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: eber Congenital AmaurosisRetinitis pigmentosa; MedDRA version: 14.1Level: PTClassification code 10038914Term: Retinitis pigmentosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 14.1Level: PTClassification code 10070667Term: Leber's congenital amaurosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2011-004214-42-NL
• Lead Sponsor: QLT Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-05-23
• Last Update Posted: 11 August 2014

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 31

Inclusion Criteria

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): i.e., follow-up GVF increased =20% from baseline
or
b. Decrease in GVF (in at least 1 eye): i.e., follow-up GVF decreased below the highest previous response by >20%
or
c. Considered a reasonable candidate for treatment, 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 prior to study dosing. 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. (Women are considered postmenopausal after 1 complete year of amenorrhea).
4. Subjects who are boys or men must (1) agree to completely abstain from sexual intercourse, (2) have had a vasectomy, (3) use a barrier method (condoms) with spermicide and, if his partner is not using oral contraceptives, implantable or injectable contraceptives, or an IUD (as described in the criterion above), ensure that she is using a diaphragm with spermicidal gel 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. The parent or guardian must sign an approved informed consent form for the study for subjects younger than the age of majority.
6. 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.
7. 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

Exclusion Criteria

1. Subjects with any clinically important abnormal physical finding at Screening as determined by the Investigator.
2. Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane/Roaccutane® or Soriatane/Neotigason®) excluding study medication QLT091001 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, cirrhosis, hepatitis, uncontrolled thyroid disease, or chronic hyperlipidemia.
4. Subjects with a hypersensitivity to retinoids or hypervitaminosis A syndrome.
5. Subjects with any of the following findings at Screening:
• Blood pressure reading upon repeated measurement (i.e., 2 measurements) taken in a sitting or supine position of the following (by age group).
5-9 years:
120/80 mmHg or higher
10-13 years:
132/83 mmHg or higher
14-17 years:
140/90 mmHg or higher
18 years and older:
150/95 mmHg or higher
• Resting heart rate upon repeated measurement of the following (unless the subject has a known consistent lower heart rate):
5-9 years:
<70 bpm or >130 bpm
10-13 years:
<45 bpm or >105 bpm
14-17 years:
<40 bpm or >100 bpm
18 years and older:
<40 bpm or >100 bpm
• ALT, AST, or fasting triglycerides >3 times the upper limit of the clinical laboratory value normal range (upon repeated measurement)
• Thyroid stimulating hormone (TSH) >2 times the upper limit, or 0.75 times the lower limit, of the clinical laboratory value normal range (upon repeated measurement); other thyroid function tests to be evaluated by Investigator to exclude subjects with uncontrolled thyroid disease.
• Serum retinol clinical laboratory value above 98 µg/dL.
• 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 QTc prolongation (>450 milliseconds [ms]) upon repeated demonstration (e.g., 2 of 3 assessments) at screening or baseline, which is considered to be clinically significant by the Investigator.
7. Subjects with a history of 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.
10. Subjects with a documented and known allergy to soy.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified