Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mut… (NCT03963154) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation
France7 participantsStarted 2019-08-19
Plain-language summary
Phase I/II 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.
Study non randomized single group assignment consisting in 2 sequential cohorts of patients:
* First cohort of 2 patients with very advanced loss of visual acuity (legally blind)
* Second cohort of 10 patients with less advanced loss of visual acuity:
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion Criteria:
* Documented diagnosis of retinitis 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:
* Visual acuity ≤ 20/200 in the best eye (legally blind)
\- For patient of the second cohort:
* 20/63 \> Visual acuity \> 20/200 in the worst eye And
* Visible photoreceptor outer nuclear layer (ONL) on a spectral domain optical coherence tomography (OCT) scan
For the two cohorts:
* 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)
* 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
* 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
* Signed informed consents by the patient or legal guardian(s). For patients unable to give consent, authorization to participate to the study will be collected close to their legally authorized representative
* Affiliated to or a beneficiary of a health care syst…