IT and IV Lentiviral Gene Therapy for X-ALD

Launched by SHENZHEN GENO-IMMUNE MEDICAL INSTITUTE · Oct 31, 2018

Keywords

ClinConnect Summary

This clinical trial is testing a new gene therapy for a rare condition called X-linked adrenoleukodystrophy (X-ALD), which affects the brain and nervous system. The therapy uses a special tool (called a lentiviral vector) to try to fix the gene that doesn’t work properly in patients with this condition. The main goals of the trial are to see if this treatment is safe and if it can help improve the health of the patients. The trial is currently looking for participants, including children and adults who are at least 1 year old and have been diagnosed with X-ALD.

To be eligible for the trial, participants need to have certain test results that show they have X-ALD and some level of neurological impairment. They also need to agree to participate and understand the potential risks involved. During the trial, participants will receive the gene therapy through injections, and their health will be closely monitored to check for any side effects and to see how well the treatment is working. It’s important for families to know that this is an experimental treatment, and while it offers hope, it also comes with uncertainties.

Gender

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