Stargardt Disease MedDRA version: 20.1 Level: PT Classification code 10062766 Term: Stargardt's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Katairo GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Elevated qAF in at least one eye at screening (value =300 Units). • Male or female of any ethnicity and = 18 years old. • Onset of STGD disease before the age of 45 years • Visual acuity of the study eye: BCVA 0.2-0.8 (decimal unit). • Clinical diagnosis of typical autosomal recessive Stargardt’s macular dystrophy (STGD1). • Genetic report indicating at least two ABCA4 mutations (one with confirmed pathogenesis by a certified lab, one reported previously). • Study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eye drops and with sufficient fixation to permit good quality retinal imaging. • Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 12-months study. • Signed and dated informed consent form. • Female subjects of childbearing potential and male subjects participating in the study who are sexually active must use acceptable contraception. Male and female subjects documented as being of non-child bearing potential (e.g. infertile, surgically sterile, postmenopausal) are exempt from the contraceptive requirements. • Willing to avoid excessive exposure to sun light (e.g. by using a hat, UV absorbing sunglasses and sunscreen with a minimum SPF of 30). Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 90 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 90
Exclusion criteria
Exclusion criteria: • Intolerance to acid pump antagonists (APAs). • Hypersensitivity to soraprazan or to any of the excipients. • Intake of prohibited medications/supplements (supplements containing vitamin A or beta-carotene, medications to treat any liver disease, or oral retinoid medications) within 28 days prior to screening and throughout the study. • Intake of other drugs with a pH dependent absorption, e.g. ketoconazole. • Breastfeeding, pregnant, or positive urine pregnancy test at screening or visit 2 (first intake of Investigational Medicinal Product, IMP). • At screening, clinically significant abnormal haematology or biochemistry findings. Levels of aspartate aminotransferase (AST), alanine aminotransferase (ALT), and/or total bilirubin >1.5 x ULN (upper limit of normal) at screening will lead to exclusion. • Acute or unstable severe disease or history of disease which in the opinion of the investigator would preclude participation in the study. • Active or history of an additional ocular disorder in the primary study eye that, in the opinion of the investigator, may confound the study results. These include, but are not limited to, any reason that might interfere with the imaging techniques used in the study (such as optic media opacity or poor pupil dilatation), inflammatory eye disease, other retinal disorders besides STGD, confirmed glaucoma or baseline intraocular pressure of =25mmHg, optic neuritis, high myopia (>8D spherical equivalent), amblyopia. • Intraocular surgery or injections in the primary study eye within 180 days of the screening visit. • Clinically significant abnormal electrocardiogram (ECG), or a corrected QT interval (QTc) of =450 ms in males or =470 ms in females. • Participation in any other investigational clinical trial within 28 days of the screening visit.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the efficacy of soraprazan in reducing the amount of lipofuscin in RPE cells of subjects with Stargardt disease by assessing the change in quantitative autofluorescence (qAF8) from baseline to after treatment with soraprazan compared to placebo for up to 12 months.;Secondary Objective: Evaluate the efficacy of soraprazan in reducing the amount of lipofuscin in RPE cells of subjects with Stargardt disease by assessing the change in quantitative auto-fluorescence (qAF8) from baseline to after treatment with soraprazan compared to placebo for up to 24 months. Assess the safety and efficacy of soraprazan based on safety parameters and secondary efficacy parameters such as change in visual function and structural changes after treatment with soraprazan compared to placebo for up to 24 months. ;Primary end point(s): Change in qAF8 score from baseline to Month 12 (Visit 10) for soraprazan compared to placebo treated subjects.;Timepoint(s) of evaluation of this end point: Baseline to Month 12 (Visit 10) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change in qAF8 score from baseline to Month 24 for soraprazan compared to placebo treated subjects. Evaluation of qAF8 score for all subjects will be done at a central reading centre. Comparison will be made between soraprazan and placebo treatment arms at Month 12 and Month 24 for the following: Functional 1. Best-corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) charts 2. BCVA as measured by ETDRS chart under low luminance conditions (LLVA) 3. Low Luminance Deficit (LLD) calculated as the difference between BCVA and LLVA 4. Change from baseline in binocular and monocular maximum reading speed as assessed by Radner Charts 5. Change from baseline in binocular and monocular critical print size as assessed by Radner charts. 6. Macular functional response as assessed by mesopic microperimetry 7. Subject-reported visual function as assessed by the National Eye Institute Visual Functioning Questionnaire 25 (NEI VFQ-25) and Functional Reading Independence (FRI) Index Structural 8. Changes in central subfield retinal thickness (CSRT) as assessed by spectral-domain optical coherence tomography (SD-OCT) 9. Changes in macular volume as assessed by SD-OCT 10. Changes in outer nuclear layer (ONL) thickness as assessed by SD-OCT 11. Changes in Ellipsoid Zones (EZ) as assessed by SD-OCT 12. Presence and changes of macular atrophy by fundus autofluorescence 13. Lesion progression Exploratory 14. Pupillographic Campimetry (only site Tübingen) 15. Adaptive Optics (only site Tübingen);Timepoint(s) of evaluation of this end point: Baseline and until end of treatment | — |
Countries
Germany, Netherlands, United Kingdom
Contacts
Public ContactProject Manager
SMERUD Medical Research
Outcome results
None listed