An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Launched by XINHUA HOSPITAL, SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE · Feb 21, 2024

Keywords

ClinConnect Summary

This clinical trial is exploring a new treatment called VGN-R08b for infants with Type II Gaucher Disease, a rare genetic condition that affects the body's ability to break down certain fats. The main goal of the study is to see if this treatment is safe and well-tolerated in young children. The trial is currently recruiting participants who are infants up to 24 months old and have been diagnosed with this condition based on specific genetic tests. To participate, parents or guardians must give their consent and agree to help with the study requirements, such as sharing information about the child's symptoms.

Participants in the trial can expect to receive the new treatment and will be closely monitored for any side effects or reactions. It's important to note that some children may not qualify if they have other serious health issues or if they have recently received certain treatments that could interfere with the study. Overall, this trial aims to find a potential new option for treating infants with Type II Gaucher Disease, offering hope for better management of this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Infants with age of ≤24 months.
• • 2. Historical diagnosis of Gaucher disease confirmed by GCase enzyme activity test, and with GBA1 biallelic mutations.
• • 3. Neurological signs and/or symptoms consistent with diagnosis of GD2.
• • 4. Parent(s)/legal guardian(s) of subject must give their consent for subject to enroll in the study.
• • 5. Parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including providing disease information and support disease assessment of symptoms.
• Exclusion Criteria:
• • 1. Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound study objectives.
• • 2. Achieved independent gait.
• • 3. Severe visceral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• • 4. Clinically active infection (including HIV, HBV, HCV or syphilis).
• • 5. For those receiving enzyme replacement therapy and/or substrate reduction therapy and/or ambroxol for Gaucher disease, stable treatment ≤2 months before enrollment.
• • 6. Use of strong inhibitors or inducers of cytochrome CYP3A4 or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents.
• • 7. Any type of prior gene or cell therapy.
• • 8. Immunizations (live vaccines) in the prior 4 weeks.
• • 9. Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical preparations for dermatological conditions are allowed).
• • 10. Patients with anti-AAV9 neutralizing antibody titer over 1:5.
• • 11. Brain MRI (magnetic resonance imaging) showing clinically significant abnormality considered to prevent intracisternal injection.
• • 12. Contraindication to sedation during surgery or imaging studies (PET).
• • 13. Presence of other significant medical conditions that would create an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.