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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

Primary Purpose

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

Status

Completed

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

QLT091001

About this trial

This is an interventional treatment trial for LCA (Leber Congenital Amaurosis)

Eligibility Criteria

5 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases)
Wilmer Eye Institute (Johns Hopkins University)
Scheie Eye Institute
Montreal Children's Hospital, McGill University Health Centre
Institute for Ophthalmic Research, University of Tubingen
The Rotterdam Eye Hospital
Moorefield Eye Hospital

Outcomes

Primary Outcome Measures

Visual Field

Secondary Outcome Measures

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

Full Information

NCT ID

NCT01014052

First Posted

November 12, 2009

Last Updated

May 13, 2013

Sponsor

QLT Inc.

1. Study Identification

Unique Protocol Identification Number

NCT01014052

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)

Study Type

Interventional

2. Study Status

Record Verification Date

May 2013

Overall Recruitment Status

Completed

Study Start Date

November 2009 (undefined)

Primary Completion Date

August 2012 (Actual)

Study Completion Date

August 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

QLT Inc.

4. Oversight

Data Monitoring Committee

5. 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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

32 (Actual)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

QLT091001

Intervention Description

oral QLT091001 administered once daily for 7 days

Primary Outcome Measure Information:

Title

Visual Field

Time Frame

12 months

Secondary Outcome Measure Information:

Title

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

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Sushanta Mallick

Organizational Affiliation

QLT Inc.

Official's Role

Study Director

Facility Information:

Facility Name

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

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60608

Country

United States

Facility Name

Wilmer Eye Institute (Johns Hopkins University)

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21287

Country

United States

Facility Name

Scheie Eye Institute

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Montreal Children's Hospital, McGill University Health Centre

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H3H 1P3

Country

Canada

Facility Name

Institute for Ophthalmic Research, University of Tubingen

City

Tubingen

Country

Germany

Facility Name

The Rotterdam Eye Hospital

City

Rotterdam

Country

Netherlands

Facility Name

Moorefield Eye Hospital

City

London

ZIP/Postal Code

EC1 V2PD

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

26656277

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.

Results Reference

derived

PubMed Identifier

26448901

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.

Results Reference

derived

PubMed Identifier

25030840

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.

Results Reference

derived

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