Status:

RECRUITING

IT and IV Lentiviral Gene Therapy for X-ALD

Lead Sponsor:

Shenzhen Geno-Immune Medical Institute

Conditions:

X-linked Adrenoleukodystrophy

Eligibility:

All Genders

1-60 years

Phase:

NA

Brief Summary

This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally ...

Detailed Description

X-linked adrenoleukodystrophy (X-ALD) is a devastating neurological disorder caused by mutations in the ABCD1 gene that encodes a peroxisomal ATP-binding cassette transporter (ABCD1). ABCD1 is respons...

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

Key Trial Info

Start Date :

August 31 2025

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 31 2028

Estimated Enrollment :

30 Patients enrolled

Trial Details

Trial ID

NCT03727555

Start Date

August 31 2025

End Date

December 31 2028

Last Update

September 9 2025

Active Locations (1)

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Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, China, 518000