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Safety and Tolerability of hRPC in Retinitis Pigmentosa (hRPCRP)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02464436
Recruitment Status : Active, not recruiting
First Posted : June 8, 2015
Last Update Posted : January 20, 2022
Sponsor:
Information provided by (Responsible Party):
ReNeuron Limited

Tracking Information
First Submitted Date  ICMJE May 18, 2015
First Posted Date  ICMJE June 8, 2015
Last Update Posted Date January 20, 2022
Study Start Date  ICMJE December 2015
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 19, 2019)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: June 4, 2015)
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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 19, 2019)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 4, 2015)
  • 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)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Tolerability of hRPC in Retinitis Pigmentosa
Official Title  ICMJE 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)
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Retinitis Pigmentosa
Intervention  ICMJE Drug: hRPC
Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye.
Other Name: human retinal progenitor cells
Study Arms  ICMJE Experimental: human retinal progenitor cells (hRPC)
Single subretinal administration of human retinal progenitor cells (hRPC)
Intervention: Drug: hRPC
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 19, 2022)
29
Original Estimated Enrollment  ICMJE
 (submitted: June 4, 2015)
15
Estimated Study Completion Date  ICMJE December 2023
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02464436
Other Study ID Numbers  ICMJE RN03-CP-0001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party ReNeuron Limited
Original Responsible Party Same as current
Current Study Sponsor  ICMJE ReNeuron Limited
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Jason Comander, MD Massachusetts Eye and Ear Infirmary (MEEI)
Study Director: Vince Holmes ReNeuron Limited
PRS Account ReNeuron Limited
Verification Date January 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP