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

NCT ID: NCT01543906

Last Updated: 2014-12-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-02-29

Study Completion Date

2014-08-31

Brief Summary

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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

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Conditions

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Retinitis Pigmentosa (RP)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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QLT091001

oral QLT091001 administered once daily for 7 days

Group Type EXPERIMENTAL

QLT091001

Intervention Type DRUG

oral QLT091001 administered once daily for 7 days

Interventions

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QLT091001

oral QLT091001 administered once daily for 7 days

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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QLT Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sushanta Mallick

Role: STUDY_DIRECTOR

QLT Inc.

Locations

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Montreal Children's Hospital, McGill University Health Centre

Montreal, Quebec, Canada

Site Status

Royal Victoria Eye and Ear Hospital

Dublin, , Ireland

Site Status

Countries

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Canada Ireland

References

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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.

Reference Type DERIVED
PMID: 32573764 (View on PubMed)

Other Identifiers

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RET RP 01

Identifier Type: -

Identifier Source: org_study_id