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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
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Drug: hRPC Phase 1 Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
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)
Study Start Date : December 2015
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : July 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

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
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Contact: Jason Comander, MD 617-573-6060 ophthalmologyclinicalresearch@meei.harvard.edu
Contact: Vince Holmes Vince-Holmes@reneuron.com

Locations
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United States, Arizona
Retinal Research Institute Recruiting
Phoenix, Arizona, United States, 85053
Contact: Pravin Dugel, MD    602-222-2221    research@retinalconsultantsaz.com   
United States, Massachusetts
Massachusetts Eye and Ear Infirmary Recruiting
Boston, Massachusetts, United States, 02114
Contact: Jason Comander, MD    617-573-6060    ophthalmologyclinicalresearch@meei.harvard.edu   
Sponsors and Collaborators
ReNeuron Limited
Investigators
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Principal Investigator: Jason Comander, MD Massachusetts Eye and Ear Infirmary (MEEI)
Study Director: Vince Holmes ReNeuron Limited

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Responsible Party: ReNeuron Limited
ClinicalTrials.gov Identifier: NCT02464436     History of Changes
Other Study ID Numbers: RN03-CP-0001
First Posted: June 8, 2015    Key Record Dates
Last Update Posted: February 21, 2019
Last Verified: February 2019
Keywords provided by ReNeuron Limited:
RP
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn