Pompe Disease (Late-onset)Early Phase 1NCT06178432
Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
A Early Phase 1 study testing CRG003 injection in people with Pompe Disease (Late-onset), run by Huashan Hospital at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease (Late-onset)
- What is being tested?
- CRG003 injection
- 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
- Dec 2023to Dec 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Huashan Hospital of Fudan University
Shanghai
Wenhua Zhu, MD,PhD, +8621-52889999 ext. 8160, zhuwenhua@huashan.org.cn
About this study
Official title: 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
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.
- Conditions
- Pompe Disease (Late-onset)
- Being tested
- CRG003 injection
- Keywords
- LOPD
- 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.
CRG003 injectionGenetic
Also called BBM-G102 injection
The dose of CRG003 will be calculated according to the participant's weight with single intravenous infusion.
| Study group | Role | What they receive |
|---|---|---|
| Arm of CRG003 injection | Experimental | The dose of CRG003 injection will be calculated according to the participant's weight with single intravenous infusion. Receives: Genetic: CRG003 injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose limited toxicities
Incidence of dose limited toxicities (DLTs) as determined by the safety review committee (SRC) within 12 weeks following CRG003 infusion;
Measured: 12 weeks
Incidence of adverse events and serious adverse events
Incidence of adverse events (AEs) and serious adverse events (SAEs) within 26 weeks and 52 weeks following CRG003 infusion
Measured: 26 weeks and 52 weeks
The change of hepatic enzyme concentration
Alkaline phosphatase (ALP)
Measured: 26 weeks and 52 weeks
Changes from baseline in liver function
Changes from baseline in liver function [Alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transpeptidase (GGT) and total bilirubin (TBIL)] within 26 weeks and 52 weeks following CRG003 infusion
Measured: 26 weeks 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 posted21 Dec 2023
- Study startDec 2023 (estimated)
- Primary completionDec 2024 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe Disease (Late-onset)Studies of the same condition, open and under way.
- More trials run by Huashan HospitalThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Huashan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chongbo Zhao, Huashan Hospital
- Investigators
- Chongbo Zhao, MD,PhD, Principal investigator, Huashan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06178432
- Sponsor's protocol ID
- KY2023-783
- Status last verified
- Dec 2023
- First submitted
- 30 Nov 2023
- Last updated
- 21 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06178432
- Acronym / sponsor ID
- KY2023-783
- Conditions
- Pompe Disease (Late-onset)
- Keywords
- LOPD
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Huashan HospitalUniversity, hospital or other
- Study start
- Dec 2023 (estimated)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 21 Dec 2023 (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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm of CRG003 injection | Experimental | Genetic: CRG003 injectionThe dose of CRG003 injection will be calculated according to the participant's weight with single intravenous infusion. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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;
Key exclusion criteria (14 listed)
- 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;
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 |
|---|---|---|
| CRG003 injectionAlso: BBM-G102 injectionThe dose of CRG003 will be calculated according to the participant's weight with single intravenous infusion. | Genetic | Arm of CRG003 injection |
Primary outcome
Incidence of dose limited toxicities
Time frame: 12 weeks
Incidence of dose limited toxicities (DLTs) as determined by the safety review committee (SRC) within 12 weeks following CRG003 infusion;
Incidence of adverse events and serious adverse events
Time frame: 26 weeks and 52 weeks
Incidence of adverse events (AEs) and serious adverse events (SAEs) within 26 weeks and 52 weeks following CRG003 infusion
The change of hepatic enzyme concentration
Time frame: 26 weeks and 52 weeks
Alkaline phosphatase (ALP)
Changes from baseline in liver function
Time frame: 26 weeks and 52 weeks
Changes from baseline in liver function [Alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transpeptidase (GGT) and total bilirubin (TBIL)] within 26 weeks and 52 weeks following CRG003 infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2023.
- Individual participant dataSharing planned: No
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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.
Chongbo Zhao, MD,PhD principal investigator
Huashan Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CRG003 injection in people with Pompe Disease (Late-onset), run by Huashan 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 11 inclusion and 14 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: opening soon, as of the sponsor's last update on 21 Dec 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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wenhua Zhu, MD,PhD (+8621-52889999 ext. 8160, zhuwenhua@huashan.org.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose limited toxicities (time frame: 12 weeks). The study lists 4 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 21 Dec 2023.