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

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ClinicalTrials.gov Identifier: NCT01543906
Recruitment Status : Completed
First Posted : March 5, 2012
Last Update Posted : December 15, 2014
Sponsor:
Information provided by (Responsible Party):
QLT Inc.

Brief Summary:

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 or disease Intervention/treatment Phase
Retinitis Pigmentosa (RP) Drug: QLT091001 Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (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)
Study Start Date : February 2012
Actual Primary Completion Date : July 2014
Actual Study Completion Date : August 2014


Arm Intervention/treatment
Experimental: QLT091001
oral QLT091001 administered once daily for 7 days
Drug: QLT091001
oral QLT091001 administered once daily for 7 days



Primary Outcome Measures :
  1. Visual field [ Time Frame: 12 months ]

Secondary Outcome Measures :
  1. Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs [ Time Frame: 12 months ]


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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01543906


Locations
Canada, Quebec
Montreal Children's Hospital, McGill University Health Centre
Montreal, Quebec, Canada
Ireland
Royal Victoria Eye and Ear Hospital
Dublin, Ireland
Sponsors and Collaborators
QLT Inc.
Investigators
Study Director: Sushanta Mallick QLT Inc.

Responsible Party: QLT Inc.
ClinicalTrials.gov Identifier: NCT01543906     History of Changes
Other Study ID Numbers: RET RP 01
First Posted: March 5, 2012    Key Record Dates
Last Update Posted: December 15, 2014
Last Verified: December 2014

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Cone-Rod Dystrophies
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn
Retinol acetate
Vitamin A
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Anticarcinogenic Agents
Protective Agents
Antineoplastic Agents
Vitamins
Micronutrients
Growth Substances