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NFS-02-101 LHON (Leber Hereditary Optic Neuropathy) - Clinical Trial

What is the Purpose of this Study?

We are doing this study to find out if a new investigational gene therapy called NFS-02 (the study drug) is a safe and effective option for the treatment of Leber Hereditary Optic Neuropathy (LHON) that is associated with a mutation in the ND1 gene. The study will also look at how the immune system (the body's natural defense system) responds to the study drug, and we want to see what dose of the study drug seems to be the most appropriate for most people.

What is the Condition Being Studied?

Leber Hereditary Optic Neuropathy (LHON)

Who Can Participate in the Study?

Adults ages 18-75 who:

  • Are diagnosed with LHON and have lost vision within the past 6 months to 10 years
  • Have not had eye surgery in the past 3 months
  • Have not received any prior gene therapy

For more information about who can be in this study, please contact the study team at

Age Group

What is Involved?

If you choose to join the study, you will receive in one of your eyes one single injection of the study drug. The dose you receive will depend on when you join the study. The doses used in this study are:

  • 50,000,000 vg, 0.05 mL/eye (low dose)
  • 150,000,000 vg, 0.05 mL/eye (starting dose)
  • 500,000,000 vg, 0.05 mL/eye (intermediate dose)
  • 1,500,000,000 vg, 0.05 mL/eye (high dose)

At various visits, you will have your vision tested and eyes examined, answer questionnaires, and have blood tests performed. The study will last for up to 5 years and you will visit our clinic at the Duke Eye Center about 20 times.

Study Details

Full Title
A Phase 1/2, Multi regional Single Arm, Open Label, Dose Finding Clinical Trial to Evaluate the Safety Tolerability and Efficacy of Gene Therapy for Leber?s Hereditary Optic Neuropathy (LHON) Associated with ND1 Mutation - NFS-02-101
Principal Investigator
Protocol Number
IRB: PRO00114643
NCT: NCT05820152
Phase I/II
Enrollment Status
Open for Enrollment Soon