Fabry DiseasePhase 1/2NCT06819514
The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
A Phase 1/2 study testing EXG110 Injection in people with Fabry Disease, run by Guangzhou Jiayin Biotech Ltd at 3 sites in China.
Trial summary
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- EXG110 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 3 sitesChina
- Study dates
- 1 Mar 2025to 15 Mar 2028
- Phase
- Phase 1/2
- 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
- Age ≥18 years old, male or female
- Clinical symptoms (at least one Fabry disease related symptom) and genetic diagnosis of Fabry disease
- Prior or no prior ERT treatment
- Have renal or cardiac involvement
- The participant voluntarily participate and are fully informed, fully understood the study, can comply with the requirements of the protocol, and voluntarily provide biological samples for testing according to the requirements of the protocol
Who cannot join
- Screening period laboratory test results: a) aspartate aminotransferase or alanine aminotransferase > 1.5× upper limit of normal (ULN);b) Total bilirubin > 1.5× upper limit of normal (ULN);c) Alkaline phosphatase > 2× upper limit of normal (ULN);d) Hypoalbuminemia ≥ grade 2
- Serum virology test: a) Hepatitis B: Hepatitis B virus surface antigen (HBsAg) positive, and hepatitis B virus-deoxyribonucleic acid (HBV-DNA) higher than the upper limit of normal detection;b) Hepatitis C: if the hepatitis C virus (HCV) antibody is positive, and the hepatitis C virus-ribonucleic acid (HCV-RNA) is higher than the upper limit of normal test value;c) Syphilis: positive for syphilis screening (Tp-Ab) and positive for syphile-specific antibodies;d) HIV: Known human immunodeficiency virus (HIV) positive history or HIV screening positive
- Current or have a history of serious cardiovascular disease and surgical history
- Current underlying liver disease or history of liver disease, as assessed by the investigator, that may affect the safety assessment of the product
- Acute/chronic infection or other chronic disease that the investigator evaluated will increase the risk of participants in the study
- Those who have a history of epilepsy, mental illness (such as schizophrenia, depression, mania or anxiety, etc.) or obvious mental disorders, and are incapacitated or cognitively incapacitated due to other reasons.
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- Participant with a history of malignant tumor or currently suffering from any malignant tumor (except for the following tumor diseases: skin basal cell carcinoma, cervical carcinoma in situ, breast carcinoma in situ , skin squamous cell carcinoma has been controlled after treatment);
- Participant with active autoimmune diseases (such as rheumatoid arthritis, systemic lupus erythematosus, multiple sclerosis, immune vasculitis, inflammatory bowel disease, etc.);
- known history of allergy to the components of the investigational products
- Participant with a history of drug use or drug abuse or alcoholism
- Has received, or currently receiving, a clinical trial of another investigational drug/medical device or treatment (other than vitamins and minerals) within 3 months prior to signing the informed consent form (or within 5 half-lives of the investigational drug, whichever is longer)
- Previous treatment with gene therapy products
- Those who had received live attenuated vaccine/vaccine within 12 weeks prior to screening or planned to receive it during the study
- Other clinical conditions that the investigators evaluated needed to be excluded
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China3 sites
Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang
Jianhua Mao, PhD, +86 13616819071, maojh88@zju.edu.cn
Jianhua Mao, PhD
First Affiliated Hospital of Zhejiang University
Hangzhou, Zhejiang
Xiaogang Guo, PhD, +86 13867741856, michelleyan2000@126.com
Xiaogang Guo, PhD
General Hospital of Eastern Theater Command
Nanjing, Jiangsu
Peiling Chen, MD, +86 137 0158 9469, chenpl166@163.com
Zhihong Liu, MD
About this study
Official title: A Phase 1/2, Multicenter, Open-label,Dose-escalation Study to Evaluate the Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease
Read the detailed description
Phase 1: Dose -escalation，2 Groups Phase 2: Dose- expansion，1 Group
- Conditions
- Fabry Disease
- Being tested
- EXG110 Injection
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EXG110 InjectionDrug
EXG110 Injection is gene therapy for Fabry Disease , uses a proprietary AAV capsid with improved liver and muscle specificity.
| Study group | Role | What they receive |
|---|---|---|
| Low dose arm | Experimental | EXG110 injection, use once by intravenous Receives: Drug: EXG110 Injection |
| High dose arm | Experimental | EXG110 injection, use once by intravenous Receives: Drug: EXG110 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: To evaluate the safety and tolerability of EXG110 following a single intravenous infusion
Adverse events (AEs), serious adverse events (SAEs), dose-limited toxicity types, severity, incidence,
Measured: 52 weeks
Phase 2: The changes from the baseline of kidney function
Proportion of participants with a reduction in Gb3 inclusion body accumulation to scores 0 (the minimum values are 0 scores, the maximum values are 3, higher scores mean a worse outcome ) on renal interstitial capillary biopsy as determined by light microscopy.
Measured: 6 months
Phase 2: The changes from the baseline of cardiac function
The change of LVMI (left ventricular mass index，g/m^2) from the baseline
Measured: 6 months
Phase 2: The changes from the baseline of pain
The scores changes of BPI (The Brief Pain Inventory, short form) from the baseline, each iteam is scored on a 0-10 scale, with higher scores indicating greater severity.
