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Safety and Tolerability of hRPC in Retinitis Pigmentosa (hRPCRP)

This study is currently recruiting participants.
Verified April 2017 by ReNeuron Limited
Sponsor:
ClinicalTrials.gov Identifier:
NCT02464436
First Posted: June 8, 2015
Last Update Posted: April 25, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
ReNeuron Limited
  Purpose

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 one year to evaluate the safety and tolerability of hRPC Additional testing will seek to establish any preliminary efficacy from hRPC.


Condition Intervention Phase
Retinitis Pigmentosa Drug: hRPC Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)

Resource links provided by NLM:


Further study details as provided by ReNeuron Limited:

Primary Outcome Measures:
  • Safety as assessed by the absence of any grade 3 or greater AE considered "related" to hRPC [ Time Frame: 6 months ]
    The therapy will be considered safe in the absence of any grade 3 or greater adverse event (AE) considered "related" to hRPC.


Secondary Outcome Measures:
  • Efficacy (Visual function measure: change in visual acuity) [ Time Frame: 12 months ]
    Visual function measure: change in visual acuity

  • Efficacy (Visual function measure: change in visual field sensitivity) [ Time Frame: 12 months ]
    Visual function measure: change in visual field sensitivity

  • Efficacy (Transplant and host retina integrity and survival: retinal photography) [ Time Frame: 12 months ]
    Transplant and host retina integrity and survival: retinal photography

  • Efficacy (Transplant and host retina integrity and survival: fundus autofluorescence) [ Time Frame: 12 months ]
    Transplant and host retina integrity and survival: fundus autofluorescence

  • Efficacy (Transplant and host retina integrity and survival: spectral domain-OCT (SD-OCT)) [ Time Frame: 12 months ]
    Transplant and host retina integrity and survival: spectral domain-OCT (SD-OCT)


Estimated Enrollment: 15
Study Start Date: December 2015
Estimated Study Completion Date: April 2018
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: human retinal progenitor cells (hRPC)
Single subretinal administration of human retinal progenitor cells (hRPC)
Drug: hRPC
Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye.
Other Name: human retinal progenitor cells

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 1 year.

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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Adult male or female over 18 years of age.
  • Clinical diagnosis of RP, based upon one or more of the following: clinical features, electrophysiological measures and genetic testing, if available (genetic confirmation is not obligatory).
  • Best corrected Visual acuity of 20/200 or worse in the study eye (N.B. in patients with differing acuities between eyes, the worse eye will be enrolled as the study eye).
  • Medically able to undergo vitrectomy and subretinal injection, a surgery which may require general anesthesia.
  • Good general health as defined by:
  • Normal serum chemistry and hematology. Out of normal range laboratory findings deemed not clinical 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)
  • Female patients of child-bearing potential must use a reliable method of contraception (e.g. oral contraceptive and condom, intra-uterine device (IUD) and condom, diaphragm with spermicide and condom) during the study.
  • Females of childbearing potential must have a confirmed negative pregnancy test at time of treatment.
  • Males must be willing to use barrier and spermicide contraception during the study.
  • Ability to give written informed consent as evidenced by signature on the patient consent form, to communicate well with the investigator and to comply with the expectations of the study.

Exclusion Criteria:

  • The presence of ocular disease or ocular media opacity, which in the opinion of the investigator, precludes accurate evaluation during the study.
  • Prior vitrectomy in the study eye
  • Patients with a history of amblyopia will be excluded
  • High myopia (>6 diopters) in the study eye
  • Cataract surgery in the study within 3 months
  • Participation within 6 months in any clinical trial involving a drug or device treatment
  • No prior stem cell injections in any part of the body
  • Use of systemic immunosuppressive agents in the six months prior to treatment
  Contacts and Locations
Information from the National Library of Medicine

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


Contacts
Contact: Eric Pierce, MD 617-573-6060 ophthalmologyclinicalresearch@meei.harvard.edu

Locations
United States, Massachusetts
Massachusetts Eye and Ear Infirmary Recruiting
Boston, Massachusetts, United States, 02114
Contact: Eric Pierce, MD    617-573-6060    ophthalmologyclinicalresearch@meei.harvard.edu   
Sponsors and Collaborators
ReNeuron Limited
Investigators
Principal Investigator: Eric Pierce Massachusetts Eye and Ear Infirmary (MEEI)
  More Information

Responsible Party: ReNeuron Limited
ClinicalTrials.gov Identifier: NCT02464436     History of Changes
Other Study ID Numbers: RN03-CP-0001
First Submitted: May 18, 2015
First Posted: June 8, 2015
Last Update Posted: April 25, 2017
Last Verified: April 2017

Keywords provided by ReNeuron Limited:
RP

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Cone-Rod Dystrophies
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn