Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.

2024-511411-25-00 Protocol MGT-RPGR-022 Therapeutic confirmatory (Phase III) Ongoing, recruitment ended

Start 25 Aug 2022 · Status Ongoing, recruitment ended · 6 EU/EEA countries · 8 sites · Protocol MGT-RPGR-022

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Ongoing, recruitment ended
Participants planned 95
Countries 6
Sites 8

X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene

To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRP.

Key facts

Sponsor
Janssen Cilag International
Participant type
Pediatric, Patients
Age range
0-17 years, 18-64 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Eye Diseases [C11]
Trial duration
25 Aug 2022 → ongoing
Decision date (initial)
2024-06-21
Transition trial
Yes
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
Yes
Funding sources
Janssen Research & Development, LLC

External identifiers

EU CT number
2024-511411-25-00
EudraCT number
2020-002255-37

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Dose response, Efficacy

To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRP.

Conditions and MedDRA coding

X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene

VersionLevelCodeTermSystem organ class
20.0 PT 10038914 Retinitis pigmentosa 100000004850

Regulatory references

Scientific advice from competent authorities
European Medicines Agency
EMA paediatric investigation plan (PIP)
EMEA-002827-PIP01-20
Plan to share IPD
Yes
IPD plan description
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As note on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

  1. 1.Previously completed participation in Study MGT-RPGR-021.
  2. 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 1

  1. There are no specific exclusion criteria.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. 1. Adverse events 2. Laboratory assessments 3. Change from baseline in best corrected visual acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in monocular assessment 4. Change from baseline in low luminance visual acuity (LLVA) by ETDRS chart letter score in monocular assessment

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

JNJ-74765340

PRD11131460 · Product

Active substance
Botaretigene Sparoparvovec
Substance synonyms
A-004, JNJ-74765340, AAV2/5-HRKP.RPGR
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBRETINAL USE
Max daily dose
0 ml millilitre(s)
Max total dose
0 ml millilitre(s)
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
MA holder
JANSSEN-CILAG INTERNATIONAL N.V.
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/16/1715

JNJ-74765340

PRD11131459 · Product

Active substance
Botaretigene Sparoparvovec
Substance synonyms
A-004, JNJ-74765340, AAV2/5-HRKP.RPGR
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBRETINAL USE
Max daily dose
0 ml millilitre(s)
Max total dose
0 ml millilitre(s)
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
MA holder
JANSSEN-CILAG INTERNATIONAL N.V.
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/16/1715

Auxiliary 7

Vancomycin

SCP148257 · ATC

Active substance
Vancomycin
Substance synonyms
VANCOMYCINUM
Route of administration
SUBCONJUNCTIVAL USE
Max daily dose
0 mg milligram(s)
Max total dose
0 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Authorised
ATC code
J01XA01 — VANCOMYCIN
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Local administration of an antibiotic by subconjunctival injection to prevent infection is a standard component of any intraocular surgery and is not specific for this procedure. No antibiotics are approved for administration by subconjunctival administration but local post-operative administration of antibiotic is considered ‘standard of care’.

Triamcinolone Acetonide

SCP131459 · ATC

Active substance
Triamcinolone Acetonide
Substance synonyms
9Α-FLUORO-11Β,21-DIHYDROXY-16Α,17Α-ISOPROPYLIDENEDIOXYPREGNA-1,4-DIENE-3,20-DIONE
Route of administration
RETROBULBAR USE
Max daily dose
0 mg milligram(s)
Max total dose
0 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Authorised
ATC code
H02AB08 — TRIAMCINOLONE
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
This AxMP is being administered by SubTenon injection which is different to the registered route of administration in the Marketing Authorization

Betamethasone Sodium Phosphate

SCP11394729 · ATC

Active substance
Betamethasone Sodium Phosphate
Substance synonyms
BETAMETHASONE DISODIUM PHOSPHATE
Route of administration
SUBCONJUNCTIVAL USE
Max daily dose
0 mg milligram(s)
Max total dose
0 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Authorised
ATC code
H02AB01 — BETAMETHASONE
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Administration of a corticosteroid by subconjunctival injection to prevent local surgical inflammation is a standard component of any intraocular surgery and is not specific for this procedure. No corticosteroids are approved for subconjunctival administration but use of local post-operative corticosteroid is considered ‘standard of care’.

