Clinical trial • Phase III • Ophthalmology
BOTARETIGENE SPAROPARVOVEC for X-linked retinitis pigmentosa (RPGR-associated)
Phase III trial of BOTARETIGENE SPAROPARVOVEC for X-linked retinitis pigmentosa (RPGR-associated). 64 participants.
Overview
- Trial Therapeutic Area
- Ophthalmology
- Trial Disease
- X-linked retinitis pigmentosa (RPGR-associated)
- Trial Stage
- Phase III
- Drug Modality
- Gene therapy | Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 16-05-2024
- First CTIS Authorization Date
- 21-06-2024
Trial design
Phase III trial across 8 sites in France, Netherlands, Denmark and others.
- Target Sample Size
- 64
- Trial Duration For Participant
- 2163
Eligibility
Recruits 64 adults.
Inclusion criteria
- {"criterion_text":"- 1.Previously completed participation in Study MGT-RPGR-021.\n- 2.Must reconfirm that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study."}
Exclusion criteria
- {"criterion_text":"- There are no specific exclusion criteria."}
Endpoints
Primary endpoints
- {"endpoint_text":"- 1.\tAdverse events\n- 2.\tLaboratory assessments\n- 3.\tChange from baseline in best corrected visual acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in monocular assessment\n- 4.\tChange from baseline in low luminance visual acuity (LLVA) by ETDRS chart letter score in monocular assessment","definition_or_measurement_approach":"Adverse events: not further defined in record; Laboratory assessments: not further specified; BCVA: measured by ETDRS chart letter score in monocular assessment; LLVA: measured by ETDRS chart letter score in monocular assessment."}
Recruitment
- Planned Sample Size
- 64
- Recruitment Window Months
- 71
- Consent Approach
- Informed consent is obtained directly from the participant; inclusion criteria require the participant to "reconfirm that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study." Subject information and informed consent forms (SIS/ICF) are available for multiple countries and languages (examples in the documentation list: FRA, NL, ES, IT, DK, and BE (EN/NL/FR) versions). Companion and Pregnant Partner information materials are also provided as separate documents.
Geography
- Total Number Of Sites
- 8
- Total Number Of Participants
- 31
France
- Latest Decision Or Authorization Date
- 07-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Quinze-Vingts National Ophthalmology Hospital
- Department Name
- Ophtalmology
- Contact Person Name
- Isabelle Audo
- Contact Person Email
- Isabelle.audo@inserm.fr
Netherlands
- Latest Decision Or Authorization Date
- 06-01-2026
- Number Of Sites
- 2
- Number Of Participants
- 9
Sites
- Site Name
- Stichting Amsterdam UMC
- Department Name
- Opthalmology
- Contact Person Name
- Camiel Jan Fons Boon
- Contact Person Email
- Camiel.boon@amsterdamumc.nl
- Site Name
- Stichting Radboud universitair medisch centrum
- Department Name
- Opthalmology
- Contact Person Name
- Carel Hoyng
- Contact Person Email
- Carel.hoyng@radboudumc.nl
Denmark
- Latest Decision Or Authorization Date
- 08-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Rigshospitalet
- Department Name
- Department of Ophthalmology
- Contact Person Name
- Line Kessel
- Contact Person Email
- line.kessel.01@regionh.dk
Spain
- Latest Decision Or Authorization Date
- 08-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 7
Sites
- Site Name
- Hospital Universitario Fundacion Jimenez Diaz
- Department Name
- Ophthalmology
- Contact Person Name
- Blanca Garcia Sandoval
- Contact Person Email
- bgarcia@fjd.es
Belgium
- Latest Decision Or Authorization Date
- 08-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 7
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- Ophtalmology
- Contact Person Name
- Bart Leroy
- Contact Person Email
- bart.leroy@uzgent.be
Italy
- Latest Decision Or Authorization Date
- 14-04-2026
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Careggi University Hospital
- Department Name
- Clinica Oftalmologica
- Contact Person Name
- Fabrizio Giansanti
- Contact Person Email
- Fabrizio.giansanti@unifi.it
- Site Name
- Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
- Department Name
- UOC Oculistica
- Contact Person Name
- Francesca Simonelli
- Contact Person Email
- francesca.simonelli@unicampania.it
Sponsor
Primary sponsor
- Full Name
- Janssen Cilag International
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Belgium
Contract research organisations
- Name
- Syneos Health Netherlands B.V.
