Retinitis PigmentosaPhase 1/2hRPCRPNCT02464436
Safety and Tolerability of hRPC in Retinitis Pigmentosa
A Phase 1/2 study testing hRPC in people with Retinitis Pigmentosa, run by ReNeuron Limited at 5 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- hRPC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 5 sitesin 3 countries
- Study dates
- Dec 2015to Dec 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have ability to give written informed consent as evidenced by signature on the subject consent form.
- Be adult male or female over 18 years of age.
- 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.
- 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.
- 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.
- Be medically able to undergo vitrectomy and subretinal injection.
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- 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).
- 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.
- 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.
- 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.
Who cannot join
- Exhibits a difference in ETDRS BCVA of 15 letters of more in either eye between any of the baseline visits.
- 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.
- 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.
- 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
- Active ocular infection or inflammation, or any history of intraocular inflammation, that would expose subject to risk during or following surgery.
- Prior vitrectomy in the study eye.
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- A history of amblyopia in the study eye.
- High myopia (>6 diopters) in the study eye.
- 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.
- 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
- 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).
- 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)
- (For females) Be breastfeeding or planning a pregnancy.
- Known hypersensitivity to any of ingredients of the excipient.
Trial locations and contacts
5 sites in 3 countries.
5 sites in 3 countries
United States3 sites
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114
Retinal Research Institute
Phoenix, Arizona, 85053
Oregon Health and Science University
Portland, Oregon, 97239
About this study
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)
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.
Read the 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.
- Conditions
- Retinitis Pigmentosa
- Being tested
- hRPC
- Keywords
- RP
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hRPCDrug
Also called human retinal progenitor cells
Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye.
| Study group | Role | What they receive |
|---|---|---|
| human retinal progenitor cells (hRPC) | Experimental | Single subretinal administration of human retinal progenitor cells (hRPC) Receives: Drug: hRPC |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
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.
Measured: 6 months
Secondary outcomes (6)
Safety (Visual function measure: change in visual acuity)
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.
Measured: 24 months
Safety (Visual function measure: change in visual field: Goldmann visual field, microperimetry and FST)
A summary of the perimetry and change from baseline to end of study in the treated eye presented by treatment group.
Measured: 24 months
Safety (Change in retinal sensitivity in the area overlying the implanted hRPC as compared with untreated retina)
ERG results and change from baseline to end of study summarized descriptively and presented by treatment group.
Measured: 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Color Fundus Photography)
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.
Measured: 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Fundus autofluorescence)
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.
Measured: 24 months
Safety (Anatomical endpoint relating to retinal function in implant location - Spectral domain-OCT)
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.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2015 (estimated)
- Study startDec 2015
- Primary completionJun 2022
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- ReNeuron Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jason Comander, MD, Principal investigator, Massachusetts Eye and Ear Infirmary (MEEI)Vince Holmes, Study director, ReNeuron Limited
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02464436
- Sponsor's protocol ID
- RN03-CP-0001
- Acronym
- hRPCRP
- Status last verified
- Jul 2023
- First submitted
- 18 May 2015
- Last updated
- 6 Jul 2023
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02464436
- Acronym / sponsor ID
- hRPCRP / RN03-CP-0001
- Conditions
- Retinitis Pigmentosa
- Keywords
- RP
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 29 participants (actual)
- Lead sponsor
- ReNeuron LimitedIndustry (pharma, biotech, devices)
- Study start
- Dec 2015
- Primary completion
- Jun 2022 (actual)
- Study completion
- Dec 2023 (estimated)
- First posted
- 8 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| human retinal progenitor cells (hRPC) | Experimental | Drug: hRPCSingle subretinal administration of human retinal progenitor cells (hRPC) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Have ability to give written informed consent as evidenced by signature on the subject consent form.
- Be adult male or female over 18 years of age.
- 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.
- 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.
- 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.
Key exclusion criteria (14 listed)
- Exhibits a difference in ETDRS BCVA of 15 letters of more in either eye between any of the baseline visits.
- 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.
- 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.
- 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
- Active ocular infection or inflammation, or any history of intraocular inflammation, that would expose subject to risk during or following surgery.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hRPCAlso: human retinal progenitor cellsParticipants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye. | Drug | human retinal progenitor cells (hRPC) |
Primary outcome
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.
6 secondary outcomes
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
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 - Color Fundus Photography)
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jason Comander, MD principal investigator
Massachusetts Eye and Ear Infirmary (MEEI)
Vince Holmes study director
ReNeuron Limited
5 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing hRPC in people with Retinitis Pigmentosa, run by ReNeuron Limited at 5 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2023.