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Completed

NCT Number: NCT01014052

Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

The purpose of this study is:

* to evaluate the safety of oral QLT091001 * to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations * to evaluate duration of visual function improvement (if observed)

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Key information

Conditions

LCA (Leber Congenital Amaurosis) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Leber Congenital Amaurosis RP (Retinitis Pigmentosa) Retinal Degeneration Retinal Diseases Retinal Dystrophies Retinitis Pigmentosa

Age range

5 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Montreal Children's Hospital, McGill University Health Centre, Montreal, Quebec, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subjects diagnosed with LCA or RP (with a mutation in either RPE65 or LRAT)
  • Subjects with LCA must be 5-65 years of age
  • Subjects with RP must be 18-65 years of age
  • Subjects who have a "best-corrected" visual acuity of 3 letters or better (20/800 Snellen equivalent) or viable photoreceptors on OCT/FAF.

Exclusion criteria

  • Subjects who are actively participating in an experimental therapy study or who have received experimental therapy within 60 days of Day 0.
  • Subjects with any clinically important abnormal physical finding at Screening.
  • Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane,® Soriatane®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.
  • Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis.
  • Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of screening

Treatment and study plan

QLT091001

Drug

oral QLT091001 administered once daily for 7 days

Primary outcomes

  1. Visual Field

    Time frame: 12 months

Secondary outcomes

  1. Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events

    Time frame: 12 months

Sponsors and collaborators

Lead sponsor

QLT Inc.

Industry

Registry information

Official study title

Phase 1b Study to Evaluate QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)

Important dates

Study start
2009
Primary completion
2012
Study completion
2012
First posted
Nov 16, 2009
Registry last updated
May 14, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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