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New treatment option for X-linked Adrenoleukodystrophy
Official title IT and IV Lentiviral Gene Therapy for X-ALD
ClinicalTrials.gov ID: NCT03727555
What this study is testing
What is Intrathecal and intravenous LV gene therapy?
Intrathecal and intravenous LV gene therapy is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for x-linked adrenoleukodystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 60
You may be able to join 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
You likely can't join 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...
- Cannot perform an MRI
- Infection or dermatosis at pre-injection site
See the full eligibility criteria
- 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Shenzhen, Guangdong, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shenzhen, Guangdong, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.