Gene therapy for X-linked adrenoleukodystrophy
Intrathecal and Intravenous Lentiviral Gene Therapy for X-linked Adrenoleukodystrophy (X-ALD)
This study is testing a new gene therapy for X-linked adrenoleukodystrophy to see if it can help improve symptoms and slow down the disease in patients with different stages of the condition.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 1 Year to 60 Years |
| Sex | All |
| Sponsor | Shenzhen Geno-Immune Medical Institute Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 1 site (Shenzhen, Guangdong) |
| Trial ID | NCT03727555 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and efficacy of a novel gene therapy approach for treating X-linked adrenoleukodystrophy (X-ALD) using a self-inactivating lentiviral vector to correct the defective ABCD1 gene. The therapy involves both intrathecal and intravenous injections to directly address the genetic defect associated with this severe neurological disorder. The study aims to include patients with varying stages of the disease, including those who already exhibit symptoms, thereby expanding treatment opportunities beyond traditional hematopoietic stem cell transplantation. The trial will assess the impact of this innovative approach on neurological function and disease progression.
Who should consider this trial
Good fit: Ideal candidates for this study are X-ALD patients aged 1 year and older who have a confirmed diagnosis and are willing to participate in the trial.
Not a fit: Patients who are HIV positive, have stabilized their condition through other treatments, or have severe infections or other significant health issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a new treatment option for patients with X-ALD, potentially improving their neurological function and quality of life.
How similar studies have performed: While gene therapy approaches for genetic disorders are being explored, this specific application for X-ALD using a lentiviral vector is relatively novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Shenzhen, Guangdong
- Shenzhen Geno-immune Medical Institute — Shenzhen, Guangdong, China (Recruiting)
Study contacts
- Study coordinator: Lung-Ji Chang, Ph.D
- Email: c@szgimi.org
- Phone: 86-13671121909
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.