EUCTR2011-004214-42-GB进行中（未招募）1 期
An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome 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) (Extension of Study RET IRD 01)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- QLT Inc.
- 入组人数
- 31
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •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
排除标准
- •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.
研究者
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