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Tests treatment safety and results for Fabry Disease
Official title Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
ClinicalTrials.gov ID: NCT06539624
What this study is testing
What is EXG110 injection?
EXG110 injection is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fabry disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 and older
You may be able to join if
- At the time of signing the informed consent, age ≥7, 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 (adults only)
- All people of reproductive age voluntarily took effective contraception and prohibited sperm donation from entering the screening period until 52...
You likely can't join if
- Screening period laboratory test results: a) aspartate aminotransferase or alanine aminotransferase \> 1.5× upper limit of normal (ULN);b) Total...
- There was a clinically significant increase in AFP during the screening period
- Serum virology test: a) Hepatitis B: Hepatitis B virus surface antigen (HBsAg) positive, and hepatitis B virus-deoxyribonucleic acid (HBV-DNA) higher...
- AVT917 (\>1:50), anti-AGA antibody positive(\>1:2560)
- C3 lower than the normal range, C5b-9 higher than the normal range, anti-AVT917 IgM positive
- Current or have a history of serious cardiovascular disease and surgical history
See the full eligibility criteria
- At the time of signing the informed consent, age ≥7, 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 (adults only)
- All people of reproductive age voluntarily took effective contraception and prohibited sperm donation from entering the screening period until 52 weeks after dosing (main study period)
- The people voluntarily participate and are fully informed, fully understand the research, can comply with the requirements of the research protocol, and are willing to complete the research as planned, and voluntarily...
- 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 \>...
- There was a clinically significant increase in AFP during the screening period
- 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...
- AVT917 (\>1:50), anti-AGA antibody positive(\>1:2560)
- C3 lower than the normal range, C5b-9 higher than the normal range, anti-AVT917 IgM 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 drug
- Renal disease in adult and the slope of kidney \>5 mL/min/1.73m²/year
- people with poorly controlled diabetes after drug treatment (e.g., HbA1c≥8%);
- Acute/chronic infection or other chronic disease that the investigator determines will increase the risk of participants participating in the study
- Patients 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...
- Have malignancy cancer
- Patients 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
- Patients with a history of drug use or drug abuse or alcoholism
- Use of systemic (intravenous or oral) immunomodulators within the past 6 months or currently
- Initiation of treatment with blood pressure lowering drugs that affect proteinuria levels (such as angiotensin-converting enzyme inhibitors, angiotensin-receptor blockers, or angiotensin-receptor/enkephalin inhibitors)...
- Has received, or is 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 (or within 5...
- 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 felt needed to be ruled out
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
- Hangzhou, Zhejiang, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 7 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China; Hangzhou, Zhejiang, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.