Biomarkers in Retinitis Pigmentosa

NCT ID: NCT06306690

Last Updated: 2024-03-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

35 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-02-01

Study Completion Date

2025-04-30

Brief Summary

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The objective of this study is to discover biomarkers that demonstrate a correlation between the severity of retinitis pigmentosa (RP) and the thickness of the retinal pigment epithelium (RPE). These biomarkers will serve as prognostic indicators for various kinds of retinitis pigmentosa. The objective of this study is to find biomarkers that establish a correlation between the severity of retinitis pigmentosa and the thickness of the retinal pigment epithelium (RPE), which can serve as a prognostic indicator for Retinitis Pigmentosa.

Detailed Description

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After a genetic confirmation of RP and classification, the patients will undergo a comprehensive ophthalmological examination that includes the following tests: slit-lamp anterior segment, visual acuity direct and indirect ophthalmoscopy, intraocular pressure, and family history.In order to evaluate the potential role of RPE in the advancement of RP, HD-OCT and OCT angiography images of the outer retina using OCT devices will be performed.

Analysis of high-resolution images captured with an ultrawidefield system using a Zeiss Clarus device in order to determine the condition of the peripheral retina.Finally, Flicker Electroretinogram (fERG) performed on the central retina (macula), to assess the central macular function within an 18° field of view. This assessment involved measuring the response of the macula to a flickering stimulus with a frequency of 41 Hz, which is commonly done in routine clinical practice.

Conditions

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Retinitis Pigmentosa

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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1. Patients with rhodopsin mutation (RHO)

Following a clinical diagnosis, patients undergo genetic testing. Patients with rhodopsin mutation (RHO) a mutation are categorised into subgroup 1.

OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.

Intervention Type DIAGNOSTIC_TEST

the best visual acuity will be evaluated using decimal tables and then converted to logMar.

Ocular fundus examination will be evaluated following pharmacological mydriasis with Tropicamide 1%.

OCT will be essential to quantify the central macular thickness and the thickness of the RPE.

OCT angiography is a non-invasive method to assess the presence or absence of neovascular membrane and macular flow density.

Color and ultra wide field autofluorescence images of the retina will be performed with Zeiss Clarus retinography to search for signs that identify different types of RP and predict their activity and evolution.

Electroretinogram flicker (ERG) is a common measure of cone pathway function that is used to study the normal visual system and that of patients with inherited and acquired retinal dysfunction Measurements will be performed using an electrophysiological recording system (CSO). Intermittent stimuli are presented using a Ganzfeld dome (CSO).

2. Patients with pre-mRNA factor 8 (PRPF8) mutation

Following a clinical diagnosis, patients undergo genetic testing. Patients with pre-mRNA factor 8 (PRPF8) mutation are categorised into subgroup 2.

OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.

Intervention Type DIAGNOSTIC_TEST

the best visual acuity will be evaluated using decimal tables and then converted to logMar.

Ocular fundus examination will be evaluated following pharmacological mydriasis with Tropicamide 1%.

OCT will be essential to quantify the central macular thickness and the thickness of the RPE.

OCT angiography is a non-invasive method to assess the presence or absence of neovascular membrane and macular flow density.

Color and ultra wide field autofluorescence images of the retina will be performed with Zeiss Clarus retinography to search for signs that identify different types of RP and predict their activity and evolution.

Electroretinogram flicker (ERG) is a common measure of cone pathway function that is used to study the normal visual system and that of patients with inherited and acquired retinal dysfunction Measurements will be performed using an electrophysiological recording system (CSO). Intermittent stimuli are presented using a Ganzfeld dome (CSO).

3.Patients a cone-specific phosphodiesterase, i.e. PDE6B, mutation

Following a clinical diagnosis, patients undergo genetic testing. Patients with a cone-specific phosphodiesterase, i.e. PDE6B, mutation are categorised into subgroup 3.

OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.

Intervention Type DIAGNOSTIC_TEST

the best visual acuity will be evaluated using decimal tables and then converted to logMar.

Ocular fundus examination will be evaluated following pharmacological mydriasis with Tropicamide 1%.

OCT will be essential to quantify the central macular thickness and the thickness of the RPE.

OCT angiography is a non-invasive method to assess the presence or absence of neovascular membrane and macular flow density.

Color and ultra wide field autofluorescence images of the retina will be performed with Zeiss Clarus retinography to search for signs that identify different types of RP and predict their activity and evolution.

Electroretinogram flicker (ERG) is a common measure of cone pathway function that is used to study the normal visual system and that of patients with inherited and acquired retinal dysfunction Measurements will be performed using an electrophysiological recording system (CSO). Intermittent stimuli are presented using a Ganzfeld dome (CSO).

4. Patients with Chromosome 2-Open (C2orf71) mutation

Following a clinical diagnosis, patients undergo genetic testing. Patients with Chromosome 2-Open (C2orf71) mutation are categorised into subgroup 4.

OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.

Intervention Type DIAGNOSTIC_TEST

the best visual acuity will be evaluated using decimal tables and then converted to logMar.

Ocular fundus examination will be evaluated following pharmacological mydriasis with Tropicamide 1%.

OCT will be essential to quantify the central macular thickness and the thickness of the RPE.

OCT angiography is a non-invasive method to assess the presence or absence of neovascular membrane and macular flow density.

Color and ultra wide field autofluorescence images of the retina will be performed with Zeiss Clarus retinography to search for signs that identify different types of RP and predict their activity and evolution.

Electroretinogram flicker (ERG) is a common measure of cone pathway function that is used to study the normal visual system and that of patients with inherited and acquired retinal dysfunction Measurements will be performed using an electrophysiological recording system (CSO). Intermittent stimuli are presented using a Ganzfeld dome (CSO).

5. Patients with Guanylate Cyclase (GUCY2D) mutation

Following a clinical diagnosis, patients undergo genetic testing. Patients with Guanylate Cyclase (GUCY2D) mutation are categorised into subgroup 5.

OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.

Intervention Type DIAGNOSTIC_TEST

the best visual acuity will be evaluated using decimal tables and then converted to logMar.

Ocular fundus examination will be evaluated following pharmacological mydriasis with Tropicamide 1%.

OCT will be essential to quantify the central macular thickness and the thickness of the RPE.

OCT angiography is a non-invasive method to assess the presence or absence of neovascular membrane and macular flow density.

Color and ultra wide field autofluorescence images of the retina will be performed with Zeiss Clarus retinography to search for signs that identify different types of RP and predict their activity and evolution.

Electroretinogram flicker (ERG) is a common measure of cone pathway function that is used to study the normal visual system and that of patients with inherited and acquired retinal dysfunction Measurements will be performed using an electrophysiological recording system (CSO). Intermittent stimuli are presented using a Ganzfeld dome (CSO).

6. Patients with RP- specific nuclear receptor (Nr2E3) mutation

Following a clinical diagnosis, patients undergo genetic testing. Patients with RP- specific nuclear receptor (Nr2E3) mutation are categorised into subgroup 6.

OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.

Intervention Type DIAGNOSTIC_TEST

the best visual acuity will be evaluated using decimal tables and then converted to logMar.

Ocular fundus examination will be evaluated following pharmacological mydriasis with Tropicamide 1%.

OCT will be essential to quantify the central macular thickness and the thickness of the RPE.

OCT angiography is a non-invasive method to assess the presence or absence of neovascular membrane and macular flow density.

Color and ultra wide field autofluorescence images of the retina will be performed with Zeiss Clarus retinography to search for signs that identify different types of RP and predict their activity and evolution.

Electroretinogram flicker (ERG) is a common measure of cone pathway function that is used to study the normal visual system and that of patients with inherited and acquired retinal dysfunction Measurements will be performed using an electrophysiological recording system (CSO). Intermittent stimuli are presented using a Ganzfeld dome (CSO).

Interventions

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OCT, OCT angiography, flicker ERG and Ultra Wide Field retinography and autofluorescence.

the best visual acuity will be evaluated using decimal tables and then converted to logMar.

Ocular fundus examination will be evaluated following pharmacological mydriasis with Tropicamide 1%.

OCT will be essential to quantify the central macular thickness and the thickness of the RPE.

OCT angiography is a non-invasive method to assess the presence or absence of neovascular membrane and macular flow density.

Color and ultra wide field autofluorescence images of the retina will be performed with Zeiss Clarus retinography to search for signs that identify different types of RP and predict their activity and evolution.

Electroretinogram flicker (ERG) is a common measure of cone pathway function that is used to study the normal visual system and that of patients with inherited and acquired retinal dysfunction Measurements will be performed using an electrophysiological recording system (CSO). Intermittent stimuli are presented using a Ganzfeld dome (CSO).

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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Inclusion Criteria

* Patients who are able to read and sign informed consent
* Patients with Retinitis pigmentosa confirmed by genetic test.
* Patients older than or equal to 18 years of age

Exclusion Criteria

* Other retinal diseases such as macular hole, retinal detachment, macular neovascularization.
* Corneal surgery in the last 12 months
* Glaucoma with pressure above 25 mmHg in the last three months.
* Best Correct Visual Acuity below 1/10 in at least one eye.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fondazione Policlinico Universitario Agostino Gemelli IRCCS

OTHER

Sponsor Role lead

Responsible Party

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RIZZO STANISLAO

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Maria Cristina Savastano

Roma, , Italy

Site Status RECRUITING

Countries

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Italy

Central Contacts

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Stanislao Rizzo, MD, Prof

Role: CONTACT

Valentina Cestrone

Role: CONTACT

3200609905

Facility Contacts

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Maria C Savastano

Role: primary

3200609905

References

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Campochiaro PA, Mir TA. The mechanism of cone cell death in Retinitis Pigmentosa. Prog Retin Eye Res. 2018 Jan;62:24-37. doi: 10.1016/j.preteyeres.2017.08.004. Epub 2017 Sep 27.

