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Gene Therapy Trial Report

Summary

IT and IV Lentiviral Gene Therapy for X-ALD


NCTID NCT03727555 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication X-linked Adrenoleukodystrophy
Disease Ontology Term 🔄 DOID:10588
Compound Name 🔄 (LV) TYF-ABCD1
Sponsor Shenzhen Geno-Immune Medical Institute
Funder Type Other
Recruitment Status
Enrollment Count 30 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 ABCD1
Therapy Type 🔄 Gene transfer
Therapy Route 🔄 In-vivo
Mechanism of Action 🔄 Functional gene replacement
Route of Administration 🔄 Intrathecal, intravenous
Drug Product Type 🔄 Viral vector
Delivery System 🔄 Viral transduction
Vector Type 🔄 LV
Dose 1 🔄 1-2E9 MOI/ml

Study Record Dates


Current Stage Na
Submit Date 2018-09-25
Completion Date 2028-12-31
Last Update 2025-09-09

Participation Criteria


Eligible Age 1 Year - 60 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. X-ALD patients ≥ 1 year of age 2. ALD diagnosis of the brain: evaluation of the VLCFA value in plasma 3. Central imaging of the MRI to examine the damage on the CNS. 4. Neurological function score (NFS) ≥ 1 5. Parent / guardian / patient signing informed consent 6. Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the failure of the trial, and sign an informed consent form Exclusion Criteria: 1. HIV positive patients 2. Stablized condition after statins, Lorenzo's oil, or diet to reduce VLCFA levels 3. Patients who are experiencing severe viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency 4. Cannot perform an MRI 5. Infection or dermatosis at pre-injection site
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links