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Bascom Palmer Eye Institute

Frequently Asked Questions about the LHON Study

Why is this study being done?

The purpose of this study is to identify and follow a large number of LHON patients and their family members (who have not lost vision in either eye) in a normal clinic setting over a period of 4 years. With a large number of subjects, we hope to evaluate the long-term course of visual function in patients with LHON and to develop a course of treatment of visual function. The intent is to eventually choose subjects for the future phase 1 or phase 2 of the gene therapy study from the participants in this preparatory phase of the study.

How many subjects will take part in the study?

Approximately 50 subjects will participate in this study. While you take part in this study, you may not take part in any other research studies with an investigational drug, device, or procedure.

Are there any fees that I need to pay if I have no insurance?

The study will pay all expenses related to the study whether you are an LHON patient or a family member to an LHON patient.

Are there are any fees I need to pay if I have insurance?

If you are a patient with LHON and have insurance, your insurance will be billed for examination and testing that are considered standard of care, and the study will pay for examination and testing that are not considered standard of care. For the Initial visit, the cost for Genetic testing will be billed to your insurance provider. For the initial visit, 12 month visit, 24 month visit, 36 month visit, and 48 month visit, the cost for a routine eye examination, visual field testing, optic nerve photography, optical coherence tomography, will be billed to your insurance provider. You will be responsible for your own co-pay/deductible payments. For the 6 month visit, 18 month visit, 30 month visit, and 42 month visit, the study will pay all expenses involved for clinical examination and testing. The study will pay for the pattern electroretinogram for all visits. If your insurance refuses to pay for the clinical examination, genetic testing, and clinical testing billed as mentioned, the study will pay for the examinations and the testing refused by your insurance.

If you are a family member of a LHON patient, the study will pay all expenses involved in the study including genetic testing, clinical examination, and clinical testing. Your insurance will not be billed. Should you develop LHON, your insurance will be billed for examination and testing that are considered standard of care, and the study will pay for examination and testing that are not considered standard of care.

Who covers for traveling expenses?

You will not be paid for your participation in this study. You will not be reimbursed for parking and travel expenses.

Will I benefit from this study?

By studying a large number of individuals with LHON, we expect to learn more about the changes in vision and degeneration of the retina and optic nerve in order to treat this rare eye disease over time.

You will not receive any direct medical benefit from participating in this particular study, but an indirect benefit is that the National Institute of Health grant supporting this study is designed to bring gene replacement therapy out of the laboratory to patients with Lebers hereditary optic neuropathy caused by the most common genetic change (G11778A LHON mutation) that causes LHON. Patients screened as part of this study may be asked to be subjects in a future research study of this gene therapy. This may include you or your family members who have the genetic mutation. Even though they do not have visual loss, 50% of the males and 10% of the females will develop visual loss and could be potentially protected by gene replacement in a future clinical trial; so there may be direct benefit for the family member.

Will I be receiving any treatment for my condition during the study?

There is no treatment of any kind in this preparatory phase of the study, but Patients screened as part of this study may be asked to be subjects in a future research study of this gene therapy. This may include you or your family members who have the genetic mutation.

How often do I need to come during the year?

The study will last for four years, and you need to come two times a year (every 6 monhts).

How much time will I need for my appointment?

It will take all day, usually the visit start at 8 am and lasts until 5 pm.

Where should I go the day of my appointment?

Bascom Palmer Eye Institute
900 NW 17th Street – Fourth Floor
Miami, Florida 33136

If I need to reschedule, who could I contact?

Alexis Morante
Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine
Phone 305-202-4731

Which of my family members would be good candidates to participate in the study?

Maternally-related family members who do not have any symptoms of LHON, because this condition is maternally inherited.

When will I know if I qualify for this study?

One month after the Initial Visit.

What is the total length of the study?

The total length of your participation in the study could last up to 4 years.

What are the risks of this study?

The risks of blood drawing are minimal. They include fainting, pain and/or bruise at the site of puncture. Rarely, infection or the formation of a small clot or swelling of the vein and surrounding area may occur.

Your near vision will be blurred for 4-6 hours after having your eyes dilated, and you may be sensitive to bright light during this time as well. You should have someone accompany you to your appointment and you may want to wear sunglasses to reduce any discomfort to bright light.

Will I be paid for participating?

You will not be paid for your participation in this study. You will not be reimbursed for parking and travel expenses.

Can I drop out of the study at any time?

Your participation in this study is voluntary. You may decide not to participate or you may leave the study at any time. Your decision will not result in any penalty or loss of benefits to which you are entitled. The study doctor or sponsor may also remove you from the study at any time without your consent if he/she feels it is in the best interest for you medically, or for administrative reasons.

Who can I contact if I have any other questions?

Study doctors will be happy to answer any questions that you may have regarding this study plan. You may call Byron L. Lam M.D. 305-326-6021 or Alexis Morante, study coordinator, at 305-202-4731 to have any questions regarding this study answered.

Who will provide funding?

Funding for this research study is provided by the National Eye Institute (NEI).