Safety and Tolerability of hRPC in Retinitis Pigmentosa

• Register: ClinicalTrials.gov
• Last refreshed on: 18 July 2023
• Main ID: NCT02464436
• Date of registration: 18/05/2015
• Prospective Registration: Yes
• Primary sponsor: ReNeuron Limited
• Public title: Safety and Tolerability of hRPC in Retinitis Pigmentosa hRPCRP
• Scientific 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)
• Date of first enrolment: December 2015
• Target sample size: 29
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02464436
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Spain, United Kingdom, United States

Contacts

• Name: Vince Holmes
• Affiliation: ReNeuron Limited

• Name: Jason Comander, MD
• Affiliation: Massachusetts Eye and Ear Infirmary (MEEI)

Key inclusion & exclusion 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 6-8, and Best Corrected ETDRS visual acuity of 8 letters (approximately
20/800) to 68 letters (approximately 20/50) for cohorts 9 and on.

5. Be able to complete the entire microperimetry test, and demonstrate adequate fixation
and consistency between baseline readings such that the accuracy of both baseline and
follow on testing should enable the detection of clinically significant changes in
retinal sensitivity.

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

7. 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 as
well as medically fit to undergo a short perioperative course of systemic
corticosteroid therapy

- 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).

8. Females of childbearing potential must have a confirmed negative pregnancy test at
Visits 1 and 3; and be willing to use highly effective 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.

9. 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.

10. 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. Exhibits a difference in ETDRS BCVA of 15 letters of more in either eye between any of
the baseline visits.

2. Exhibits a difference in ETDRS BCVA of 20 or more letters between eyes at the time of
any of the screening or baseline visits attributed to asymmetry in the progression of
RP.

3. 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.

4. 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.Specifically,
subjects in whom significant pre-existing vitreoretinal pathology might influence
visual acuity outcomes should be excluded

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

6. Prior vitrectomy in the study eye.

7. A history of amblyopia in the study eye.

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

9. 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.

10. Participation in any clinical study involving an investigational drug or device within
6 months prior to treatment or 5 half-lives of the drug (whichever is longer) prior to
initiation of treatment

11. 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).

12. Use of systemic immunosuppressive agents (e.g. corticosteroid) in the 6 months prior
to treatment or 5 half-lives of the drug (whichever is longer) prior to initiation of
treatment (Note: inhaled, intranasal, and/or topical dermatologic steroids are
allowed)

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

m) Known hypersensitivity to any of ingredients of the excipient.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Retinitis Pigmentosa

Intervention(s)

Drug: hRPC

Primary Outcome(s)

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]

Secondary Outcome(s)

Safety (Change in retinal sensitivity in the area overlying the implanted hRPC as compared with untreated retina) [Time Frame: 24 months]

Safety (Anatomical endpoint relating to retinal function in implant location - Fundus autofluorescence) [Time Frame: 24 months]

Safety (Anatomical endpoint relating to retinal function in implant location - Spectral domain-OCT) [Time Frame: 24 months]

Safety (Anatomical endpoint relating to retinal function in implant location - Color Fundus Photography) [Time Frame: 24 months]

Safety (Visual function measure: change in visual acuity) [Time Frame: 24 months]

Safety (Visual function measure: change in visual field: Goldmann visual field, microperimetry and FST) [Time Frame: 24 months]

Secondary ID(s)

RN03-CP-0001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors