Safety and Tolerability of hRPC in Retinitis Pigmentosa (hRPCRP)
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|ClinicalTrials.gov Identifier: NCT02464436|
Recruitment Status : Recruiting
First Posted : June 8, 2015
Last Update Posted : February 21, 2019
hRPC is a cell therapy for retinitis pigmentosa. This is a first-in-human, dose escalation study in which participants with retinitis pigmentosa will receive a single subretinal injection of hRPC cells in one eye to evaluate safety and tolerability.
Participants will be followed for two years to evaluate the safety and tolerability of hRPC Additional testing will seek to establish any preliminary efficacy from hRPC.
|Condition or disease||Intervention/treatment||Phase|
|Retinitis Pigmentosa||Drug: hRPC||Phase 1 Phase 2|
This is a first-in-human open label phase I/II dose-escalation study in which participants with retinitis pigmentosa will receive a single uni-ocular subretinal implantation of one of three doses of hRPC.
Treated eyes will be carefully monitored for any ocular or systemic adverse events for 2 years.
Testing will comprise a series of detailed ophthalmic examinations and imaging together with blood testing and systemic evaluations, as necessary.
Ophthalmic testing will also be evaluated for any preliminary efficacy signal.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||21 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||First-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Patients With Retinitis Pigmentosa (RP)|
|Study Start Date :||December 2015|
|Estimated Primary Completion Date :||January 2020|
|Estimated Study Completion Date :||July 2021|
Experimental: human retinal progenitor cells (hRPC)
Single subretinal administration of human retinal progenitor cells (hRPC)
Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye.
Other Name: human retinal progenitor cells
- Safety over the six months after treatment as assessed by the incidence of treatment emergent adverse events (TEAEs) and changes from baseline in other safety parameters. [ Time Frame: 6 months ]Safety measures will be assessed by review of important events, including but not limited to inflammation, complications of the surgical procedure and worsening of vision.
- Safety (Visual function measure: change in visual acuity) [ Time Frame: 24 months ]A summary of the ETDRS +/- BRVT BCVA letter score and the change from baseline to end of study in the treated eye presented by treatment group.
- Safety (Visual function measure: change in visual field: Goldmann visual field, microperimetry and FST) [ Time Frame: 24 months ]A summary of the perimetry and change from baseline to end of study in the treated eye presented by treatment group.
- Safety (Change in retinal sensitivity in the area overlying the implanted hRPC as compared with untreated retina) [ Time Frame: 24 months ]ERG results and change from baseline to end of study summarized descriptively and presented by treatment group.
- Safety (Anatomical endpoint relating to retinal function in implant location - Color Fundus Photography) [ Time Frame: 24 months ]A qualitative description of the change in retinal appearance of treated and untreated retinal area in the treated eye from baseline to end of study presented by treatment group.
- Safety (Anatomical endpoint relating to retinal function in implant location - Fundus autofluorescence) [ Time Frame: 24 months ]A qualitative description of the change in retinal appearance of treated and untreated retinal area in the treated eye from baseline to end of study presented by treatment group.
- Safety (Anatomical endpoint relating to retinal function in implant location - Spectral domain-OCT) [ Time Frame: 24 months ]Summary of the overall retinal thickness, outer nuclear layer thickness and ellipsoid zone measurement and change from baseline to the end of study of treated retina compared with untreated retina in the treated eye by treatment group.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02464436
|Contact: Jason Comander, MDfirstname.lastname@example.org|
|Contact: Vince Holmes||Vince-Holmes@reneuron.com|
|United States, Arizona|
|Retinal Research Institute||Recruiting|
|Phoenix, Arizona, United States, 85053|
|Contact: Pravin Dugel, MD 602-222-2221 email@example.com|
|United States, Massachusetts|
|Massachusetts Eye and Ear Infirmary||Recruiting|
|Boston, Massachusetts, United States, 02114|
|Contact: Jason Comander, MD 617-573-6060 firstname.lastname@example.org|
|Principal Investigator:||Jason Comander, MD||Massachusetts Eye and Ear Infirmary (MEEI)|
|Study Director:||Vince Holmes||ReNeuron Limited|