Search / Trial NCT00001735

Gene Therapy for Gyrate Atrophy

Launched by NATIONAL EYE INSTITUTE (NEI) · Dec 9, 2002

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Trial Information

Current as of March 05, 2024

Completed

Keywords

Gene Therapy Graft Ornithine Retina Retinovirus Retrovirus Skin Gyrate Atrophy

Description

The primary purpose of this phase I study is to evaluate the safety of a skin engraftment procedure for transplanting ex-vivo transduced keratinocytes in patients with a deficiency of the ornithine transferase gene (OAT). The safety of this procedure will be evaluated in terms of technical complications and the immune response of the patient to the presence of the transduced keratinocytes. Adult patients with gyrate atrophy, a disease defined by a deficiency of OAT, who have been followed for an extended period of time at the National Eye Institute and whose natural history is known to the ...

Gender

All

Eligibility criteria

  • Elevated serum ornithine level greater than or equal to 200 micro M, as measured by the median of three values obtained monthly for two months prior to the screening visit and at the screening visit.
  • Absence of OAT activity measured by western blot analysis and isolated enzyme activity in skin biopsy samples.
  • Fundus findings indicative of gyrate atrophy as characterized by sharply demarcated circular patches of chorioretinal atrophy.
  • Must be at least 18 years of age.
  • Female patients of child bearing potential must have a pregnancy test done, which demonstrates a negative result and must agree to practice effective birth control for 12 months following study initiation, or until patch removal, whichever comes first.
  • Must sign and date the informed consent and is willing and able to follow study procedures.
  • Patients demonstrates progression of ocular disease due to grate atrophy evidenced by reduction in electrophysiologic testing, visual field testing or progression of retinal findings.
  • Patient has been offered an arginine-restricted diet and is unable to attain ornithine levels less than 200 micro M prior to patch placement.
  • Patient has previously undergone a skin biopsy.
  • Not currently participating in other experimental protocols or therapeutic trials.
  • Does not have a known malignancy or chronic infection yielding immunoincompetence.
  • Patient must be able to maintain follow-up and follow details of the protocol.

Attachments

readout_NCT00001735_2024-03-05.pdf

4.5 MB

NCT00001735_study_protocol.pdf

4.5 MB

About company

The National Institute of Allergy and Infectious Diseases (NIAID, /ˈnaɪ.æd/) is one of the 27 institutes and centers that make up the National Institutes of Health (NIH), an agency of the United States Department of Health and Human Services (HHS). NIAID's mission is to conduct basic and applied research to better understand, treat, and prevent infectious, immunologic, and allergic diseases.

Contacts

JC

Jennifer Cobb

Immunology at National Institute of Allergy and Infectious Diseases (NIAID)

Locations

Bethesda, Maryland, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Reviews (48)

4.6

All reviews come from applied patients

5 stars
41
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Leslie Alexander
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Michael Foster
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Dries Vincent
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Leslie Alexander
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

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