Retinitis PigmentosaPhase 2NCT04604899
Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
A Phase 2 study testing human retinal progenitor cells in people with Retinitis Pigmentosa, run by jCyte, Inc at 3 sites in United States.
Trial summary
The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell. 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
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Dec 2020to 22 Mar 2022
- 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
- Willing to give written informed consent, able to make the required study visits and follow study protocol instructions.
- Completed the 12 months of follow up in the subject's most recent jCell study and did not withdraw from the study for any reason.
- Adequate organ function:
- blood counts (hematocrit, Hgb, WBC, platelets and differential) within normal range, or if outside of normal range, not clinically significant as judged by the investigator
- liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper limit of the normal range
- total bilirubin ≤1.5 times the upper limit of the normal range
- renal function: serum creatinine ≤1.25 times the upper limit of the normal range
- A female patient of childbearing potential (not surgically sterilized and less than one year postmenopausal) must have a negative pregnancy test (urine human chorionic gonadotropin) at entry (prior to injection) and must have used medically accepted contraception for at least one month prior to treatment. Women of childbearing potential and men must be advised to use a medically accepted method of contraception for at least 12 months following treatment.
Who cannot join
- Malignancy, end-stage major organ disease (heart failure, significant arrhythmias, stroke or transient ischemic attacks, diabetes, immunosuppressive or autoimmune state, major psychiatric disorder, epilepsy, thyroid disease, COPD, renal failure, or any chronic systemic disease requiring continuous treatment with systemic steroids, anticoagulants or immunosuppressive agents.
- History of eye disease other than RP that impairs visual function, including retinal vascular disease, elevated intraocular pressure/glaucoma, severe posterior uveitis, clinically significant macular edema, media opacity precluding visual exam, amblyopia and/or longstanding constant strabismus, as well as patients who require other intravitreal therapies
- Allergy to penicillin or streptomycin.
- Adverse reaction to DMSO.
- Unable or unwilling to undergo pupil dilation, topical anesthesia or any protocol-required procedure.
- Women who are nursing or who are planning to nurse during the 12 months that would follow study treatment.
Show all 9Show fewer
- Any circumstance that in the opinion of the investigator, would interfere with participation in, or compliance with the study protocol
- Treatment with corticosteroids (systemic, periocular or intravitreal) or any other non-approved, experimental, investigational or neuroprotectant therapy (systemic, topical, intravitreal) in either eye within 90 days of planned second injection.
- Cataract surgery within three months prior to treatment or anticipated to need cataract surgery within a year of treatment
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 Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell.
Read the detailed description
This is a prospective, multi-center, single arm, Phase 2 study of human retinal progenitor cells (jCell) for the treatment of retinitis pigmentosa (RP). The study will include only subjects previously treated with jCell.
To assess reinjection of a previously treated eye, subjects who have previously been treated with jCell and desire a second treatment in the same eye will be enrolled. Subjects must have completed at least 12 months of follow up since the prior injection of jCell. Subjects who have had both eyes previously treated with jCell will only have one eye retreated; the eye to be retreated will preferably be the better seeing eye, but exceptions may be made by the study investigator, taking into consideration BCVA, prior response to treatment, and any other medical conditions that may indicate which eye is the best candidate for retreatment. Subjects will be followed for 12 months for safety and efficacy.
- Conditions
- Retinitis Pigmentosa
- Being tested
- human retinal progenitor cells
- 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
single intravitreal injection of 6.0 million human retinal progenitor cells (hRPC)
| Study group | Role | What they receive |
|---|---|---|
| Retreated subjects | Experimental | Subjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. Receives: Biological: human retinal progenitor cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of Intravitreal Injection of hRPC
Assessed by percentage of subjects with treatment emergent adverse events
Measured: 12 months
Secondary outcomes (4)
Best Corrected Visual Acuity (BCVA)
Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS. 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.
Measured: 12 months
Kinetic Visual Field Area
Mean change in total kinetic visual field (KVF) area (degrees squared) of all islands of vision from baseline to 12 months. The Octopus 900 will be used for KVF testing using a specified target of V4e for subjects with a more severely impaired visual field (<10,000deg2) and a target of III4e and I4e for better seeing subjects (>10,000deg2). Target size is selected based on the Baseline visit. Whatever target size(s) is/are selected, the same size will be used throughout the study on that particular eye for that particular patient.
Measured: 12 months
Contrast Sensitivity (Peak)
Contrast sensitivity (CS) measures the ability of a subject to distinguish between finer and finer increments of light versus dark (contrast), as measured with a vertical striped pattern that varies in stripe width (cycles per degree or CPD); CS thresholds are created by taking the mean of multiple trials at each size of the target (i.e., at various CPDs). A CS curve is created by using the threshold of CS means at each target size, with the highest or most sensitive value (regardless of the CPD) representing the peak of the curve (i.e., peak contrast sensitivity). The unit of measure is therefore the peak contrast sensitivity regardless of the target size (i.e., CPD) being used to perform the measurement. The higher the value, the better the ability to detect contrast. The data shown here represent the mean change from Baseline to 12 months in the subjects' peak of the CS curve.