Dexamethasone Acetate

SCP10332310 · ATC

Active substance
Dexamethasone Acetate
Route of administration
SUBCONJUNCTIVAL USE
Max daily dose
0 mg milligram(s)
Max total dose
0 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Authorised
ATC code
H02AB02 — DEXAMETHASONE
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Administration of a corticosteroid by subconjunctival injection to prevent local surgical inflammation is a standard component of any intraocular surgery and is not specific for this procedure. No corticosteroids are approved for subconjunctival administration but use of local post-operative corticosteroid is considered ‘standard of care’.

Cefuroxime

SCP13260501 · ATC

Active substance
Cefuroxime
Route of administration
SUBCONJUNCTIVAL USE
Max daily dose
0 mg milligram(s)
Max total dose
0 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Authorised
ATC code
S01AA27 — CEFUROXIME
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Local administration of an antibiotic by subconjunctival injection to prevent infection is a standard component of any intraocular surgery and is not specific for this procedure. No antibiotics are approved for administration by subconjunctival administration but local post-operative administration of antibiotic is considered ‘standard of care’.

Omeprazole Magnesium

SCP101851519 · ATC

Active substance
Omeprazole Magnesium
Substance synonyms
Omeprazole hemimagnesium
Route of administration
ORAL USE
Max daily dose
0 mg milligram(s)
Max total dose
0 mg milligram(s)
Max treatment duration
8 Week(s)
Authorisation status
Authorised
ATC code
A02BC01 — OMEPRAZOLE
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Omeprazole (or similar H2 antagonists) is recommended in this study for those participants who may have risk factors for gastrointestinal toxicity such as bleeding risk from concomitant NSAID. Although not labeled for this use, H2 antagonists are sometimes used in practice in the setting of prolonged systemic steroid therapy, particularly in patients who have a history of gastric ulcers or are taking concomitant non-steroidal anti-inflammatory agents.

Cefazolin Sodium

SCP107201252 · ATC

Active substance
Cefazolin Sodium
Route of administration
SUBCONJUNCTIVAL USE
Max daily dose
0 mg milligram(s)
Max total dose
0 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Authorised
ATC code
J01DB04 — CEFAZOLIN
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Local administration of an antibiotic by subconjunctival injection to prevent infection is a standard component of any intraocular surgery and is not specific for this procedure. No antibiotics are approved for administration by subconjunctival administration but local post-operative administration of antibiotic is considered ‘standard of care’.

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Janssen Cilag International

84 Total trials 38 Recruiting 44 Ended
Commercial
Sponsor organisation
Janssen Cilag International
Address
Turnhoutseweg 30
City
Beerse
Postcode
2340
Country
Belgium

Scientific contact point

Organisation
Janssen Cilag International
Contact name
CTIS Point of Contact

Public contact point

Organisation
Janssen Cilag International
Contact name
CTIS Point of Contact

Third parties 9

OrganisationCity, countryDuties
Oregon Health & Science University
ORG-100051760
 Portland, United States Other
Omnitrace Corp.
ORG-100045579
 Palm Beach Gardens, United States Other
Labcorp Central Laboratory Services SARL
ORG-100011524
 Meyrin, Switzerland Other, Laboratory analysis
Emas Pharma Limited
ORG-100010010
 Hitchin, United Kingdom Code 8
Bioclinica Inc.
ORG-100033079
 Princeton, United States Other
Boston Image Reading Center LLC
ORG-100046514
 Boston, United States Other
4g Clinical LLC
ORG-100042775
 Wellesley, United States Interactive response technologies (IRT)
Adelphi Values LLC
ORG-100048639
 Boston, United States Other
Syneos Health Netherlands B.V.
ORG-100013861
 Amsterdam, Netherlands On site monitoring, Code 12, Other, Code 2, Code 5, Data management, E-data capture

Locations

6 EU/EEA countries · 8 investigational sites

By country

CountryMS statusPlanned subjectsSites
Belgium Ongoing, recruitment ended 7 1
Denmark Ongoing, recruitment ended 2 1
France Ongoing, recruitment ended 2 1
Italy Ongoing, recruitment ended 4 2
Netherlands Ongoing, recruitment ended 9 2
Spain Ongoing, recruitment ended 7 1
Rest of world
United Kingdom, Israel, United States, Canada, Switzerland
 — 64 —

Investigational sites

Belgium

1 site · Ongoing, recruitment ended
Universitair Ziekenhuis Gent
Ophtalmology, Corneel Heymanslaan 10, 9000, Gent

Denmark

1 site · Ongoing, recruitment ended
Rigshospitalet
Department of Ophthalmology, Valdemar Hansens Vej 1-23, 2600, Glostrup

France

1 site · Ongoing, recruitment ended
Quinze-Vingts National Ophthalmology Hospital
Ophtalmology, 28 Rue De Charenton, 75012, Paris

Italy

2 sites · Ongoing, recruitment ended
Careggi University Hospital
Clinica Oftalmologica, Largo Giovanni Alessandro Brambilla 3, 50134, Florence
Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
UOC Oculistica, Via Sergio Pansini 5, 80131, Naples

Netherlands

2 sites · Ongoing, recruitment ended
Stichting Amsterdam UMC
Opthalmology, Meibergdreef 9, 1105 AZ, Amsterdam
Stichting Radboud universitair medisch centrum
Opthalmology, Geert Grooteplein Zuid 10, 6525 GA, Nijmegen

Spain

1 site · Ongoing, recruitment ended
Hospital Universitario Fundacion Jimenez Diaz
Ophthalmology, Avenida De Los Reyes Catolicos 2, 28040, Madrid

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Belgium 2023-03-06 2023-03-07 2023-08-11
Denmark 2022-08-31 2022-09-07 2023-08-11
France 2023-01-26 2023-02-06 2023-08-11
Italy 2023-05-26 2023-05-29 2023-08-11
Netherlands 2022-08-25 2022-09-05 2023-08-11
Spain 2022-11-08 2023-02-06 2023-08-11

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 56 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protocol 2024-51141-25-00_Rationale for the Use of Concomitant Medication_Redacted 3.0
Protocol (for publication) D1_Protocol 2024-51141-25-00_Redacted Amdt. 4
Protocol (for publication) D1_Protocol Clarification Communication_2024-51141-25-00_redacted N/A
Protocol (for publication) D1_Protocol_2024-51141-25-00_Surgical manual_redacted N/A
Protocol (for publication) D4_Patient facing documents_questionnaire_IVI-A_redacted N/A
Protocol (for publication) D4_Patient facing documents_questionnaire_LLQ_redacted N/A
Protocol (for publication) D4_Patient facing documents_questionnaire_Participant interview_redacted N/A
Protocol (for publication) D4_Patient facing documents_questionnaire_PGI-Daily Activities_redacted N/A
Protocol (for publication) D4_Patient facing documents_questionnaire_PGI-Mobility_redacted N/A
Protocol (for publication) D4_Patient facing documents_questionnaire_PGIC-DA_redacted N/A
Protocol (for publication) D4_Patient facing documents_questionnaire_PGIC-Mob_redacted N/A
Recruitment arrangements (for publication) K1_Recruitment arrangements_blank document_BE 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements_blank document_DK 2.0
Recruitment arrangements (for publication) K1_Recruitment arrangements_blank document_ES 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements_blank document_FRA 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements_Blank_NL 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements_IT N.A
Subject information and informed consent form (for publication) L1_ SIS and ICF Main Adult_DK_Redacted 16.2.0
Subject information and informed consent form (for publication) L1_SIS and ICF Adult_IT_Redacted 16.2.0
Subject information and informed consent form (for publication) L1_SIS and ICF Main_Redacted_ES 16.1.1
Subject information and informed consent form (for publication) L1_SIS and ICF Pregnant Partner_ES 4.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Companion Greenphire_ES 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Companion_NL 2.2.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Participant_BE_DUT 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Participant_BE_ENG 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Participant_BE_FRE 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Companion_BE_DUT 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Companion_BE_ENG 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Companion_BE_FRE 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Companion_FRA_FR 2.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Greenphire Participant_FRA_FR 2.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Main Greenphire_ES 2.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Main RadboudUMC_NL_Redacted 16.2.1
Subject information and informed consent form (for publication) L1_SIS and ICF_Main_BE_DUT_Redacted 16.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Main_BE_ENG_Redacted 16.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Main_BE_FRE_Redacted 16.1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Main_FRA_FR_Redacted 16.1.1
Subject information and informed consent form (for publication) L1_SIS and ICF_Main_NL_Redacted 16.2.1
Subject information and informed consent form (for publication) L1_SIS and ICF_Pregnancy_NL 4.5.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Pregnant Partner_BE_DUT 4.2.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Pregnant Partner_BE_ENG 4.2.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Pregnant Partner_BE_FRE 4.2.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Pregnant Partner_FRA_FR 5.1.0
Subject information and informed consent form (for publication) L1_SIS-ICF Greenphire Companion_IT 2.1.0
Subject information and informed consent form (for publication) L1_SIS-ICF Greenphire Participant_IT 2.1.0
Subject information and informed consent form (for publication) L1_SIS-ICF HIV Testing_IT 2.1.0
Subject information and informed consent form (for publication) L1_SIS-ICF Pregnant Partner_IT 4.2.0
Subject information and informed consent form (for publication) L2_Other Subject information material_Rights_DK 1.0
Synopsis of the protocol (for publication) D1_Protocol synopsis_BE_DUT_2024-51141-25-00_Redacted Amdt. 4
Synopsis of the protocol (for publication) D1_Protocol synopsis_BE_FRE_2024-51141-25-00_Redacted Amdt. 4
Synopsis of the protocol (for publication) D1_Protocol synopsis_BE_GER_2024-51141-25-00_Redacted Amdt. 4
Synopsis of the protocol (for publication) D1_Protocol synopsis_EN_2024-51141-25-00_Redacted Amdt. 4
Synopsis of the protocol (for publication) D1_Protocol synopsis_ES_2024-51141-25-00_Redacted Amdt. 4
Synopsis of the protocol (for publication) D1_Protocol synopsis_FR_2024-51141-25-00_Redacted Amdt. 4
Synopsis of the protocol (for publication) D1_Protocol synopsis_IT_2024-51141-25-00_Redacted Amdt. 4
Synopsis of the protocol (for publication) D1_Protocol synopsis_NL_DUT_2024-51141-25-00_Redacted Amdt. 4

Application history

6 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-05-16 Netherlands Acceptable with conditions
2024-06-21
 2024-06-21
2 SUBSTANTIAL MODIFICATION SM-1 2024-10-04 Netherlands No conclusion
2024-12-09
 2024-12-10
3 SUBSTANTIAL MODIFICATION SM-2 2025-10-09 Netherlands Acceptable
2026-01-06
 2026-01-06
4 NON SUBSTANTIAL MODIFICATION NSM-1 2026-01-22 Netherlands Acceptable
2026-01-06
 2026-01-22
5 SUBSTANTIAL MODIFICATION SM-3 2026-02-18 Acceptable 2026-04-14
6 NON SUBSTANTIAL MODIFICATION NSM-2 2026-06-01 Netherlands Acceptable
2026-01-06
 2026-06-01

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