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Gene Therapy Trial Report

Summary

PDE6A Gene Therapy for Retinitis Pigmentosa


NCTID NCT04611503 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Retinitis Pigmentosa
Disease Ontology Term DOID:0110379
Compound Name RAAV.hPDE6A
Sponsor STZ eyetrial
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 9 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant PDE6A
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 1E9 vg
Dose 2 5E9 vg
Dose 3 1E10 vg
Dose 4 5E10 vg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-05-20
Completion Date 2027-07
Last Update 2024-04-18

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * clinical diagnosis of retinitis pigmentosa * confirmed mutation in PDE6A gene * ≥ 18 years of age * visual acuity ≥ 20/400 * no infection with Human Immundeficiency Virus (HIV) * negative pregnancy test in women with childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential) * Male patients must agree to use condoms during the first 6 months post treatment. * Female patients of childbearing potential must agree to use an effective method of birth control during the first 6 months post treatment. * ability to understand and willingness to consent to study protocol Exclusion Criteria: Ocular (study eye \& fellow eye) * additional interfering ocular conditions with impact on study results (e.g. ocular opacity and advanced cataract, uveitis, amblyopia) * recent (6 months) ocular surgery, intravitreal or subretinal implantation of a medical device * disease causing mutations in another known retinitis pigmentosa gene * ocular infection with herpes simplex virus in medical history * history of ocular malignancies * disorders of the internal retina (e.g. retinal detachment in the patients history) * glaucoma defined as damage of the optic nerve * vascular retinal occlusion * diabetic patients suffering from retinopathy and/or macula edema * any other retinopathy due to other diseases e.g. (but not limited to) arterial hypertension, trauma or acquired inflammatory diseases (uveitis serology), contraindication to pharmacological mydriasis (e.g. history of angle block glaucoma) * absence of visual function on the contralateral eye Systemic * systemic conditions (e.g. coronary heart disease, autoimmune disorders) which may affect study participation or outcome measures * History of poorly controlled Diabetes Mellitus type 1 or type 2 * systemic illness or medically relevant abnormal laboratory values in blood analysis including renal and hepatic functions at inclusion * patients treated with oral corticoids within 14 days prior inclusion * current or recent participation in other study/or administration of biologic agent within the last three months * known sensitivity to any compound used in the study * contraindications to systemic immunosuppression * contraindications in view of the planned surgery (e.g. but not limited to anaemia Hb\<10g/dl, coagulopathy with PT/PTT \>1,5 fold upper limit, hypertension with values above 180 mmHg systolic and 110 mmHg diastolic) including intolerance and contraindications to general anaesthesia * intolerance to contrast agents used for diagnostic methods like angiography with fluoresceine or indocyanine green (e.g. but not limited to hyperthyroidism, hepatic insufficiency) * subject/partner of childbearing potential unwilling to use adequate contraception for four months * nursing or pregnant women * any other cause that, in the investigator's opinion, renders potential subjects not suitable for the study
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations Germany

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links