IT and IV Lentiviral Gene Therapy for X-ALD

NCT03727555 | Recruiting

• 1 other identifier: GIMI-IRB-18006
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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 correct the defective gene. The objectives are to evaluate the safety and efficacy of the intrathecal and intravenous lentiviral gene transfer clinical protocol.

Conditions

X-linked Adrenoleukodystrophy

Keywords

X-linked adrenoleukodystrophy; Lentiviral vector; Intrathecal injection; Intravenous injection

Outcome Measures

Primary Outcomes (3)

• Safety evaluation of IT and IV injections of lentiviral TYF-ABCD1, determined by number of participants with treatment-related adverse events (AEs), according to scheduled assessments, vital signs, & physical examinations as assessed by CTCAE v4.0.

  Safety of direct IT and IV injections of lentiviral TYF-ABCD1, determined by number of participants with treatment-related adverse events (AEs), according to scheduled assessments, vital signs, \& physical examinations as assessed by CTCAE v4.0. AEs \& clinically significant abnormalities (meeting grade 3, 4, or 5 criteria according to CTCAE) will be summarized by maximum intensity \& relationship to study drug(s). Grade 1 \& 2 AEs will be summarized if related to study therapy.

  Minimum 1 day, maximum 1 year follow up

• Altered disease progression

  Altered disease progression based on biochemicaland neurological analysis.

  Minimum 6 months, maximum 3 year follow up

• Assess disease progression

  Assess disease progression based on MRI brain imaging analysis.

  Minimum 6 months, maximum 3 year follow up

Study Arms (1)

Lentivirus-mediated delivery of ABCD1 to the CNS and the body

EXPERIMENTAL

Intrathecal and intravenous injections with lentiviral TYF-ABCD1 vector carrying the functional gene

Genetic: Intrathecal and intravenous LV gene therapy

Interventions

Intrathecal and intravenous LV gene therapy GENETIC

Direct IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\^9 multiplicity of infection/ml which carry normal ABCD1 gene

Lentivirus-mediated delivery of ABCD1 to the CNS and the body

Eligibility Criteria

• Age: 1 Year - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• X-ALD patients ≥ 1 year of age
• ALD diagnosis of the brain: evaluation of the VLCFA value in plasma
• Central imaging of the MRI to examine the damage on the CNS.
• Neurological function score (NFS) ≥ 1
• Parent / guardian / patient signing informed consent
• 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

You may not qualify if:

• HIV positive patients
• Stablized condition after statins, Lorenzo's oil, or diet to reduce VLCFA levels
• Patients who are experiencing severe viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
• Cannot perform an MRI
• Infection or dermatosis at pre-injection site

Sponsors & Collaborators

• Shenzhen Geno-Immune Medical Institute: lead

Study Sites (1)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, 518000, China

RECRUITING

MeSH Terms

Conditions

Adrenoleukodystrophy

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Central Study Contacts

Lung-Ji Chang, Ph.D

CONTACT

86-13671121909; c@szgimi.org

Rui Zhang, MSc

CONTACT

86-13488887093; 13488887093@163.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 25, 2018
• First Posted: November 1, 2018
• Study Start: August 31, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2028
• Last Updated: September 9, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)