2024-515553-17-00
Active, not recruiting
Phase 1/2
Safety and efficacy of a bilateral single subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia investigated in a randomized, wait list controlled, observer-masked trial (Colour Bridge)
Universitaetsklinikum Tuebingen AöR1 site in 1 country14 target enrollmentJuly 16, 2024
Overview
- Phase
- Phase 1/2
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- Universitaetsklinikum Tuebingen AöR
- Enrollment
- 14
- Locations
- 1
- Primary Endpoint
- Contrast sensitivity (Pelli Robson 3 m) 6 months after treatment
- Status
- Active, not recruiting
- Last Updated
- last year
Overview
Brief Summary
To proof the safety and efficacy of rAAV.hCNGA3 in patients with achromatopsia if applied bilaterally in adults and minors.
Investigators
Dr. med Tobias Peters
Scientific
Universitaetsklinikum Tuebingen AöR
Eligibility Criteria
Inclusion Criteria
- •clinical diagnosis of achromatopsia
- •Female patients of childbearing potential must agree to use an effective method ofbirth control during the first 6 months post treatment.
- •negative pregnancy test in women with childbearing potential (a woman who istwo years post-menopausal or surgically sterile is not considered to be ofchildbearing potential)
- •6-12 years of age
- •≥ 18 years of age
- •bi-allelic pathogenic or likely pathogenic mutation in CNGA3
- •BCVA ≥ 20/400
- •a minimal outer nuclear layer thickness of 10μm at 3° eccentricity (normal =38±6μm)
- •ability to understand and willingness to consent to study protocol
- •no infection with Human Immundeficiency Virus (HIV)
Exclusion Criteria
- •any other retinopathy due to other diseases e.g. (but not limited to) arterialhypertension, trauma or acquired inflammatory diseases (uveitis serology) ,retinopathy of the premature
- •causal mutations in other genes for hereditary retinal diseases
- •contraindications in view of the planned surgery (e.g. anaemia Hb<8g/dl, severecoagulopathy, severe blood pressure fluctuations) including intolerance andcontraindications to general anaesthesia
- •ocular opacity and mature cataract
- •ocular infection with herpes simplex virus in medical history
- •history of ocular malignancies
- •disorders of the inner retina (e.g. retinal vascular occlusions in the patients history)
- •glaucoma defined as damage of the optic nerve
- •history of poorly controlled (HbA1c > 7%) Diabetes Mellitus type 1 or type 2
- •patients treated with systemic corticoids within 14 days prior inclusion
Outcomes
Primary Outcomes
Contrast sensitivity (Pelli Robson 3 m) 6 months after treatment
Contrast sensitivity (Pelli Robson 3 m) 6 months after treatment
Secondary Outcomes
- FrACT (Freiburg Visual Acuity & Contrast Test)
- Roth FM28 sat
- Contrast sensitivity (Pelli Robson 3 m)
- BCVA assessed using the ETDRS visual acuity protocol
- VA-CAL (Visual acuity under different conditions of contrast and ambient light)
- Patient reported outcomes (VFQ25/CVFQ, A3-PRO)
- Electroretinography
- Chromatic pupil campimetry (CPC) cone protocol – exploratory - functional
Study Sites (1)
Loading locations...
Similar Trials
Recruiting
Phase 1
Safety and efficacy of a bilateral single subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia investigated in a randomized, wait list controlled, observer-masked trialCNGA3-linked achromatopsiaMedDRA version: 20.0Level: LLTClassification code: 10000454Term: Achromatopsia Class: 10010331Therapeutic area: Diseases [C] - Eye Diseases [C11]CTIS2024-515553-17-00niversitaetsklinikum Tuebingen AöR14
Active, not recruiting
Phase 1
Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked AchromatopsiaAchromatopsiaNCT02610582STZ eyetrial13
Recruiting
Phase 1
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expressioretinitis pigmentosaMedDRA version: 20.0Level: PTClassification code: 10038914Term: Retinitis pigmentosa Class: 100000004850Therapeutic area: Diseases [C] - Eye Diseases [C11]CTIS2024-511687-90-00eyeDNA Therapeutics23
Recruiting
Phase 1/2
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression2024-511687-90-00eyeDNA Therapeutics23
Recruiting
Not Applicable
Single tube drainage versus double tube drainage for reduction of jaundice in cancers of bile duct.CTRI/2015/04/005725nil120