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.

Tracking Information

• First Submitted Date: November 12, 2009
• First Posted Date: November 16, 2009
• Last Update Posted Date: May 14, 2013
• Study Start Date: November 2009
• Actual Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: February 28, 2012): Visual Field [ Time Frame: 12 months ]
• Original Primary Outcome Measures (submitted: November 13, 2009): Best-corrected visual acuity, color vision, visual fields, electroretinogram (ERG) and full-field stimulus threshold test (FST) [ Time Frame: 14 days ]
• Current Secondary Outcome Measures (submitted: September 16, 2011): Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 12 months ]
• Original Secondary Outcome Measures (submitted: November 13, 2009): Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events [ Time Frame: 14 days ]
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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)

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)

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

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

Intervention

Drug: QLT091001

oral QLT091001 administered once daily for 7 days

• Study Arms: Not Provided

Publications *

• 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. doi: 10.1167/tvst.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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 13, 2013): 32
• Original Estimated Enrollment (submitted: November 13, 2009): 8
• Actual Study Completion Date: August 2012
• Actual Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 5 Years to 65 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, Germany, Netherlands, United Kingdom, United States

Administrative Information

• NCT Number: NCT01014052
• Other Study ID Numbers: RET IRD 01
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: QLT Inc.
• Original Responsible Party: Study Manager, QLT Inc.
• Current Study Sponsor: QLT Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Sushanta Mallick; QLT Inc.

• PRS Account: QLT Inc.
• Verification Date: May 2013