Reference Type BACKGROUND
PMID: 28962928 (View on PubMed)

Dias MF, Joo K, Kemp JA, Fialho SL, da Silva Cunha A Jr, Woo SJ, Kwon YJ. Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives. Prog Retin Eye Res. 2018 Mar;63:107-131. doi: 10.1016/j.preteyeres.2017.10.004. Epub 2017 Oct 31.

Reference Type BACKGROUND
PMID: 29097191 (View on PubMed)

Cross N, van Steen C, Zegaoui Y, Satherley A, Angelillo L. Retinitis Pigmentosa: Burden of Disease and Current Unmet Needs. Clin Ophthalmol. 2022 Jun 20;16:1993-2010. doi: 10.2147/OPTH.S365486. eCollection 2022.

Reference Type BACKGROUND
PMID: 35757022 (View on PubMed)

Chaumet-Riffaud AE, Chaumet-Riffaud P, Cariou A, Devisme C, Audo I, Sahel JA, Mohand-Said S. Impact of Retinitis Pigmentosa on Quality of Life, Mental Health, and Employment Among Young Adults. Am J Ophthalmol. 2017 May;177:169-174. doi: 10.1016/j.ajo.2017.02.016. Epub 2017 Feb 22.

Reference Type BACKGROUND
PMID: 28237413 (View on PubMed)

Hartong DT, Berson EL, Dryja TP. Retinitis pigmentosa. Lancet. 2006 Nov 18;368(9549):1795-809. doi: 10.1016/S0140-6736(06)69740-7.

Reference Type BACKGROUND
PMID: 17113430 (View on PubMed)

Wu KY, Kulbay M, Toameh D, Xu AQ, Kalevar A, Tran SD. Retinitis Pigmentosa: Novel Therapeutic Targets and Drug Development. Pharmaceutics. 2023 Feb 17;15(2):685. doi: 10.3390/pharmaceutics15020685.

Reference Type BACKGROUND
PMID: 36840007 (View on PubMed)

Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14.

Reference Type BACKGROUND
PMID: 28712537 (View on PubMed)

Iftikhar M, Lemus M, Usmani B, Campochiaro PA, Sahel JA, Scholl HPN, Shah SMA. Classification of disease severity in retinitis pigmentosa. Br J Ophthalmol. 2019 Nov;103(11):1595-1599. doi: 10.1136/bjophthalmol-2018-313669. Epub 2019 Jan 31.

Reference Type BACKGROUND
PMID: 30705041 (View on PubMed)

Savastano MC, Falsini B, Ferrara S, Scampoli A, Piccardi M, Savastano A, Rizzo S. Subretinal Pigment Epithelium Illumination Combined With Focal Electroretinogram and Visual Acuity for Early Diagnosis and Prognosis of Non-Exudative Age-Related Macular Degeneration: New Insights for Personalized Medicine. Transl Vis Sci Technol. 2022 Jan 3;11(1):35. doi: 10.1167/tvst.11.1.35.

Reference Type BACKGROUND
PMID: 35077530 (View on PubMed)

Guymer RH, Rosenfeld PJ, Curcio CA, Holz FG, Staurenghi G, Freund KB, Schmitz-Valckenberg S, Sparrow J, Spaide RF, Tufail A, Chakravarthy U, Jaffe GJ, Csaky K, Sarraf D, Mones JM, Tadayoni R, Grunwald J, Bottoni F, Liakopoulos S, Pauleikhoff D, Pagliarini S, Chew EY, Viola F, Fleckenstein M, Blodi BA, Lim TH, Chong V, Lutty J, Bird AC, Sadda SR. Incomplete Retinal Pigment Epithelial and Outer Retinal Atrophy in Age-Related Macular Degeneration: Classification of Atrophy Meeting Report 4. Ophthalmology. 2020 Mar;127(3):394-409. doi: 10.1016/j.ophtha.2019.09.035. Epub 2019 Sep 30.

Reference Type BACKGROUND
PMID: 31708275 (View on PubMed)

Placidi G, Maltese PE, Savastano MC, D'Agostino E, Cestrone V, Bertelli M, Chiurazzi P, Maceroni M, Minnella AM, Ziccardi L, Parisi V, Rizzo S, Falsini B. Retinitis Pigmentosa Associated with EYS Gene Mutations: Disease Severity Staging and Central Retina Atrophy. Diagnostics (Basel). 2023 Feb 23;13(5):850. doi: 10.3390/diagnostics13050850.

Reference Type BACKGROUND
PMID: 36899994 (View on PubMed)

Other Identifiers

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6401

Identifier Type: -

Identifier Source: org_study_id

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