Measured: 6 months
Phase 2: The changes from the baseline of gastrointestinal function
The scores changes of GSRS (Gastrointestinal Symptom Rating Scale) from the baseline the minimum values are 0 scores, the maximum values are 35, higher scores mean a worse outcome
Measured: 6 months
Secondary outcomes (2)
eGFR
eGFR change from baseline in mL/min/(1.73m^2)
Measured: 52 weeks
Cardiac function
LVMI change from baseline
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2025
- Study start1 Mar 2025 (estimated)
- Primary completion15 Mar 2028 (estimated)
- Study completion15 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 16 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangzhou Jiayin Biotech Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Zhihong Liu, MD, Principal investigator, The General Hospital of Eastern Theater Command
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06819514
- Sponsor's protocol ID
- EXG110-111
- Status last verified
- Feb 2025
- First submitted
- 22 Jan 2025
- Last updated
- 11 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: All data will be generated in this study are the property of Hangzhou Jiayin and should be kept strictly confidential together with the information provided by Hangzhou Jiayin. Investigator or any other delegated staff are not permitted to independently analyze and/or publish these data without the prior written authorization of Hangzhou Jiain.
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Multicenter, Open-label,Dose-escalation Study to Evaluate the Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT06819514
- Acronym / sponsor ID
- EXG110-111
- Conditions
- Fabry Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- Guangzhou Jiayin Biotech LtdIndustry (pharma, biotech, devices)
- Study start
- 1 Mar 2025 (estimated)
- Primary completion
- 15 Mar 2028 (estimated)
- Study completion
- 15 Mar 2028 (estimated)
- First posted
- 11 Feb 2025 (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.
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease
Detailed description
Phase 1: Dose -escalation，2 Groups Phase 2: Dose- expansion，1 Group
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Low dose arm | Experimental | Drug: EXG110 InjectionEXG110 injection, use once by intravenous |
| High dose arm | Experimental | Drug: EXG110 InjectionEXG110 injection, use once by intravenous |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥18 years old, male or female
- Clinical symptoms (at least one Fabry disease related symptom) and genetic diagnosis of Fabry disease
- Prior or no prior ERT treatment
- Have renal or cardiac involvement
- The participant voluntarily participate and are fully informed, fully understood the study, can comply with the requirements of the protocol, and voluntarily provide biological samples for testing according to the requirements of the protocol
Key exclusion criteria (14 listed)
- Screening period laboratory test results: a) aspartate aminotransferase or alanine aminotransferase > 1.5× upper limit of normal (ULN);b) Total bilirubin > 1.5× upper limit of normal (ULN);c) Alkaline phosphatase > 2× upper limit of normal (ULN);d) Hypoalbuminemia ≥ grade 2
- Serum virology test: a) Hepatitis B: Hepatitis B virus surface antigen (HBsAg) positive, and hepatitis B virus-deoxyribonucleic acid (HBV-DNA) higher than the upper limit of normal detection;b) Hepatitis C: if the hepatitis C virus (HCV) antibody is positive, and the hepatitis C virus-ribonucleic acid (HCV-RNA) is higher than the upper limit of normal test value;c) Syphilis: positive for syphilis screening (Tp-Ab) and positive for syphile-specific antibodies;d) HIV: Known human immunodeficiency virus (HIV) positive history or HIV screening positive
- Current or have a history of serious cardiovascular disease and surgical history
- Current underlying liver disease or history of liver disease, as assessed by the investigator, that may affect the safety assessment of the product
- Acute/chronic infection or other chronic disease that the investigator evaluated will increase the risk of participants in the study
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 |
|---|---|---|
| EXG110 InjectionEXG110 Injection is gene therapy for Fabry Disease , uses a proprietary AAV capsid with improved liver and muscle specificity. | Drug | High dose arm, Low dose arm |
Primary outcome
Phase 1: To evaluate the safety and tolerability of EXG110 following a single intravenous infusion
Time frame: 52 weeks
Adverse events (AEs), serious adverse events (SAEs), dose-limited toxicity types, severity, incidence,
Phase 2: The changes from the baseline of kidney function
Time frame: 6 months
Proportion of participants with a reduction in Gb3 inclusion body accumulation to scores 0 (the minimum values are 0 scores, the maximum values are 3, higher scores mean a worse outcome ) on renal interstitial capillary biopsy as determined by light microscopy.
Phase 2: The changes from the baseline of cardiac function
Time frame: 6 months
The change of LVMI (left ventricular mass index，g/m^2) from the baseline
Phase 2: The changes from the baseline of pain
Time frame: 6 months
The scores changes of BPI (The Brief Pain Inventory, short form) from the baseline, each iteam is scored on a 0-10 scale, with higher scores indicating greater severity.
Phase 2: The changes from the baseline of gastrointestinal function
Time frame: 6 months
The scores changes of GSRS (Gastrointestinal Symptom Rating Scale) from the baseline the minimum values are 0 scores, the maximum values are 35, higher scores mean a worse outcome
2 secondary outcomes
eGFR
Time frame: 52 weeks
Cardiac function
Time frame: 52 weeks
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 11 Feb 2025.
- Individual participant dataSharing planned: No. All data will be generated in this study are the property of Hangzhou Jiayin and should be kept strictly confidential together with the information provided by Hangzhou Jiayin. Investigator or any other delegated staff are not permitted to independently analyze and/or publish these data without the prior written authorization of Hangzhou Jiain.
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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.
Zhihong Liu, MD principal investigator
The General Hospital of Eastern Theater Command
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing EXG110 Injection in people with Fabry Disease, run by Guangzhou Jiayin Biotech Ltd at 3 sites 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 5 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 11 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 3 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Zhihong Liu, MD (+86 137 0158 9469, chenpl166@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 1: To evaluate the safety and tolerability of EXG110 following a single intravenous infusion (time frame: 52 weeks). The study lists 5 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 11 Feb 2025.