Retinitis PigmentosaPhase 2NCT03073733
Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa
A Phase 2 study testing human retinal progenitor cells and Mock injection in people with Retinitis Pigmentosa, run by jCyte, Inc at 3 sites in United States.
Trial summary
This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP. 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?
- human retinal progenitor cells and Mock injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 84 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Mar 2017to 13 Nov 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Clinical diagnosis of RP confirmed by ERG and willing to consent to mutation typing, if not already done Best corrected visual acuity (BCVA) 20/80 or worse and no worse than 20/800 Adequate organ function and negative infectious disease screen Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study
Who cannot join
- Eye disease other than RP that impairs visual function Pseudo-RP, cancer-associated retinopathies History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents Known allergy to penicillin or streptomycin Treatment with corticosteroids or any investigational or neuroprotectant therapy within 90 days of enrollment Cataract surgery within 3 months prior to enrollment
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Ophthalmic Consultants of Boston
Boston, Massachusetts, 02114
Gavin Herbert Eye Inst, Univ Cal Irvine
Irvine, California, 92697
Retina-Vitreous Associates Medical Group
Los Angeles, California, 90074
About this study
Official title: A Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP.
Read the detailed description
There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate. The rate of deterioration of vision varies from person to person, with most people with RP legally blind by age 40. Preclinical studies demonstrated that transplantation of retinal progenitor cells into the eye can result in both photoreceptor replacement and significant slowing of host photoreceptor loss. Thus, the primary goal of this therapy is to preserve, and potentially improve, vision by intervening in the disease at a time when dystrophic host photoreceptors can be protected and reactivated. Based on the demonstration of acceptable safety and tolerability in a phase 1/2a study, this phase 2b study is designed as a controlled comparison of the changes in visual function and functional vision in subjects who receive a single jCell injection in comparison to a comparable sham-treated control group of subjects with RP.
- Conditions
- Retinitis Pigmentosa
- Being tested
- human retinal progenitor cells, Mock injection
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
human retinal progenitor cellsBiological
Also called jCell
live suspension of 3.0 or 6.0 x 10e6 human retinal progenitor cells (hRPC) suspended in clinical grade medium injected intravitreally under local anesthesia
Mock injectionOther
pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection
| Study group | Role | What they receive |
|---|---|---|
| Test (jCell injection) dose level 1 | Experimental | single intravitreal injection of 3.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye Receives: Biological: human retinal progenitor cells |
| Sham treated Control | Other | a mock injection will be performed on the eye with the poorest vision in each Control subject (designated as the "study eye") Receives: Other: Mock injection |
| test (jCell injection) dose level 2 | Experimental | single intravitreal injection of 6.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye Receives: Biological: human retinal progenitor cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best Corrected Visual Acuity (BCVA)
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in ITT population. A letter score is used to compare change over time, with a higher number of letters representing better visual function, and a lower number of letters representing worse visual function. For example, 85 letters is equivalent to 20/20 visual acuity and 5 letters is equivalent to 20/800 visual acuity. A change value is derived for each subject by taking the letter score at 12 months and subtracting the letter score at baseline. A mean of all change values is then calculated for each arm.
Measured: 12 months
Secondary outcomes (5)
Contrast Sensitivity (CS) at 1.0 CPD
Assessment of the ability to detect changes in shades of grey, as measured with a vertical striped pattern that varies in width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each pattern width. A CS curve is created using a minimum of three pattern widths, one at the subject's peak or highest sensitivity, and then one larger and smaller pattern width on either side of the peak. For example, if a subject's CS peak appears to be at 2.0 CPD, then additional testing occurs at 1.0 and 4.0 CPD. RP patients have suppressed CS curves and the most common pattern widths are at 0.5, 1.0, 2.0, and 4.0 CPD. Severely impaired subjects (e.g., BCVA < 20/400) usually have flat curves with low values (e.g., 1.28), while in mildly impaired RP subjects the values can be higher (e.g., 7.12), but are rarely near normal. These data represent mean change in CS from baseline to 12 months, with greater values representing greater improvement in visual function.
Measured: 12 months
Kinetic Visual Field (KVF)
Mean change in total area (degrees squared) of all islands of vision from baseline to 12 months.
Measured: 12 months
Low Luminance Mobility Test (LLMT)
The LLMT identifies the performance of RP patients as they walk along an indoor pathway of arrows and obstacles at varying lighting levels. The Critical Illumination Level (CIL) is the light level below which the patient has a markedly slower pace and more errors than all light levels above (brighter than) that point. The LLMT uses light levels that go from very dim (0.12 lux) to a bright indoor room (500 lux), with evenly spaced increments that increase light by doubling the brightness of the room from the prior level. These evenly spaced light levels have been converted to a scale score to enable easier calculation of change scores. The dimmest light level of 0 lux (completely dark room) corresponds to a scale score of 13, whereas the brightest light level of 500 lux corresponds to a scale score of 0. A positive scale score change from baseline to 12 months represents improvement in low light vision, whereas a negative scale score change represents a decline in low light vision.
