Clinical trial NCT04636853: CB-PRP in Retinitis Pigmentosa and Dry Age-related Macular Degeneration
Official title: Cord Blood Platelet-rich Plasma (CB-PRP) in Retinitis Pigmentosa and Dry Age-related Macular Degeneration
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Retinitis Pigmentosa
Dry Age-related Macular Degeneration
Keywords: Cord blood platelet-rich plasma
Retinitis pigmentosa
Dry Age-related Macular degeneration
Macular Geographic Atrophy
Lead sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sponsor class: OTHER
First posted: 2020-11-19
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

Detailed description: All patients underwent genetic evaluation and they reported different types of mutations that induce anatomic-functional impairment of the rods.

Patients affected by bilateral dry age-related macular degeneration (Geographic type) will be recruited as well.

After selecting the patients to be enrolled, the study will proceed with the vitrectomy surgery and subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) provided by the Umbilical Cord Bank according to the procedures provided for by the current legislation on blood components for non-transfusion use.

A complete ophthalmological examination will be performed at 1, 3, 6, and 12 months. They will include:

* Anterior segment biomicroscopy
* direct and indirect ophthalmoscopy
* ETDRS visual acuity assessment
* intraocular pressure measurement
* optical coherence tomography (OCT)
* OCT Angiography (OCTA)

The following assessments will be performed at baseline and at 6 and 12 months.

* Microperimetry
* Electroretinogram (ERG)
* Visually evoked potential (VEP)
* contrast sensitivity (MARS tables)
* Goldmann perimetry

Some morpho-functional data will also be collected in the contralateral eye and used as the control group.

To ensure the best safety for the patient, the first 5 eyes will be separated by a minimum of 20 days in order to observe the post-surgical evolution.

Eligibility criteria: Inclusion Criteria:

* 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)

Exclusion Criteria:

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

Primary outcomes:
measure: Optical coherence tomography (OCT) and optical coherence tomography angiography (OCTA) morphological changes
timeFrame: 3 years
Secondary outcomes:
Other outcomes:
Locations:
city: Rome
country: Italy
facility: Alfonso Savastano
Study officials:
affiliation: Fondazione Policlinico Universitario A. Gemelli, IRCCS
name: Alfonso Savastano
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.