NCT04636853

Brief Summary

The study will provides the enrollment of patients with genetic retinal dystrophies with primary rod impairment and dry age-related macular degeneration (Geographic type) A subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) will be performed only in one eye, the other eye will be considered as a control group. A complete morpho-functional ophthalmological evaluation will be performed in all patients at each control.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Dec 2020

Shorter than P25 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 14, 2020

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 19, 2020

Completed
1 month until next milestone

Study Start

First participant enrolled

December 23, 2020

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2021

Completed
1 day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 2, 2021

Completed
Last Updated

January 11, 2023

Status Verified

December 1, 2020

Enrollment Period

11 months

First QC Date

November 14, 2020

Last Update Submit

January 9, 2023

Conditions

Retinitis PigmentosaDry Age-related Macular Degeneration

Keywords

Cord blood platelet-rich plasmaRetinitis pigmentosaDry Age-related Macular degenerationMacular Geographic Atrophy

Outcome Measures

Primary Outcomes (1)

  • Optical coherence tomography (OCT) and optical coherence tomography angiography (OCTA) morphological changes

    Differences in structural optical coherence tomography (OCT) and OCT Angiography (OCTA) images

    3 years

Study Arms (1)

Affected Individual

EXPERIMENTAL

A subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) will be performed only in one eye, the other eye will be considered as a control group.

Biological: Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP)

Interventions

Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP)BIOLOGICAL

A subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) will be performed only in one eye, the other eye will be considered as a control group. A complete morpho-functional ophthalmological evaluation will be performed in all patient at each control.

Affected Individual

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Retinal dystrophies with compromised rods (only for RP patients)
  • Visual Field (Manual Goldmann) V / 4e \< 30 ° (only for RP patients)
  • Best corrected visual acuity \> Light perception
  • Known genotype
  • No or minimal opacity of ocular media
  • No concomitant ocular (eg glaucoma, amblyopia)
  • Dry Age-related Macular Degeneration (Geographic type)

You may not qualify if:

  • Age\<18 years
  • Pregnancy
  • Previous inflammatory / infectious events involving the eyes

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Alfonso Savastano

Rome, 00168, Italy

Location

Related Publications (2)

  • Valentini CG, Nuzzolo ER, Bianchi M, Orlando N, Iachininoto MG, Pinci P, Teofili L. Cord Blood Platelet Lysate: In Vitro Evaluation to Support the Use in Regenerative Medicine. Mediterr J Hematol Infect Dis. 2019 Jan 1;11(1):e2019021. doi: 10.4084/MJHID.2019.021. eCollection 2019.

    PMID: 30858959RESULT

  • Rizzo S, Savastano MC, Falsini B, Bernardinelli P, Boselli F, De Vico U, Carla MM, Giannuzzi F, Fossataro C, Gambini G, Crincoli E, Ferrara S, Ripa M, Killian R, Rizzo C, Valentini CG, Orlando N, Placidi G, Teofili L, Savastano A. Safety Results for Geographic Atrophy Associated with Age-Related Macular Degeneration Using Subretinal Cord Blood Platelet-Rich Plasma. Ophthalmol Sci. 2024 Jan 24;4(6):100476. doi: 10.1016/j.xops.2024.100476. eCollection 2024 Nov-Dec.

    PMID: 39149709DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Alfonso Savastano

    Fondazione Policlinico Universitario A. Gemelli, IRCCS

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 14, 2020

First Posted

November 19, 2020

Study Start

December 23, 2020

Primary Completion

December 1, 2021

Study Completion

December 2, 2021

Last Updated

January 11, 2023

Record last verified: 2020-12

Data Sharing

IPD Sharing
Will not share

Locations

IT(1)