NCT01014052

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)

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Nov 2009

Typical duration for phase_1

Geographic Reach
5 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2009

Completed
11 days until next milestone

First Submitted

Initial submission to the registry

November 12, 2009

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 16, 2009

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2012

Completed
Last Updated

May 14, 2013

Status Verified

May 1, 2013

Enrollment Period

2.8 years

First QC Date

November 12, 2009

Last Update Submit

May 13, 2013

Conditions

LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)

Outcome Measures

Primary Outcomes (1)

  • Visual Field

    12 months

Secondary Outcomes (1)

  • Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events

    12 months

Interventions

QLT091001DRUG

oral QLT091001 administered once daily for 7 days

Eligibility Criteria

Age5 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

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

Chicago, Illinois, 60608, United States

Location

Wilmer Eye Institute (Johns Hopkins University)

Baltimore, Maryland, 21287, United States

Location

Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

Location

Montreal Children's Hospital, McGill University Health Centre

Montreal, Quebec, H3H 1P3, Canada

Location

Institute for Ophthalmic Research, University of Tubingen

Tübingen, Germany

Location

The Rotterdam Eye Hospital

Rotterdam, Netherlands

Location

Moorefield Eye Hospital

London, EC1 V2PD, United Kingdom

Location

Related Publications (3)

  • 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.

    PMID: 26656277DERIVED

  • 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.

    PMID: 26448901DERIVED

  • 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.

    PMID: 25030840DERIVED

MeSH Terms

Conditions

Leber Congenital AmaurosisRetinitis Pigmentosa

Interventions

retinol acetate

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DiseasesRetinal DystrophiesRetinal DegenerationGenetic Diseases, InbornCongenital, 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

November 12, 2009

First Posted

November 16, 2009

Study Start

November 1, 2009

Primary Completion

August 1, 2012

Study Completion

August 1, 2012

Last Updated

May 14, 2013

Record last verified: 2013-05

Locations

CA(1)DE(1)NL(1)GB(1)US(3)