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

This study is currently recruiting participants.
Verified January 2013 by QLT Inc.
Sponsor:
Information provided by (Responsible Party):
QLT Inc.
ClinicalTrials.gov Identifier:
NCT01543906
First received: February 17, 2012
Last updated: January 7, 2013
Last verified: January 2013
History of Changes
  Purpose

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.

Condition Intervention Phase
Retinitis Pigmentosa (RP)
 Drug: QLT091001
 Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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)

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by QLT Inc.:

Primary Outcome Measures:
  • Visual field [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 5
Study Start Date: February 2012
Estimated Study Completion Date: June 2014
Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: QLT091001
oral QLT091001 administered once daily for 7 days
 Drug: QLT091001
oral QLT091001 administered once daily for 7 days

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01543906

Contacts
Contact: Medical Information 1-877-764-3131 medaffairs@qltinc.com

Locations
Canada, Quebec
Montreal Children's Hospital, McGill University Health Centre Active, not recruiting
Montreal, Quebec, Canada
Ireland
Royal Victoria Eye and Ear Hospital Recruiting
Dublin, Ireland
Principal Investigator: Paul Kenna, MD            
Sponsors and Collaborators
QLT Inc.
Investigators
Study Director: Sushanta Mallick QLT Inc.
  More Information

No publications provided

Responsible Party: QLT Inc.
ClinicalTrials.gov Identifier: NCT01543906     History of Changes
Other Study ID Numbers: RET RP 01
Study First Received: February 17, 2012
Last Updated: January 7, 2013
Health Authority: Canada: Health Canada
Ireland: Irish Medicines Board

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 23, 2013