Clinical trial • Phase III • Ophthalmology
TIMREPIGENE EMPARVOVEC for Choroideremia | X-linked retinitis pigmentosa
Phase III trial of TIMREPIGENE EMPARVOVEC for Choroideremia | X-linked retinitis pigmentosa.
Overview
- Trial Therapeutic Area
- Ophthalmology
- Trial Disease
- Choroideremia | X-linked retinitis pigmentosa
- Trial Stage
- Phase III
- Drug Modality
- Gene therapy
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 18-09-2024
- First CTIS Authorization Date
- 01-10-2024
Trial design
Untreated control participants (participants who exited the STAR antecedent study); no drug, dose or schedule specified Phase III trial across 3 sites in France, Germany.
- Comparator
- Untreated control participants (participants who exited the STAR antecedent study); no drug, dose or schedule specified
- Target Sample Size
- 276
- Trial Duration For Participant
- 2191
Eligibility
Recruits 276 No vulnerable population selected (isVulnerablePopulationSelected: false). Participants must be willing and able to give informed consent (inclusion criteria specify willingness and ability to give informed consent). Caregiver informed consent forms are present in the document list (Caregiver ICF / caregiver ICF documents), indicating caregiver-specific materials are available where applicable..
- Vulnerable Population
- No vulnerable population selected (isVulnerablePopulationSelected: false). Participants must be willing and able to give informed consent (inclusion criteria specify willingness and ability to give informed consent). Caregiver informed consent forms are present in the document list (Caregiver ICF / caregiver ICF documents), indicating caregiver-specific materials are available where applicable.
Inclusion criteria
- {"criterion_text":"- CHM Participants: a. Are willing and able to give informed consent for participation in the study, and\n- CHM Participants: b. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of AAV2- REP1 for CHM\n- XLRP Participants: a. Are willing and able to give informed consent for participation in the study\n- XLRP Participants: b. Have received a sub-retinal injection of AAV8-RPGR for XLRP and have exited an antecedent study"}
Exclusion criteria
- {"criterion_text":"- In the opinion of the investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Safety assessments will be performed as per protocol","definition_or_measurement_approach":"Safety assessments performed as per protocol (no further detail provided in the available record)"}
Secondary endpoints
- {"endpoint_text":"- Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart\n- Proportion of participants with no decrease from Baseline in BCVA or a decrease from Baseline in BCVA of <5 ETDRS letters (in CHM subjects only)\n- Proportion of participants with an increase from Baseline in BCVA of ≥ 10 ETDRS letters (in CHM subjects only)\n- Proportion of participants with an increase from Baseline in BCVA of ≥ 15 ETDRS letters (in CHM subjects only)\n- Available assessments of fundus autofluorescence at each visit\n- Available assessments of fundus photography at each visit\n- Available assessments of spectral-domain optical coherence tomography (SD-OCT) at each visit\n- Available assessments of microperimetry at each visit\n- Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25)\n- Change from Baseline in visual field (in AAV8-RPGR-treated participants only)\n- Proportion of participants with an increase from Baseline in low luminance visual acuity (LLVA) of ≥10 ETDRS letters (in AAV8-RPGR-treated participants only)\n- Proportion of participants with an increase from Baseline in LLVA of ≥ 15 ETDRS letters (in AAV8-RPGR-treated participants only)\n- Datasets from the 2 disease populations will be analyzed separately.","definition_or_measurement_approach":"BCVA measured by ETDRS chart where stated; many endpoints defined as change from Baseline or proportion meeting a change threshold (ETDRS letters). Other endpoints are availability of imaging/functional assessments at each visit (fundus autofluorescence, fundus photography, SD-OCT, microperimetry). VFQ-25 change from Baseline as patient-reported outcome. Visual field and LLVA endpoints applied to specified treatment populations as noted."}
Recruitment
- Planned Sample Size
- 276
- Recruitment Window Months
- 72
- Consent Approach
- Participants must be willing and able to give informed consent (inclusion criteria). Subject information and informed consent forms (SIS and ICF) and caregiver ICF documents are present in the document list for participating Member States. No assent procedures or age-specific consent for minors are described in the available record; no languages for consent forms are explicitly listed in the extracted content.
Geography
- Total Number Of Sites
- 3
- Total Number Of Participants
- 54
France
- Earliest CTIS Part Ii Submission Date
- 11-09-2024
- Latest Decision Or Authorization Date
- 01-10-2024
- Processing Time Days
- 20
- Number Of Sites
- 2
- Number Of Participants
- 31
Sites
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- Ophtalmology
- Contact Person Name
- Isabelle MEUNIER
- Contact Person Email
- i-meunier@chu-montpellier.fr
- Site Name
- CHNO des Quinze Vingts Paris
- Department Name
- Ophtalmology
- Contact Person Name
- Isabelle AUDO
- Contact Person Email
- isabelle.audo@inserm.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 11-09-2024
- Latest Decision Or Authorization Date
- 07-10-2024
- Processing Time Days
- 26
- Number Of Sites
- 1
- Number Of Participants
- 23
Sites
- Site Name
- Universitaetsklinikum Tuebingen AöR
- Department Name
- Forschungsinstitut für Augenheilkunde
- Contact Person Name
- Dominik Fischer
- Contact Person Email
- Dominik.Fischer@med.uni-tuebingen.de
Sponsor
Primary sponsor
- Full Name
- Biogen Idec Research Limited
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United Kingdom
Contract research organisations
- Name
- Labcorp Early Development Laboratories Limited
- Responsibilities
- ELISA and ELISPOT immunoassays
- Name
- Medidata Solutions Inc.
- Responsibilities
- Sponsor duties codes: 7
- Name
- Fortrea Belgium
- Responsibilities
- Sponsor duties codes: 1, 10, 12, 2
- Name
- Immunologix
- Responsibilities
- Serum ADA Analysis
Third parties
- {"country":"United Kingdom","full_name":"Labcorp Early Development Laboratories Limited","duties_or_roles":"ELISA and ELISPOT immunoassays","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Sponsor duties codes: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Belgium","full_name":"Fortrea Belgium","duties_or_roles":"Sponsor duties codes: 1, 10, 12, 2","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Immunologix","duties_or_roles":"Serum ADA Analysis","organisation_type":"Health care"}
Investigational products
- Investigational Product Name
- BIIB111
- Active Substance
- TIMREPIGENE EMPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- Sub-retinal injection (intraocular use)
- Route
- Sub-retinal injection (intraocular)
- Orphan Designation
- Yes
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