Safety and Tolerability of hRPC in Retinitis Pigmentosa

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)

Sponsors

Lead Sponsor: ReNeuron Limited

Source ReNeuron Limited
Brief Summary

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.

Detailed Description

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.

Overall Status Recruiting
Start Date December 2015
Completion Date July 2021
Primary Completion Date January 2020
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
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. 6 months
Secondary Outcome
Measure Time Frame
Safety (Visual function measure: change in visual acuity) 24 months
Safety (Visual function measure: change in visual field: Goldmann visual field, microperimetry and FST) 24 months
Safety (Change in retinal sensitivity in the area overlying the implanted hRPC as compared with untreated retina) 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Color Fundus Photography) 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Fundus autofluorescence) 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Spectral domain-OCT) 24 months
Enrollment 21
Condition
Intervention

Intervention Type: Drug

Intervention Name: hRPC

Description: Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye.

Arm Group Label: human retinal progenitor cells (hRPC)

Other Name: human retinal progenitor cells

Eligibility

Criteria:

Inclusion Criteria:

1. Have ability to give written informed consent as evidenced by signature on the subject consent form.

2. Be adult male or female over 18 years of age.

3. Have clinical diagnosis of RP, based upon one or more of the following: clinical features, medical imaging, electrophysiological measures and genetic testing, if available. Genetic confirmation is not obligatory.

4. Have Best Corrected ETDRS visual acuity of 35 letters or less (approximately 20/200 or worse) in the study eye for cohorts 1-5; have Best Corrected ETDRS visual acuity of 63 letters (approximately 20/63) to 36letters (approximately 20/200) in the study eye for cohorts 6 and on.

5. Be medically able to undergo vitrectomy and subretinal injection.

6. Have good general health as defined by:

- Normal serum chemistry and hematology. Out of normal range laboratory findings deemed not clinically significant are acceptable.

- No history of malignancy, except non-melanoma skin cancer; pre-malignant conditions and cancer in situ.

- Negative serology for human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV).

- Medically fit enough to undertake surgery which may require general anesthesia.

- Free of any other systemic condition that in the opinion of the Investigator may have an impact on the safety of the subject, conduct of study procedures, or integrity of study data (e.g. severe cardiovascular or respiratory disease; poorly controlled diabetes; significant psychiatric impairment).

7. Females of childbearing potential must have a confirmed negative pregnancy test at Visits 1 and 2; and be willing to use reliable method of contraception (e.g. oral contraceptive and condom, intra-uterine device (IUD) and condom, diaphragm with spermicide and condom) for the duration of this study.

8. Males must be willing to use a reliable method of contraception (e.g. barrier and spermicide) for the duration of this study; unless have been surgically sterilized with confirmed azoospermia.

9. Be willing and able to attend all scheduled clinical assessments, ability to communicate well with the Investigator and to comply with the expectations of the study.

Exclusion Criteria:

1. Presence of ocular disease or ocular media opacity in the study eye, which in the opinion of the Investigator, will preclude an accurate evaluation at any time during the study.

2. History of any retinal and/or macular disease other than RP (e.g. retinal detachment) that in the opinion of the Investigator may have an impact on the safety of the subject, conduct of study procedures, or integrity of study data.

3. Active ocular infection or inflammation, or any history of intraocular inflammation, that would expose subject to risk during or following surgery.

4. Prior vitrectomy in the study eye.

5. A history of amblyopia in the study eye.

6. High myopia (>6 diopters) in the study eye.

7. Cataract surgery in the study eye or ocular surgery in either eye (which in the opinion of the investigator may have an impact on patient safety or the integrity of data from the study eye) during the study or within 3 months prior to treatment.

8. Participation in any clinical study involving an investigational drug or device within 6 months prior to treatment.

9. Prior stem cell administration or injections to any part of the body (subjects who have received autologous bone marrow stem cell transplant will be eligible).

10. Use of systemic immunosuppressive agents (e.g. corticosteroid) in the 6 months prior to treatment (Note: inhaled, intranasal, and/or topical dermatologic steroids are allowed).

11. (For females) Be breastfeeding or planning a pregnancy.

12. Known hypersensitivity to any of ingredients of the excipient.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Overall Contact

Last Name: Jason Comander, MD

Phone: 617-573-6060

Email: [email protected]

Location
Facility: Status: Contact:
Retinal Research Institute | Phoenix, Arizona, 85053, United States Recruiting Pravin Dugel, MD 602-222-2221 [email protected]
Massachusetts Eye and Ear Infirmary | Boston, Massachusetts, 02114, United States Recruiting Jason Comander, MD 617-573-6060 [email protected]
Location Countries

United States

Verification Date

February 2019

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: human retinal progenitor cells (hRPC)

Type: Experimental

Description: Single subretinal administration of human retinal progenitor cells (hRPC)

Acronym hRPCRP
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov