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X-linked Retinoschisis (XLRS) - Healing Genes
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X-linked Retinoschisis (XLRS)

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Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis

A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis


Phase 1 / 2

DESCRIPTION:

Doctors at the National Eye Institute (NEI) are recruiting participants with X-linked Retinoschisis to help assess the effectiveness of an investigational gene therapy to improve retina structure and function. The mutation targeted by this treatment is the RS1 gene which produces abnormal retinoschisin and leads to vision loss. The treatment, a genetically modified virus, is designed to impart the functional copy of the RS1 gene to cells of the eyes and restore normal retinoschisin production.

Participants will be assessed during 2 visits and then the investigational drug will be injected into one or both eyes at one of 3 doses. Patients will be followed closely for 18 months, then approximately once per year until 5 years post treatment, then participants will be contacted yearly by phone for up to 15 years..


PATIENT MUST:

  • Be 18 years of age or older
  • Have vision loss of a certain severity due to XLRS in at least one eye
  • Not have undergone intraocular surgery within six months prior to enrollment
  • Must agree to use of contraception for the period of one year post the treatment.

THE STUDY INVOLVES:

  1. Prescreening to confirm eligibility of the patient to participate
  2. A single intravitreal injection into one or both eyes
  3. Follow up continues closely for 18 months, then approximately once per year until 5 years post treatment, then participants will be contacted yearly by phone for up to 15 years.

LOCATIONS AND CONTACTS:

Contacts:
Amy E Turriff  |  (301) 402-4175  |  [email protected]
Paul A Sieving, M.D.  |  (301) 496-2234  |  [email protected]

 

LOCATIONS AND CONTACTS:
Trials will take place at the National Institutes of Health Clinical Center in Bethesda, MD. Map.

 

SPONSOR INFORMATION:
National Eye Institute (NEI)

 

Or go online:
https://clinicaltrials.gov/ct2/show/NCT02317887

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