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

LCA (Leber Congenital Amaurosis) Clinical Trial

Official 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01014052
• Other study ID #: RET IRD 01
• Status: Completed
• Phase: Phase 1
• First received: November 12, 2009
• Last updated: May 13, 2013
• Start date: November 2009
• Est. completion date: August 2012

Study information

• Verified date: May 2013
• Source: QLT Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health CanadaUnited States: Food and Drug AdministrationGermany: Federal Institute for Drugs and Medical DevicesNetherlands: Dutch Health Care InspectorateUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

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)

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: August 2012
• Est. primary completion date: August 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years to 65 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• LCA (Leber Congenital Amaurosis)
• Leber Congenital Amaurosis
• Retinitis
• Retinitis Pigmentosa
• RP (Retinitis Pigmentosa)

Intervention

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

Locations

Country	Name	City	State
Canada	Montreal Children's Hospital, McGill University Health Centre	Montreal	Quebec
Germany	Institute for Ophthalmic Research, University of Tubingen	Tubingen
Netherlands	The Rotterdam Eye Hospital	Rotterdam
United Kingdom	Moorefield Eye Hospital	London
United States	Wilmer Eye Institute (Johns Hopkins University)	Baltimore	Maryland
United States	The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)	Chicago	Illinois
United States	Scheie Eye Institute	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
QLT Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Visual Field		12 months	No
Secondary	Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events		12 months	Yes