A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' visio
- Conditions
- Leber Congenital Amaurosis Retinitis pigmentosaMedDRA version: 20.0 Level: PT Classification code 10070667 Term: Leber's congenital amaurosis System Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.0 Level: PT Classification code 10038914 Term: Retinitis pigmentosa System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Eye Diseases [C11]
- Registration Number
- EUCTR2011-004214-42-GB
- Lead Sponsor
- QLT Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 31
1. Subjects with LCA or RP due to RPE65 or LRAT deficiency who received a 7-day treatment course of QLT091001 and completed the Day 30 visit in Study RET IRD 01.
2. Subjects who meet any one of the following criteria at least 1 month after the start of the 7-day treatment course in Study RET IRD 01:
a. No increase in GVF (in at least 1 eye): Follow-up GVF increased =20% from baseline or
b. Decrease in GVF (in at least 1 eye): Follow-up GVF decreased below the highest previous response by >20% or
c. Considered a reasonable candidate for retreatment, as determined by the Investigator based on regression or lack of response in other parameters of visual function (e.g., subjective reports of changes in color vision or adaptation to low light) but who do not meet the other (GVF) criteria for study entry
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 last dose of study drug in Treatment Course 3.
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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 11
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Subjects with any clinically important abnormal physical finding at Screening.
2. 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.
3. Subjects with liver failure, uncontrolled thyroid disease, hypersensitivity to retinoids, or hypervitaminosis A.
4. 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, 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
5. 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.
6. Subjects with a marked baseline prolongation of QT/QTc intervals (e.g., repeated demonstration of a QTc interval >450 milliseconds [ms]).
7. 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.
8. Subjects who have taken any supplements containing =10,000 IU vitamin A within 60 days of Screening.
9. Subjects who are actively participating in an experimental therapy study (other than RET IRD 01) or who have received another experimental therapy (in addition to QLT091001) within 60 days of Day 0.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method