Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)

Overview

The purpose of this study is: – To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65. – To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day treatment with oral QLT091001. – To evaluate the safety of oral QLT091001 administered once daily for 7 days in RP subjects with an autosomal dominant mutation in RPE65.

Full Title of Study: “An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: July 2014

Interventions

  • Drug: QLT091001
    • oral QLT091001 administered once daily for 7 days

Arms, Groups and Cohorts

  • Experimental: QLT091001
    • oral QLT091001 administered once daily for 7 days

Clinical Trial Outcome Measures

Primary Measures

  • Visual field
    • Time Frame: 12 months

Secondary Measures

  • Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG’s and vital signs
    • Time Frame: 12 months

Participating in This Clinical Trial

Inclusion Criteria

  • Subjects will have RP caused by an autosomal dominant mutation in RPE65, as diagnosed by an ocular geneticist or ophthalmologist. – Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent) or visible photoreceptor outer segments on OCT/FAF. Exclusion Criteria:

  • Subjects with any clinically important abnormal physical finding at Screening. – Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane/Roaccutane® or Soriatane/Neotigason®) 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.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 70 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • QLT Inc.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Sushanta Mallick, Study Director, QLT Inc.

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