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

• ClinicalTrials.gov Identifier: NCT01014052
• Recruitment Status: Completed
• First Posted: November 16, 2009
• Last Update Posted: May 14, 2013
• Sponsor: QLT Inc.
• Information provided by (Responsible Party): QLT Inc.

Study Description

Brief 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)

Condition or disease	Intervention/treatment	Phase
LCA (Leber Congenital Amaurosis); RP (Retinitis Pigmentosa)	Drug: QLT091001	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 32 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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)
• Study Start Date: November 2009
• Actual Primary Completion Date: August 2012
• Actual Study Completion Date: August 2012

Arms and Interventions

Intervention Details:

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

Outcome Measures

Primary Outcome Measures :

1. Visual Field [ Time Frame: 12 months ]

Secondary Outcome Measures :

1. Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 12 months ]

Eligibility Criteria

• Ages Eligible for Study: 5 Years to 65 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

United States, Illinois

The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)

Chicago, Illinois, United States, 60608

United States, Maryland

Wilmer Eye Institute (Johns Hopkins University)

Baltimore, Maryland, United States, 21287

United States, Pennsylvania

Scheie Eye Institute

Philadelphia, Pennsylvania, United States, 19104

Canada, Quebec

Montreal Children's Hospital, McGill University Health Centre

Montreal, Quebec, Canada, H3H 1P3

Germany

Institute for Ophthalmic Research, University of Tubingen

Tubingen, Germany

Netherlands

The Rotterdam Eye Hospital

Rotterdam, Netherlands

United Kingdom

Moorefield Eye Hospital

London, United Kingdom, EC1 V2PD

Sponsors and Collaborators

QLT Inc.

Investigators

• Study Director: Sushanta Mallick; QLT Inc.

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Scholl HP, Moore AT, Koenekoop RK, Wen Y, Fishman GA, van den Born LI, Bittner A, Bowles K, Fletcher EC, Collison FT, Dagnelie G, Degli Eposti S, Michaelides M, Saperstein DA, Schuchard RA, Barnes C, Zein W, Zobor D, Birch DG, Mendola JD, Zrenner E; RET IRD 01 Study Group. Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT). PLoS One. 2015 Dec 10;10(12):e0143846. doi: 10.1371/journal.pone.0143846. eCollection 2015.

Wen Y, Birch DG. Outer Segment Thickness Predicts Visual Field Response to QLT091001 in Patients with RPE65 or LRAT Mutations. Transl Vis Sci Technol. 2015 Oct 1;4(5):8. eCollection 2015 Oct.

Koenekoop RK, Sui R, Sallum J, van den Born LI, Ajlan R, Khan A, den Hollander AI, Cremers FP, Mendola JD, Bittner AK, Dagnelie G, Schuchard RA, Saperstein DA. Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial. Lancet. 2014 Oct 25;384(9953):1513-20. doi: 10.1016/S0140-6736(14)60153-7. Epub 2014 Jul 13.

• Responsible Party: QLT Inc.
• ClinicalTrials.gov Identifier: NCT01014052
• Other Study ID Numbers: RET IRD 01
• First Posted: November 16, 2009
• Last Update Posted: May 14, 2013
• Last Verified: May 2013

Additional relevant MeSH terms:

Blindness; Retinitis; Retinitis Pigmentosa; Leber Congenital Amaurosis; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Genetic Diseases, Inborn; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Retinol acetate; Adjuvants, Immunologic; Immunologic Factors; Physiological Effects of Drugs; Anticarcinogenic Agents; Protective Agents; Antineoplastic Agents