Retinoid Replacement for Leber Congenital Amaurosis or Retinitis Pigmentosa


Description: This Phase IB clinical trial is evaluating the safety and proof of concept of an oral retinoid replacement therapy for children and young adults with Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in either LRAT or RPE65. QLT has reported that participants in the study have experienced improvements in visual acuity, peripheral vision, and/or retinal sensitivity.

Trial status: Recruiting participants.

Retinoid Replacement for Leber Congenital Amaurosis or Retinitis Pigmentosa

Partial List of Participation Criteria:

  1. Diagnosis of LCA or RP caused by mutation in RPE65 or LRAT
  2. 5 to 75 years of age

Additional Information:
QLT retinoid clinical trial listing on

Clinical Contact:

United States, Illinois
The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease)
Chicago, Illinois, United States, 60608
Contact: Carol White 312-447-3254
Principal Investigator: Gerald Fishman, MD

United States, Maryland
Wilmer Eye Institute (Johns Hopkins University)
Baltimore, Maryland, United States, 21287
Contact: Kristen Bowles, O.D., M.S., 410-614-6908
Principal Investigator: Hendrik Scholl, MD, M.A.

United States, Pennsylvania
Scheie Eye Institute
Philadelphia, Pennsylvania, United States, 19104
Contact: Sharon Wolfe-Schwartz 215-662-9981
Principal Investigator: Samuel Jacobson, MD, PhD

Canada, Quebec
Montreal Children's Hospital, McGill University Health Centre
Montreal, Quebec, Canada, H3H 1P3
Contact: Eunice Esteban 514.412.4400 ext 22891 or 22530
Principal Investigator: Robert Koenekoop, MD, PhD

Institute for Ophthalmic Research, University of Tubingen
Tubingen, Germany
Contact: Eberhart Zrenner +49 70 71 298 4786
Principal Investigator: Eberhart Zrenner

The Rotterdam Eye Hospital
Rotterdam, Netherlands
Contact: L. Ingeborgh van den Born, MD, PhD +31 0 10 401777
Principal Investigator: L. Ingeborgh van den Born, MD, PhD

United Kingdom
Moorefield Eye Hospital
London, United Kingdom, EC1 V2PD
Contact: Isabelle Molden +44 0 20 756 62264
Principal Investigator: Tony Moore