Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With... | NCT01543906

Studies
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)

NCT01543906

Sponsor

QLT Inc.

Status

Completed

Last Update Posted

Dec 15, 2014Estimated

Enrollment

5Actual

Phase

Phase 1

Conditions

Retinitis Pigmentosa (RP)

Interventions

QLT091001

Countries

Canada
Ireland

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2014

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Feb 2012

Primary Completion Date

Jul 2014Actual

Completion Date

Aug 2014Actual

First Submitted Date

Feb 17, 2012

First Submission Date that Met QC Criteria

Mar 1, 2012

First Posted Date

Mar 5, 2012Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 11, 2014

Last Update Posted Date

Dec 15, 2014Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The purpose of this study is:

To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day treatment with oral QLT091001.
To evaluate the safety of oral QLT091001 administered once daily for 7 days in RP subjects with an autosomal dominant mutation in RPE65.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

5Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
QLT091001	Experimental	oral QLT091001 administered once daily for 7 days	Drug: QLT091001

Interventions

Name	Type	Description	Arm Group Labels	Other Names
QLT091001	Drug	oral QLT091001 administered once daily for 7 days	QLT091001

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Visual field		12 months

Secondary Outcomes

Measure	Description	Time Frame
Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs		12 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Subjects will have RP caused by an autosomal dominant mutation in RPE65, as diagnosed by an ocular geneticist or ophthalmologist.
Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent) or visible photoreceptor outer segments on OCT/FAF.

Exclusion Criteria:

Subjects with any clinically important abnormal physical finding at Screening.
Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane/Roaccutane® or Soriatane/Neotigason®) 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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

70 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Sushanta Mallick	QLT Inc.	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Montreal Children's Hospital, McGill University Health Centre		Montreal	Quebec		Canada
Royal Victoria Eye and Ear Hospital

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
32573764	Derived	Schwartz SG, Wang X, Chavis P, Kuriyan AE, Abariga SA. Vitamin A and fish oils for preventing the progression of retinitis pigmentosa. Cochrane Database Syst Rev. 2020 Jun 18;6(6):CD008428. doi: 10.1002/14651858.CD008428.pub3.

ID	Term
D015785	Eye Diseases, Hereditary
D005128	Eye Diseases
D058499	Retinal Dystrophies
D012162	Retinal Degeneration