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Gene Therapy Trial Report

Summary

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


NCTID NCT06272149 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Type II Gaucher Disease
Disease Ontology Term DOID:0110958
Compound Name VGN-R08b
Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Funder Type Other
Recruitment Status
Enrollment Count 6 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GBA1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intracerebroventricular
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose

Study Record Dates


Current Stage Early phase1
Submit Date 2023-07-17
Completion Date 2029-02-28
Last Update 2024-02-22

Participation Criteria


Eligible Age 0 Months - 24 Months
Standard Ages Child
Sexes Eligible for Study 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations Rare Pediatric Disease Designation
Recent Updates FIH interim data presented at ASGCT 2024

Resources/Links