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IT and IV Lentiviral Gene Therapy for X-ALD

ConditionX-linked Adrenoleukodystrophy
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age1-60
SponsorShenzhen Geno-Immune Medical Institute

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • 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

Treatment groups

30 Participants
are divided into 1 treatment group
Group A: Lentivirus-mediated delivery of ABCD1 to the CNS and the bodyExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame
Minimum 1 day, maximum 1 year follow up
2

Altered disease progression

Altered disease progression based on biochemicaland neurological analysis.

Time frame
Minimum 6 months, maximum 3 year follow up
3

Assess disease progression

Assess disease progression based on MRI brain imaging analysis.

Time frame
Minimum 6 months, maximum 3 year follow up

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shenzhen Geno-Immune Medical Institute

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: