Subtenon Autologous Platelet-Rich Plasma in Inherited and Degenerative Retinal Diseases

NCT07341919 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: RETINA-PRP Study
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

his prospective, comparative pilot study investigates the safety and functional outcomes of subtenon autologous platelet-rich plasma (PRP) in patients with Retinitis Pigmentosa (RP) and Extensive Macular Atrophy with Pseudodrusen-like Appearance (EMAP). Participants will receive three subtenon injections of autologous platelet-rich plasma (1.5 mL per injection) administered at two-month intervals (M0, M2, M4). The primary objective is to assess functional changes over a 6-month period, with a focus on visual field preservation, evaluated by the Field Preservation Deviation Index (FPDI) and Mean Deviation (MD), as well as best-corrected visual acuity (BCVA, LogMAR). Secondary outcomes include changes in 30-Hz flicker electroretinography (ERG) amplitude, structural retinal parameters on optical coherence tomography (OCT)-including central macular thickness and ellipsoid zone length-and ocular safety outcomes, such as intraocular pressure, local tolerability, and the occurrence of inflammatory or adverse events related to subtenon PRP administration.

Conditions

Retinitis Pigmentosa (RP); Extensive Macular Atrophy With Pseudodrusen (EMAP)

Keywords

Retinitis pigmentosa; EMAP; Macular Atrophy; Pseudodrusen-like; platelet-rich plasma; PRP

Outcome Measures

Primary Outcomes (3)

• Change in Field Preservation Deviation Index (FPDI)

  Change (Δ) in Field Preservation Deviation Index (FPDI), expressed as a percentage (%), measured by automated perimetry using the iCare COMPASS system. The FPDI reflects the proportion of preserved visual field relative to age-matched normative data and provides a quantitative assessment of global visual field integrity. The outcome is defined as the difference between baseline (Month 0) and Month 6 values in the study eye.

  Baseline to Month 6

• . Change in Mean Deviation (MD)

  Change (Δ) in Mean Deviation (MD), expressed in decibels (dB), measured by automated perimetry using the iCare COMPASS system. Mean Deviation represents the average difference in retinal sensitivity compared with age-adjusted normative values, serving as a global index of visual field loss. The outcome corresponds to the difference between baseline (Month 0) and Month 6 measurements in the study eye.

  Baseline to Month 6

• . Change in Best-Corrected Visual Acuity (BCVA)

  Change (Δ) in Best-Corrected Visual Acuity (BCVA), expressed in logarithm of the minimum angle of resolution (LogMAR), measured using standardized Early Treatment Diabetic Retinopathy Study (ETDRS) charts under controlled testing conditions. BCVA assesses central visual function and foveal integrity. The outcome is defined as the difference between baseline (Month 0) and Month 6 BCVA values in the study eye.

  Baseline to Month 6

Secondary Outcomes (4)

• Change in Pattern Standard Deviation (PSD)

  Baseline to Month 6]

• Change in 30-Hz Flicker ERG Amplitude

  Baseline to Month 6

• Change in Central Macular Thickness (CMT)

  Baseline to Month 6

• Change in Ellipsoid Zone (EZ) Length

  Baseline to Month 6

Study Arms (1)

Subtenon Injection of Autologous Platelet-Rich Plasma

EXPERIMENTAL

Experimental: Subtenon Injection of Autologous Platelet-Rich Plasma Participants receive subtenon injections of autologous platelet-rich plasma (PRP), 1.5 mL per injection, administered at baseline (M0), month 2 (M2), and month 4 (M4). This arm evaluates the effect of local autologous regenerative and immunomodulatory therapy on visual function and retinal structure in patients with degenerative retinal diseases, including Retinitis Pigmentosa and Extensive Macular Atrophy with Pseudodrusen-like Appearance (EMAP).

Biological: Subtenon Injection of Autologous Platelet-Rich Plasma

Interventions

Subtenon Injection of Autologous Platelet-Rich Plasma BIOLOGICAL

Biological: Subtenon Autologous Platelet-Rich Plasma Participants receive subtenon injections of autologous platelet-rich plasma (PRP), 1.5 mL per injection, administered at baseline (M0), month 2 (M2), and month 4 (M4).

