An Open-Label, Phase 1b Safety/Proof-of-Concept Study to Evaluate the Effects of Oral 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)
- Conditions
- Retinitis Pigmentosa / Inherited ophthalmology disorder10047518
- Registration Number
- NL-OMON38343
- Lead Sponsor
- QLT Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 4
1. Subjects will have either LCA or RP, with a mutation in either RPE65 or LRAT, as follows:
a. Subjects with LCA will be 5-65 years of age diagnosed with LCA by an ocular geneticist or pediatric ophthalmologist.
b. Subjects with RP will be diagnosed with RP by an ocular geneticist or ophthalmologist and be:
For Canada, Germany, The Netherlands, and UK: 18-65 years of age (inclusive).
For US only: 8-65 years of age (inclusive). ;2. Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent), however; subjects with a lower ETDRS score will be eligible if spectral domain OCT and FAF reveals evidence of a viable photoreceptor layer. ;3. Subjects who are girls or women of child-bearing potential must not be pregnant or lactating, must have negative serum pregnancy tests (>=25 mIU/mL sensitivity) at Screening (i.e., >=19 days before Day -1, and on Day -1) and must have been practicing 2 adequate methods of birth control or complete abstinence for at least 2 months.
Adequate methods of birth control include (1) use of oral contraceptives (excluding low-dose oral formulation), implantable or injectable contraceptives, or an intrauterine device (IUD), with an additional barrier method (diaphragm with spermicidal gel OR condoms with spermicide); (2) a double-barrier method (diaphragm with spermicidal gel AND condoms with spermicide); (3) partner vasectomy; and (4) total abstinence. ;4. Subjects who are boys or men must (1) agree to completely abstain from sexual intercourse, (2) have had a vasectomy, or (3) use a barrier method (condoms) with spermicide during sexual intercourse, during the treatment phase of the study and for 2 months after finishing the study drug. ;5. Subjects who provide informed consent and, if applicable, assent for the study (applies to subjects 7 years and older). The parent or guardian must sign an approved informed consent form for the study for subjects younger than the age of majority.
Note that use of the term subject throughout this protocol may also include the parent/guardian of subjects younger than the age of majority, as appropriate. ;6. Subjects who are willing to comply with the protocol. ;In the Netherlands only adult RP patients (older then 18 years) will be included.
1. Subjects who are actively participating in an experimental therapy study or who have received experimental therapy within 60 days of Day 0. ;2. Subjects with any clinically important abnormal physical finding at Screening. ;3. 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. ;4. Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis. ;5. Subjects with liver failure, uncontrolled thyroid disease, hypersensitivity to retinoids, or hypervitaminosis A. ;6. Subjects with any of the following findings at Screening: ;- Untreated blood pressure 150/95 mm Hg or higher upon repeated measurement ;- Resting heart rate <40 bpm or >100 bpm upon repeated measurement ;- ALT or AST >3 times the upper limit of the clinical laboratory value normal range upon repeated measurement ;- Total cholesterol, triglycerides, HDL, or LDL >2 times the upper limit of the clinical laboratory value normal range upon repeated measurement ;- Thyroid function tests outside the clinical laboratory value normal range upon repeated measurement ;- Serum retinol clinical laboratory value above 90 µg/dL upon repeated measurement ;7. Subjects with a documented and known allergy to soy. ;8. Subjects who, in the Investigator*s opinion, have any severe acute or chronic medical condition, psychiatric condition, or laboratory abnormality that may increase the risk associated with study participation or administration of study treatment, or interfere with the interpretation of study results. ;9. Subjects with a marked baseline prolongation of QT/QTc intervals (e.g., repeated demonstration of a QTc interval >450 milliseconds [ms]). ;10. Subjects with a history of additional risk factors for torsade de pointes (TdP) (e.g., heart failure, hypokalemia, history or family history of Long QT Syndrome), and Wolff-Parkinson-White (WPW) syndrome. ;11. Subjects who have taken any supplements containing >=10,000 IU vitamin A within 60 days of Screening.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Assessment of Vision;<br /><br>Assessment of Safety</p><br>
- Secondary Outcome Measures
Name Time Method <p>not applicable</p><br>