continues for years after the study treatment, often required for gene and cell therapies.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- 6MWT
- A test of how far a person can walk on a flat surface in six minutes. It is used to measure stamina and to track change over time.
- immunosuppressants
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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