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The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease

ConditionFabry Disease
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorGuangzhou Jiayin Biotech Ltd

About this trial

A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • EXG110 Injection

    Drug

    EXG110 Injection is gene therapy for Fabry Disease , uses a proprietary AAV capsid with improved liver and muscle specificity.

Treatment groups

16 Participants
are divided into 2 treatment groups
Group A: Low dose armExperimental treatment 1 intervention
Group B: High dose armExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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,

Time frame
52 weeks
2

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.

Time frame
6 months
3

Phase 2: The changes from the baseline of cardiac function

The change of LVMI (left ventricular mass index，g/m\^2) from the baseline

Time frame
6 months
4

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.

Time frame
6 months

Secondary outcomes

1

eGFR

eGFR change from baseline in mL/min/(1.73m\^2)

Time frame
52 weeks
2

Cardiac function

LVMI change from baseline

Time frame
52 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Guangzhou Jiayin Biotech Ltd

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: