Retinitis PigmentosaPhase 3SiCordNCT04636853
CB-PRP in Retinitis Pigmentosa and Dry Age-related Macular Degeneration
A Phase 3 study testing Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) in people with Retinitis Pigmentosa and Dry Age-related Macular Degeneration, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 1 site in Italy.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Retinitis Pigmentosa, Dry Age-related Macular Degeneration
- What is being tested?
- Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteItaly
- Study dates
- 23 Dec 2020to 2 Dec 2021
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- Age<18 years
- Pregnancy
- Previous inflammatory / infectious events involving the eyes
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Cord Blood Platelet-rich Plasma (CB-PRP) in Retinitis Pigmentosa and Dry Age-related Macular Degeneration
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.
Read the 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.
- Being tested
- Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP)
- Keywords
- Cord blood platelet-rich plasma, Retinitis pigmentosa, Dry Age-related Macular degeneration, Macular Geographic Atrophy
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Optical coherence tomography (OCT) and optical coherence tomography angiography (OCTA) morphological changes
Differences in structural optical coherence tomography (OCT) and OCT Angiography (OCTA) images
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2020
- Study start23 Dec 2020
- Primary completion1 Dec 2021
- Study completion2 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- More trials run by Fondazione Policlinico Universitario Agostino Gemelli IRCCSThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alfonso Savastano, Principal investigator, Fondazione Policlinico Universitario Agostino Gemelli IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04636853
- Sponsor's protocol ID
- 3417
- Acronym
- SiCord
- Status last verified
- Dec 2020
- First submitted
- 14 Nov 2020
- Last updated
- 11 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 30858959Rizzo 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.
PubMed 39149709
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cord Blood Platelet-rich Plasma (CB-PRP) in Retinitis Pigmentosa and Dry Age-related Macular Degeneration
- ClinicalTrials.gov ID
- NCT04636853
- Acronym / sponsor ID
- SiCord / 3417
- Keywords
- Cord blood platelet-rich plasma, Retinitis pigmentosa, Dry Age-related Macular degeneration, Macular Geographic Atrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCSUniversity, hospital or other
- Study start
- 23 Dec 2020 (actual)
- Primary completion
- 1 Dec 2021 (actual)
- Study completion
- 2 Dec 2021 (actual)
- First posted
- 19 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Affected Individual | Experimental | Biological: Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP)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. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (3 listed)
- Age<18 years
- Pregnancy
- Previous inflammatory / infectious events involving the eyes
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP)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. | Biological | Affected Individual |
Primary outcome
Optical coherence tomography (OCT) and optical coherence tomography angiography (OCTA) morphological changes
Time frame: 3 years
Differences in structural optical coherence tomography (OCT) and OCT Angiography (OCTA) images
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2023.
- Results publication: PubMed 30858959Valentini 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.
- Derived from this study: PubMed 39149709Rizzo 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alfonso Savastano principal investigator
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP) in people with Retinitis Pigmentosa and Dry Age-related Macular Degeneration, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 1 site in Italy.
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 7 inclusion and 3 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: finished, as of the sponsor's last update on 11 Jan 2023. 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 Italy. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Optical coherence tomography (OCT) and optical coherence tomography angiography (OCTA) morphological changes (time frame: 3 years). 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 NCT04636853, which also lists 2 linked publications.
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 11 Jan 2023.