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

NCT01543906 | Completed Phase 1

• 1 other identifier: RET RP 01
• Study Type: interventional
• Target: 5
• Locations: 2 countries
• Sites: 2

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.

Conditions

Retinitis Pigmentosa (RP)

Outcome Measures

Primary Outcomes (1)

• Visual field

  12 months

Secondary Outcomes (1)

• Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs

  12 months

Study Arms (1)

QLT091001

EXPERIMENTAL

oral QLT091001 administered once daily for 7 days

Drug: QLT091001

Interventions

QLT091001 DRUG

oral QLT091001 administered once daily for 7 days

QLT091001

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• QLT Inc.: lead

Study Sites (2)

Montreal Children's Hospital, McGill University Health Centre

Montreal, Quebec, Canada

Location

Royal Victoria Eye and Ear Hospital

Dublin, Ireland

Location

Related Publications (1)

• Schwartz SG, Wang X, Chavis P, Kuriyan AE, Abariga SA. Vitamin A and fish oils for preventing the progression of retinitis pigmentosa. Cochrane Database Syst Rev. 2020 Jun 18;6(6):CD008428. doi: 10.1002/14651858.CD008428.pub3.

  PMID: 32573764 DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa

Interventions

retinol acetate

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Sushanta Mallick

  QLT Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 17, 2012
• First Posted: March 5, 2012
• Study Start: February 1, 2012
• Primary Completion: July 1, 2014
• Study Completion: August 1, 2014
• Last Updated: December 15, 2014

Record last verified: 2014-12

Locations

CA (1); IE (1)