Top
X-Linked Retinitis Pigmentosa - Healing Genes
1289
post-template-default,single,single-post,postid-1289,single-format-standard,mkdf-bmi-calculator-1.0,mkd-core-1.1.1,tribe-no-js,wellspring-ver-1.8,mkdf-smooth-page-transitions,mkdf-ajax,mkdf-grid-1300,mkdf-blog-installed,mkdf-header-standard,mkdf-no-behavior,mkdf-default-mobile-header,mkdf-sticky-up-mobile-header,mkdf-dropdown-slide-from-bottom,mkdf-full-width-wide-menu,mkdf-search-dropdown,elementor-default,elementor-template-full-width,elementor-kit-3486,elementor-page-2540

X-Linked Retinitis Pigmentosa

Share This Post

Share on facebook
Share on linkedin
Share on twitter
Share on email

A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa (XIRIUS)

A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)


Phase 1 / 2 /3

DESCRIPTION:
Researchers in PA, OR, and FL are recruiting male X-Linked Retinitis Pigmentosa patients for an investigation of a gene therapy designed to impart a functional retinitis pigmentosa GTPase regulator gene (RPGR) to correct the disease. The treatment would require a single intravitreal (into the retina) injection of the drug, a genetically modified and harmless virus, at 1 of 6 dosage levels. The patient will be followed for ~ 2 years to assess for adverse safety events.


PATIENT MUST:

  • Be 10 years of age or older and male
  • Have documentation of a mutation in the RPGR gene
  • Not have participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks

THE STUDY INVOLVES:

  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. Intravitreal injection of the gene therapy at one of several dose levels.
  3. Follow up for ~ 2 years to screen for adverse safety events

LOCATIONS AND CONTACTS:
The study sites are not yet disclosed. Contact the study’s contact to qualify.
Contacts:
Nightstar Therapeutics  |  +44 207 611 2077 (international) |  [email protected]

SPONSOR INFORMATION:
Nightstar Therapeutics

Or go online:
https://clinicaltrials.gov/ct2/show/NCT03116113

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader