Clinical trial NCT01014052: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: LCA (Leber Congenital Amaurosis)
RP (Retinitis Pigmentosa)
Lead sponsor: QLT Inc.
Sponsor class: INDUSTRY
First posted: 2009-11-16
Enrollment: 32
Sex: ALL
Minimum age (years): 5
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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)


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

Primary outcomes:
measure: Visual Field
timeFrame: 12 months
Secondary outcomes:
measure: Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events
timeFrame: 12 months
Other outcomes:
Locations:
city: Chicago
country: United States
facility: The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)
state: Illinois
city: Baltimore
country: United States
facility: Wilmer Eye Institute (Johns Hopkins University)
state: Maryland
city: Philadelphia
country: United States
facility: Scheie Eye Institute
state: Pennsylvania
city: Montreal
country: Canada
facility: Montreal Children's Hospital, McGill University Health Centre
state: Quebec
city: Tubingen
country: Germany
facility: Institute for Ophthalmic Research, University of Tubingen
city: Rotterdam
country: Netherlands
facility: The Rotterdam Eye Hospital
city: London
country: United Kingdom
facility: Moorefield Eye Hospital
Study officials:
affiliation: QLT Inc.
name: Sushanta Mallick
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.
citation: 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.