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Retinitis PigmentosaPhase 1NCT04284293

CNS10-NPC for the Treatment of RP

A Phase 1 study testing CNS10-NPC implantation in people with Retinitis Pigmentosa, run by Cedars-Sinai Medical Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The investigator is examining the safety of transplanting cells into the subretinal space of patients with Retinitis Pigmentosa (RP). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. 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?
CNS10-NPC implantation
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteUnited States
Study dates
22 Jul 2021to Jul 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

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

  • To participate in this study, the subject must be 18 years of age or older and must understand and sign the protocol's informed consent.
  • Participant with diagnosis of retinitis pigmentosa.
  • Clinical signs of retinitis pigmentosa.
  • A history of nyctalopia
  • Retinal pigmentary changes
  • Arteriolar attenuation
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  • Waxy disc pallor
  • Electrophysiologic evidence of rod dysfunction on full field electroretinography
  • Visual field constriction.
  • 3a. Participants in Group 1 (n=6) will have visual acuity equal to or worse than 20/200. Participants in Group 2 (n=10) will have visual acuity equal to or worse than 20/80.
  • 3b. Group 1 participants will have central visual field of 40 degrees diameter or less. Group 2, participants will have a measurable visual field defect.
  • 4. Participant will be medically able to undergo ophthalmic surgery.

Who cannot join

  • Presence of significant ocular abnormalities that would preclude the planned surgery or interfere with the interpretation of study endpoints (e.g. glaucoma, corneal or significant lens opacities, pre-existing uveitis, intraocular infection, macular edema, choroidal neovascularization). Any ocular diseases that the investigator feels would interfere with accurate ocular measurements. This would exclude potential subjects with significant cataract, corneal scars or significant corneal irregularities such as keratoconus, previous penetrating keratoplasty or glaucoma with central visual field.
  • Any pre-existing factor or history of eye disease that may predispose to an increased risk of surgical complications in the study eye (e.g. trauma, previous surgery other than uncomplicated cataract surgery, uveitis, congenital developmental or structural abnormalities).
  • Concomitant systemic diseases including those in which the disease itself, or the treatment for the disease, can alter ocular function or immune status (e.g. malignancies, uncontrolled diabetes).
  • Any ocular surgery or laser in either eye within 12 weeks of screening.
  • Any contraindication to pupil dilatation in either eye.
  • Treatment with intravitreal, subtenon or periocular steroid within 4 months of enrollment.
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  • Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study (e.g., dilation drops), or medications planned for use during the peri-operative period including corticosteroids, tacrolimus and mycophenolate.
  • Imminently life-threatening illness.
  • Abuse of alcohol or any illegal substance(s) within 12 months of the procedure.
  • Laboratory test abnormalities or abnormalities in electrocardiogram or chest X-ray, which in the opinion of the Principal Investigator are clinically significant and would make the patient unable to tolerate study procedures.
  • Intercurrent illness or infection 28 days prior to enrolment.
  • Contraindications to use of anesthesia.
  • Females of child-bearing potential (i.e. those who are not surgically sterile and not at least 12-months post-menopausal) who are not willing to comply with the study's contraception requirement (women who are unwilling to use an effective form of contraception such as the contraceptive pill or intrauterine device).
  • Women who are pregnant.
  • Females who are nursing or who intend to nurse during the first 6 months post-treatment.
  • Men or women who do not agree to use barrier or medical contraception for at least 6 months post-operatively.
  • History of any investigational agent administration within 28 days prior to administration.
  • Participation in a prior gene transfer therapy study or cell-based therapy.
  • Enrollment in any other clinical study, for any condition, including those relating to RP throughout the duration of the study.
  • Current or anticipated long-term treatment with systemic corticosteroids (for a period longer than 7 days).
  • Current treatment with immunosuppressant therapies or any contraindications to use of the immunosuppressants in this protocol.
  • A history of malignancy within a five-year period or a positive cancer screening test within a one-year period of the screening visit.
  • Any physical or mental disability that will impair the ability of the patient to travel to and from the study center or provide informed consent/assent or effective safety assessments as specified by the protocol. Any mental or psychiatric disorders that prevent the patient from having full authority to do so (i.e. the subject cannot have power of attorney signed over to another individual).
  • Inability or unwillingness to comply with the study protocol.
  • Medical history of HIV, or hepatitis A, B or C.
  • Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane® Soriatane®), or any medicines that may affect the macula (e.g. tamoxifen, mellaril, thorazine, plaquenil, niacin) within 6 months of enrollment.
  • Allergy to Beta-Lactam antibiotics.
  • The presence of cystoid macular edema.
  • Glucose-6-phosphate dehydrogenase (G6PD) Deficiency

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Retina-Vitreous Associates Medical Group

    Beverly Hills, California, 90211

Other trials in United States

About this study

Official title: Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa

The investigator is examining the safety of transplanting cells into the subretinal space of patients with Retinitis Pigmentosa (RP). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neuronal cell. The cells are called "CNS10-NPC." The investigational treatment has been tested in animals, but it has not yet been tested in people. In this study, the investigators want to learn if CNS10-NPC cells are safe to transplant into the subretinal space of people.

Read the detailed description

This study will be the first to use a human progenitor cell line to treat retinitis pigmentosa in people. This is a Phase 1/2a, single-center, open label, safety study of two escalating doses of human neural progenitor cells (CNS10-NPC) delivered unilaterally to the subretinal space of subjects with RP.