Measured: 12 months
Low Vision Functional Questionnaire (Visual Ability)
The VA LV VFQ-48 (VFQ) is used to capture changes in patients' self-reporting of their difficulty with reading, mobility and performing other daily living activities affected by visual impairment. There are 4 scales on the VFQ including Visual Information, Reading, Visual Motor, and Mobility. A fifth value, Visual Ability, is an aggregate score of the 4 scales, measured in units called logits, and is calculated for each person based on item weighting using Raasch analysis. Visual Ability is used broadly to represent changes in subject-reported outcomes from visit to visit. Higher positive values on the Visual Ability score represent better function and less impairment, whereas lower or negatives scores represent worse function or more impairment. Change in the Visual Ability scale on the VFQ is calculated by taking a subject's score at 12 months and subtracting from it the baseline score.
Measured: 12 months
Safety of Intravitreal Injection of Retinal Progenitor Cells (RPC)
Assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2017
- Study start1 Mar 2017
- Primary completion13 Nov 2020
- Study completion13 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 84 participants (actual)
Sponsor and investigators
- Lead sponsor
- jCyte, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Sponsor
- Investigators
- Mitul Mehta, MD, Principal investigator, UCIDavid Liao, MD, Principal investigator, RVAAnthony Joseph, MD, Principal investigator, OCB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03073733
- Sponsor's protocol ID
- JC-02
- Status last verified
- Jun 2024
- First submitted
- 2 Mar 2017
- Last updated
- 18 Jun 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
- ClinicalTrials.gov ID
- NCT03073733
- Acronym / sponsor ID
- JC-02
- Conditions
- Retinitis Pigmentosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 84 participants (actual)
- Lead sponsor
- jCyte, IncIndustry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Study start
- 1 Mar 2017 (actual)
- Primary completion
- 13 Nov 2020 (actual)
- Study completion
- 13 Nov 2020 (actual)
- First posted
- 8 Mar 2017 (actual)
- Results
- Posted 21 Jan 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP.
Detailed description
There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate. The rate of deterioration of vision varies from person to person, with most people with RP legally blind by age 40. Preclinical studies demonstrated that transplantation of retinal progenitor cells into the eye can result in both photoreceptor replacement and significant slowing of host photoreceptor loss. Thus, the primary goal of this therapy is to preserve, and potentially improve, vision by intervening in the disease at a time when dystrophic host photoreceptors can be protected and reactivated. Based on the demonstration of acceptable safety and tolerability in a phase 1/2a study, this phase 2b study is designed as a controlled comparison of the changes in visual function and functional vision in subjects who receive a single jCell injection in comparison to a comparable sham-treated control group of subjects with RP.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Test (jCell injection) dose level 1 | Experimental | Biological: human retinal progenitor cellssingle intravitreal injection of 3.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye |
| Sham treated Control | Other | Other: Mock injectiona mock injection will be performed on the eye with the poorest vision in each Control subject (designated as the "study eye") |
| test (jCell injection) dose level 2 | Experimental | Biological: human retinal progenitor cellssingle intravitreal injection of 6.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Clinical diagnosis of RP confirmed by ERG and willing to consent to mutation typing, if not already done Best corrected visual acuity (BCVA) 20/80 or worse and no worse than 20/800 Adequate organ function and negative infectious disease screen Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study
Key exclusion criteria (1 listed)
- Eye disease other than RP that impairs visual function Pseudo-RP, cancer-associated retinopathies History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents Known allergy to penicillin or streptomycin Treatment with corticosteroids or any investigational or neuroprotectant therapy within 90 days of enrollment Cataract surgery within 3 months prior to enrollment
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 |
|---|---|---|
| human retinal progenitor cellsAlso: jCelllive suspension of 3.0 or 6.0 x 10e6 human retinal progenitor cells (hRPC) suspended in clinical grade medium injected intravitreally under local anesthesia | Biological | Test (jCell injection) dose level 1, test (jCell injection) dose level 2 |
| Mock injectionpressing the hub of a syringe with no needle against the eye to mimic intravitreal injection | Other | Sham treated Control |
Primary outcome
Best Corrected Visual Acuity (BCVA)
Time frame: 12 months
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in ITT population. A letter score is used to compare change over time, with a higher number of letters representing better visual function, and a lower number of letters representing worse visual function. For example, 85 letters is equivalent to 20/20 visual acuity and 5 letters is equivalent to 20/800 visual acuity. A change value is derived for each subject by taking the letter score at 12 months and subtracting the letter score at baseline. A mean of all change values is then calculated for each arm.
5 secondary outcomes
Contrast Sensitivity (CS) at 1.0 CPD
Time frame: 12 months
Kinetic Visual Field (KVF)
Time frame: 12 months
Low Luminance Mobility Test (LLMT)
Time frame: 12 months
Low Vision Functional Questionnaire (Visual Ability)
Time frame: 12 months
Safety of Intravitreal Injection of Retinal Progenitor Cells (RPC)
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Mitul Mehta, MD principal investigator
UCI
David Liao, MD principal investigator
RVA
Anthony Joseph, MD principal investigator
OCB
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing human retinal progenitor cells and Mock injection in people with Retinitis Pigmentosa, run by jCyte, Inc at 3 sites in United States.
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 1 inclusion and 1 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: finished, as of the sponsor's last update on 18 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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 phase mean?
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?
Yes. Results are posted in the registry (first posted 21 Jan 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Best Corrected Visual Acuity (BCVA) (time frame: 12 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 18 Jun 2024.