Measured: 12 months
Low Luminance Mobility Test (LLMT)
The LLMT identifies the performance of 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2020
- Study start1 Dec 2020
- Primary completion22 Mar 2022
- Study completion22 Mar 2022
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 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, University of California, Irvine/Gavin Herbert Eye InstituteDavid Liao, MD, Principal investigator, Retina-Vitreous Associates Medical Group, Los Angeles CAAnthony Jospeh, MD, Principal investigator, Ophthalmic Consultants of Boston
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04604899
- Sponsor's protocol ID
- JC02-2
- Status last verified
- Jun 2024
- First submitted
- 16 Oct 2020
- Last updated
- 16 Jul 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 16 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
- ClinicalTrials.gov ID
- NCT04604899
- Acronym / sponsor ID
- JC02-2
- Conditions
- Retinitis Pigmentosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- jCyte, IncIndustry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Study start
- 1 Dec 2020 (actual)
- Primary completion
- 22 Mar 2022 (actual)
- Study completion
- 22 Mar 2022 (actual)
- First posted
- 27 Oct 2020 (actual)
- Results
- Posted 26 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell.
Detailed description
This is a prospective, multi-center, single arm, Phase 2 study of human retinal progenitor cells (jCell) for the treatment of retinitis pigmentosa (RP). The study will include only subjects previously treated with jCell.
To assess reinjection of a previously treated eye, subjects who have previously been treated with jCell and desire a second treatment in the same eye will be enrolled. Subjects must have completed at least 12 months of follow up since the prior injection of jCell. Subjects who have had both eyes previously treated with jCell will only have one eye retreated; the eye to be retreated will preferably be the better seeing eye, but exceptions may be made by the study investigator, taking into consideration BCVA, prior response to treatment, and any other medical conditions that may indicate which eye is the best candidate for retreatment. Subjects will be followed for 12 months for safety and efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retreated subjects | Experimental | Biological: human retinal progenitor cellsSubjects receiving human retinal progenitor cells (jCell) who have previously received jCell is a jCyte study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Willing to give written informed consent, able to make the required study visits and follow study protocol instructions.
- Completed the 12 months of follow up in the subject's most recent jCell study and did not withdraw from the study for any reason.
- Adequate organ function:
- A female patient of childbearing potential (not surgically sterilized and less than one year postmenopausal) must have a negative pregnancy test (urine human chorionic gonadotropin) at entry (prior to injection) and must have used medically accepted contraception for at least one month prior to treatment. Women of childbearing potential and men must be advised to use a medically accepted method of contraception for at least 12 months following treatment.
Key exclusion criteria (9 listed)
- Malignancy, end-stage major organ disease (heart failure, significant arrhythmias, stroke or transient ischemic attacks, diabetes, immunosuppressive or autoimmune state, major psychiatric disorder, epilepsy, thyroid disease, COPD, renal failure, or any chronic systemic disease requiring continuous treatment with systemic steroids, anticoagulants or immunosuppressive agents.
- History of eye disease other than RP that impairs visual function, including retinal vascular disease, elevated intraocular pressure/glaucoma, severe posterior uveitis, clinically significant macular edema, media opacity precluding visual exam, amblyopia and/or longstanding constant strabismus, as well as patients who require other intravitreal therapies
- Allergy to penicillin or streptomycin.
- Adverse reaction to DMSO.
- Unable or unwilling to undergo pupil dilation, topical anesthesia or any protocol-required procedure.
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: jCellsingle intravitreal injection of 6.0 million human retinal progenitor cells (hRPC) | Biological | Retreated subjects |
Primary outcome
Safety of Intravitreal Injection of hRPC
Time frame: 12 months
Assessed by percentage of subjects with treatment emergent adverse events
4 secondary outcomes
Best Corrected Visual Acuity (BCVA)
Time frame: 12 months
Kinetic Visual Field Area
Time frame: 12 months
Contrast Sensitivity (Peak)
Time frame: 12 months
Low Luminance Mobility Test (LLMT)
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 16 Jul 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
University of California, Irvine/Gavin Herbert Eye Institute
David Liao, MD principal investigator
Retina-Vitreous Associates Medical Group, Los Angeles CA
Anthony Jospeh, MD principal investigator
Ophthalmic Consultants of Boston
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 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 8 inclusion and 9 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 16 Jul 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 26 Sep 2023). 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?
Safety of Intravitreal Injection of hRPC (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 16 Jul 2024.