Subjects meeting all Eligibility Criteria and providing Informed Consent will be enrolled in one of two sequential dosing groups. Subjects will be treated sequentially with a minimum of one month interval between surgeries for the first three subjects in each dosing cohort. The remaining subjects in the cohort will be treated with a minimum interval of at least one week between surgeries.

Primary objective. To assess the safety and tolerability of two escalating doses of clinical grade human fetal cortical-derived neural progenitor cells (CNS10-NPC) administered in the subretinal space of one eye (unilaterally) in patients with retinitis pigmentosa (RP).

Secondary objectives. Within constraints of a small first in-human study focused on safety:

  1. Determine if CNS10-NPC can engraft and survive long-term in the retina of transiently immunosuppressed subjects,
  2. Obtain evidence that subretinal injection of CNS10-NPC can favorably impact the progression of vision loss in subjects with moderate RP.
Being tested
CNS10-NPC implantation
Keywords
RP, Retinopathy, Pigmentary Retinopathy, Retinal Degeneration
Medical subject headings
Retinitis Pigmentosa, Retinal Diseases, Retinal Degeneration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CNS10-NPC implantationBiological

    Also called Neural Progenitor Cell (NPC), Stem Cell

    All patients will receive a single, unilateral, subretinal injection of CNS10-NPC

Study groups in NCT04284293
Study groupRoleWhat they receive
Group 1AExperimentalVisual acuity of 20/200 or worse Single, unilateral, subretinal injection of 300,000 CNS10-NPC (n=3)
Receives: Biological: CNS10-NPC implantation
Group 1BExperimentalVisual acuity of 20/200 or worse Single, unilateral, subretinal injection of 1,000,000 CNS10-NPC (n=3)
Receives: Biological: CNS10-NPC implantation
Group 2ExperimentalVisual acuity between 20/80 and 20/200 Single, unilateral, subretinal injection of 1,000,000 CNS10-NPC (n=10)
Receives: Biological: CNS10-NPC implantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety as evaluated by incidence of Adverse Events (AE) and Serious Adverse Events (SAE) and their relationship to the intervention

    Measured: Subjects will be followed postoperatively for 12 months

  2. Safety, as evaluated by changes in the complete blood count

    Measured: Subjects will be followed postoperatively for 12 months

  3. Safety, as evaluated by changes in the comprehensive metabolic panel

    Measured: Subjects will be followed postoperatively for 12 months

  4. Safety, as evaluated by changes in Donor Specific Antibodies

    Measured: Subjects will be followed postoperatively for 12 months

  5. Safety, as evaluated by changes in the urinalysis

    Measured: Subjects will be followed postoperatively for 12 months

  6. Safety, as evaluated by changes in Visual Acuity

    ETRDS, or FrACT in cases of very low vision.

    Measured: Subjects will be followed postoperatively for 12 months

  7. Safety, as evaluated by changes in visual field

    Goldman perimetry will be used for central visual fields and microperimetry will be used for peripheral visual fields

    Measured: Subjects will be followed postoperatively for 12 months

  8. Safety, as evaluated by changes in Spectral Domain Optical Coherence Tomography (SD-OCT)

    Ellipsoid zone measurement through SD-OCT will be performed

    Measured: Subjects will be followed postoperatively for 12 months

Secondary outcomes (11)

  1. Slit lamp examination

    Measured: Performed 7 times over 15 months

  2. Intraocular pressure measurement

    Measured: Performed 7 times over 15 months

  3. Funduscopic examination

    Measured: Performed 7 times over 15 months

  4. Fundus photography (color, red free, or infrared)

    Measured: Performed 4 times over 15 months

  5. Fundus Autofluorescence (FAF)

    Measured: Performed 4 times over 15 months

  6. Best Corrected Visual Acuity

    Measured: Performed 7 times over 15 months

  7. Visual Function Questionnaire-25 (VFQ-25)

    The Visual Function Questionnaire-25 is graded in a scale from 0-100 where a higher number represents better function

    Measured: Performed 5 times over 15 months

  8. Spectral domain optical coherence tomography (SD-OCT)

    Retinal Thickness

    Measured: Performed 5 times over 15 months

  9. Electroretinography (ERG)

    Measured: Performed 5 times over 15 months

  10. Goldmann visual field area (microperimetry)

    Measured: Performed 5 times over 15 months

  11. Change in rate of vision field loss

    Measured: Through study completion, ~15 months.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Feb 2020
  2. Study start22 Jul 2021
  3. Primary completionJul 2026 (estimated)
  4. Study completionJul 2026 (estimated)

Resources

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04284293
Sponsor's protocol ID
Study00000375
Status last verified
Aug 2025
First submitted
18 Feb 2020
Last updated
19 Aug 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Lu B, Avalos P, Svendsen S, Zhang C, Nocito L, Jones MK, Pieplow C, Saylor J, Ghiam S, Block A, Fernandez M, Ljubimov AV, Small K, Liao D, Svendsen CN, Wang S. GMP-grade human neural progenitors delivered subretinally protect vision in rat model of retinal degeneration and survive in minipigs. J Transl Med. 2023 Sep 25;21(1):650. doi: 10.1186/s12967-023-04501-z.

    PubMed 37743503

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CNS10-NPC implantation in people with Retinitis Pigmentosa, run by Cedars-Sinai Medical Center at 1 site 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 12 inclusion and 29 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: under way, not looking for participants, as of the sponsor's last update on 19 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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 as evaluated by incidence of Adverse Events (AE) and Serious Adverse Events (SAE) and their relationship to the intervention (time frame: Subjects will be followed postoperatively for 12 months). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04284293, which also lists 1 linked publication.

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 19 Aug 2025.

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