Also known as: PRP

Subtenon Injection of Autologous Platelet-Rich Plasma

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years.
• Clinical diagnosis of retinitis pigmentosa (RP) or EMAP (Extensive Macular Atrophy with Pseudodrusen-like Appearance), confirmed by multimodal evaluation.
• Best-corrected visual acuity (BCVA) ≥ counting fingers at 1 meter (approximately ≤ 1.9 logMAR) in the study eye.
• Measurable visual field on iCare COMPASS (10-2 or 24-2) with acceptable reliability indices.
• Clear ocular media adequate for safe intravitreal injection and high-quality optical coherence tomography (OCT) imaging.
• Ability and willingness to provide written informed consent.
• Ability to comply with scheduled study visits, including:
• Baseline (M0)
• Day 7-14 after injections
• Month 2 (M2), Day 7-14
• Month 4 (M4), Day 7-14
• Month 6 (M6)
• For ERG subset only:
• o Presence of a recordable baseline 30-Hz flicker electroretinogram (ERG) response, defined as a signal-to-noise ratio ≥ 3:1 and amplitude ≥ 3.0 μV.
• Note: Absence of a measurable flicker ERG response does not exclude participation in the main study.

You may not qualify if:

• Active ocular inflammation (anterior, intermediate, or posterior uveitis) or active infectious ocular disease in the study eye.
• Active choroidal neovascularization or other macular diseases unrelated to RP or EMAP.
• Uncontrolled glaucoma (intraocular pressure \> 21 mmHg despite therapy) or optic neuropathies not related to RP or EMAP.
• Significant media opacity that may impair imaging quality or compromise the safety of intravitreal injection.
• Recent ocular interventions that may confound study outcomes, including:
• Intravitreal therapy within 3 months prior to enrollment.
• Periocular corticosteroid injection within 3 months prior to enrollment.
• Major intraocular surgery within 3 months prior to enrollment.
• Known hypersensitivity to adalimumab, povidone-iodine, local anesthetics, or any component of the study formulations.
• Coagulopathy or contraindications to ocular injections, including:
• Platelet count \< 100,000/μL, or
• INR \> 1.5 unless adequately corrected.
• Pregnancy or breastfeeding.
• Women of childbearing potential unwilling to use effective contraception during the study period.
• Uncontrolled systemic disease that, in the investigator's judgment, increases risk or interferes with study participation or completion.

Sponsors & Collaborators

• Rubens Camargo Siqueira: lead

Study Sites (1)

Centro Especializado Retina e Vítreo

São José do Rio Preto, São Paulo, 15010-100, Brazil

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Anetoderma

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Abnormalities; Skin Diseases

Study Officials

• Rubens C Siqueira, MD,PhD

  Rubens Siqueira Research Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD,PhD

Model Details: This is a prospective, single-arm, open-label interventional pilot study designed to assess the ocular safety, feasibility, and functional outcomes of subtenon administration of autologous platelet-rich plasma (PRP) in patients with degenerative retinal diseases. All participants receive three subtenon injections of autologous PRP (1.5 mL per injection) administered at baseline (M0) and at two-month intervals (M2, M4). Given the exploratory nature of the study and the heterogeneity of degenerative retinal diseases, no randomization or masking is applied. Each participant serves as their own control, with outcomes evaluated as within-subject changes from baseline to six months (M0-M6). The study aims to provide proof-of-concept data on the regenerative and immunomodulatory potential of PRP, focusing on functional outcomes and OCT-based biomarkers, to support the design of future controlled trials.

Study Record Dates

• First Submitted: December 13, 2025
• First Posted: January 14, 2026
• Study Start: January 4, 2025
• Primary Completion: March 4, 2026
• Study Completion (Estimated): January 4, 2027
• Last Updated: March 24, 2026

Record last verified: 2026-03

Locations

BR (1)