A phase 1/2, open-label, safety, tolerability and preliminary efficacy study of implantation into one eye of hESC-derived RPE (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)) in patients with retinitis pigmentosa due to monogenic mutation - STREAM

CECS/I-STEM0 个研究点目标入组 12 人开始时间: 2018年9月20日最近更新: 2024年6月24日

试验速览

暂无简介。

•- Documented diagnosis of retinis pigmentosa based on a genetic test confirming the presence of a monogenic mutation that affects a gene involved in the visual signalling process specifically at the level of RPEs, namely RPE65 or LRAT, or MerTK
•- 18 years old = Age = 65 years old
•- For patient of the first cohort:
•o Visual acuity = 20/200 in the best eye (legally blind)
•- For patient of the second cohort:
•o Visual acuity > 20/200 in the worst eye
•o Visible photoreceptor outer nuclear layer (ONL) on a spectral domain optical coherence tomography (OCT) scan
•- For the two cohorts:
•o Negative serum pregnancy test in women of childbearing potential (a woman who is two years post-menopausal confirmed by a physician or surgically sterile is not considered to be of childbearing potential)
•o Female patients of childbearing potential (if sexually active), committed to use two methods of contraception starting from the enrollment, during Mycophenolate Mofetil (MMF) treatment and for 6 weeks after the last dose of MMF
•o Sexually active men (including vasectomized men) committed to use condoms during from the first day of MMF treatment and for at least 90 days after cessation of treatment
•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 12
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•- Patient unable or unwilling to comply with the protocol requirements
•- History of allergy or sensitivity to one of the products used during the study
•- Prior treatment with a gene or cell therapy product
•- Patients with chronic hepatitis B or C, i.e. positive hepatitis B surface antigen or hepatitis C RNA viral load positive
•- Patients infected with Human immunodeficiency virus (HIV)
•- Pregnancy or breastfeeding
•- Presence of any ocular disease or ocular media opacity which in the opinion of the investigator precludes accurate evaluation
•- Participation in another drug or device clinical study within last 6 months prior to baseline
•- Patients known to be affected by pathologies for which the symptoms or associated treatments can alter the visual function and/or affect the retina
•- Systemic corticosteroid therapy or other immunosuppressive / immunomodulating or anti-retroviral drugs within 2 months prior to baseline
•- Patients with a contraindication to immunosuppressive/immunomodulating therapy (MMF)
•- Acute illness or infection within 4 weeks of the anticipated administration of study medication which may interfere with study assessments and immunosuppressive/immunomodulating therapy
•- Any other condition or history that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study

发起方

CECS/I-STEM