CNS10-NPC for the Treatment of RP

NCT04284293 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: Study00000375
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Retinitis Pigmentosa

Keywords

RP; Retinopathy; Pigmentary Retinopathy; Retinal Degeneration

Outcome Measures

Primary Outcomes (8)

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

  Subjects will be followed postoperatively for 12 months

• Safety, as evaluated by changes in the complete blood count

  Subjects will be followed postoperatively for 12 months

• Safety, as evaluated by changes in the comprehensive metabolic panel

  Subjects will be followed postoperatively for 12 months

• Safety, as evaluated by changes in Donor Specific Antibodies

  Subjects will be followed postoperatively for 12 months

• Safety, as evaluated by changes in the urinalysis

  Subjects will be followed postoperatively for 12 months

• Safety, as evaluated by changes in Visual Acuity

  ETRDS, or FrACT in cases of very low vision.

  Subjects will be followed postoperatively for 12 months

• 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

  Subjects will be followed postoperatively for 12 months

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

  Ellipsoid zone measurement through SD-OCT will be performed

  Subjects will be followed postoperatively for 12 months

Secondary Outcomes (11)

• Slit lamp examination

  Performed 7 times over 15 months

• Intraocular pressure measurement

  Performed 7 times over 15 months

• Funduscopic examination

  Performed 7 times over 15 months

• Fundus photography (color, red free, or infrared)

  Performed 4 times over 15 months

• Fundus Autofluorescence (FAF)

  Performed 4 times over 15 months

Study Arms (3)

Group 1A

EXPERIMENTAL

Visual acuity of 20/200 or worse Single, unilateral, subretinal injection of 300,000 CNS10-NPC (n=3)

Biological: CNS10-NPC implantation

Group 1B

EXPERIMENTAL

Visual acuity of 20/200 or worse Single, unilateral, subretinal injection of 1,000,000 CNS10-NPC (n=3)

Biological: CNS10-NPC implantation

Group 2

EXPERIMENTAL

Visual acuity between 20/80 and 20/200 Single, unilateral, subretinal injection of 1,000,000 CNS10-NPC (n=10)

Biological: CNS10-NPC implantation

Interventions

CNS10-NPC implantation BIOLOGICAL

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

Also known as: Neural Progenitor Cell (NPC), Stem Cell

Group 1A; Group 1B; Group 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Waxy disc pallor
• Electrophysiologic evidence of rod dysfunction on full field electroretinography
• Visual field constriction.
• a. 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.
• b. Group 1 participants will have central visual field of 40 degrees diameter or less. Group 2, participants will have a measurable visual field defect.
• \. Participant will be medically able to undergo ophthalmic surgery.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Cedars-Sinai Medical Center: lead
• California Institute for Regenerative Medicine (CIRM): collaborator

Study Sites (1)

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

Related Publications (1)

• 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.

  PMID: 37743503 DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa; Retinal Diseases; Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• David Liao, MD, PhD

  Retina-Vitreous Associates Medical Group

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Regenerative Medicine Institute

Study Record Dates

• First Submitted: February 18, 2020
• First Posted: February 25, 2020
• Study Start: July 22, 2021
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: August 19, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)