- Responsibilities
- IVRS30 – treatment randomisation, Investigators Meetings, Site Contracts, DSMB, 3rd party vendors, Project Management; project management, vendor management and trial operations
- Name
- Bioclinica Inc.
- Responsibilities
- Central review of Visual Mobility Assessments
- Name
- Labcorp Central Laboratory Services SARL
- Responsibilities
- Central laboratory testing and sample processing
Third parties
- {"country":"United States","full_name":"Boston Image Reading Center LLC","duties_or_roles":"Medical image analysis/ review - X-ray, MRI, ultrasound, etc.","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Omnitrace Corp.","duties_or_roles":"Patient Locator Services, Patient Lost To Follow-Up, Patient Vital Status Searches","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Central review of Visual Mobility Assessments","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Oregon Health & Science University","duties_or_roles":"Medical image analysis/ review - X-ray, MRI, ultrasound, etc.; licensing the grid for static perimetry","organisation_type":"Educational Institution"}
- {"country":"United States","full_name":"Adelphi Values LLC","duties_or_roles":"Treatment experience interviews","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Emas Pharma Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Syneos Health Netherlands B.V.","duties_or_roles":"IVRS30 – treatment randomisation, Investigators Meetings, Site Contracts, DSMB, 3rd party vendors, Project Management; responsibilities including project management, contracting, IVRS, DSMB coordination and vendor management","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"PT/PTT Research Sample HBV and C testing TB test (Quantiferon) HIV Pregnancy Serum for capsid; central laboratory services","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- JNJ-74765340
- Active Substance
- BOTARETIGENE SPAROPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- SUBRETINAL USE
- Route
- SUBRETINAL
- Orphan Designation
- Yes
- Investigational Product Name
- VANCOMYCIN
- Active Substance
- VANCOMYCIN
- Modality
- Small molecule
- Routes Of Administration
- SUBCONJUNCTIVAL USE
- Route
- SUBCONJUNCTIVAL
- Investigational Product Name
- CEFAZOLIN
- Active Substance
- CEFAZOLIN SODIUM
- Modality
- Small molecule
- Routes Of Administration
- SUBCONJUNCTIVAL USE
- Route
- SUBCONJUNCTIVAL
- Investigational Product Name
- BETAMETHASONE
- Active Substance
- BETAMETHASONE SODIUM PHOSPHATE
- Modality
- Small molecule
- Routes Of Administration
- SUBCONJUNCTIVAL USE
- Route
- SUBCONJUNCTIVAL
- Investigational Product Name
- OMEPRAZOLE
- Active Substance
- OMEPRAZOLE MAGNESIUM
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Investigational Product Name
- DEXAMETHASONE
- Active Substance
- DEXAMETHASONE ACETATE
- Modality
- Small molecule
- Routes Of Administration
- SUBCONJUNCTIVAL USE
- Route
- SUBCONJUNCTIVAL
- Investigational Product Name
- CEFUROXIME
- Active Substance
- CEFUROXIME
- Modality
- Small molecule
- Routes Of Administration
- SUBCONJUNCTIVAL USE
- Route
- SUBCONJUNCTIVAL
- Investigational Product Name
- TRIAMCINOLONE
- Active Substance
- TRIAMCINOLONE ACETONIDE
- Modality
- Small molecule
- Routes Of Administration
- RETROBULBAR USE
- Route
- RETROBULBAR
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