Apply to trial NCT03727555

A few quick questions so the study team can decide if you might be a fit.

RecruitingGenetic intervention

IT and IV Lentiviral Gene Therapy for X-ALD

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.

How this works

Answer a few questions
About 5 to 10 minutes. Skip-friendly where possible.
We connect you with the study team
When a site picks up your application, we introduce you to their team directly — with you on the thread.
The team reaches out to schedule screening
Usually within a few business days, via the contact you give.

EmailWe save your progress under this email and copy you when we connect you with the study team.

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IT and IV Lentiviral Gene Therapy for X-ALD

How this works

Answer a few questions

We connect you with the study team

The team reaches out to schedule screening