Informations générales (source: ClinicalTrials.gov)
A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa (PIONEER)
Interventional
Phase 1/Phase 2
GenSight Biologics (Voir sur ClinicalTrials)
septembre 2018
décembre 2025
29 juin 2024
The objective of this study is to evaluate the safety and tolerability of escalating
doses of a gene therapy called GS030-DP (injected study treatment) administered via a
single intravitreal injection and repeated light stimulation using a medical device
called GS030-MD (stimulating glasses) in subjects with documented diagnosis of
non-syndromic Retinitis Pigmentosa
Etablissements
| Les établissements d'Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CHNO DES QUINZE-VINGTS PARIS | Elise BOULANGER-SCEMAMA, MD | Contact (sur clinicalTrials) | |||
Critères
Tous
Main selection criteria:
- Age ≥18 years to ≤75 years at the time of ICF signature.
- Diagnosis of non-syndromic RP defined as:
- Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual
dysfunction, and fundoscopic appearance.
- Diagnosis of non-syndromic RP is confirmed on full-field ERG
- Visual acuity:
- Visual acuity in the dose-escalation cohorts of no better LP.
- Visual acuity in the extension cohort of no better than CF pending review of
dose-escalation cohort data by the DSMB.
- Relatively preserved ganglion cell layer volume and retinal nerve fiber layer
thickness, as measured with spectral domain optical coherence tomography (SD-OCT).
- Interpupillary distance of ≥51 mm and ≤72 mm.
- Refractive error of the study eye between -6 diopters and +6 diopters.
Main non-selection criteria
- Prior receipt of any gene therapy.
- Subjects who have undergone significant ocular surgery (per investigator
determination) within 3 months prior to Visit 1.
- Presence of narrow iridocorneal angles contraindicating pupillary dilation.
- Presence of disorders of the ocular media which interfere with visual acuity and
other ocular assessments, including SD-OCT, during the study period.
- Presence of any systemic or ocular diseases, or pathologies, other than
non-syndromic RP, or their associated therapies, that can cause or have the
potential to cause vision loss.
- Prior vitrectomy or vitreomacular surgery.
- Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker
and macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations and
assessed by the investigator to significantly affect central vision.
- Current evidence of retinal detachment assessed by the investigator to significantly
affect central vision.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune
associated uveitis.
- Presence of an Active Implantable Medical Device.
- Subjects who have undergone thermal laser procedure to the retina within 3 months of
trial entry, or any prior thermal laser procedure to the macular region.
- Age ≥18 years to ≤75 years at the time of ICF signature.
- Diagnosis of non-syndromic RP defined as:
- Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual
dysfunction, and fundoscopic appearance.
- Diagnosis of non-syndromic RP is confirmed on full-field ERG
- Visual acuity:
- Visual acuity in the dose-escalation cohorts of no better LP.
- Visual acuity in the extension cohort of no better than CF pending review of
dose-escalation cohort data by the DSMB.
- Relatively preserved ganglion cell layer volume and retinal nerve fiber layer
thickness, as measured with spectral domain optical coherence tomography (SD-OCT).
- Interpupillary distance of ≥51 mm and ≤72 mm.
- Refractive error of the study eye between -6 diopters and +6 diopters.
Main non-selection criteria
- Prior receipt of any gene therapy.
- Subjects who have undergone significant ocular surgery (per investigator
determination) within 3 months prior to Visit 1.
- Presence of narrow iridocorneal angles contraindicating pupillary dilation.
- Presence of disorders of the ocular media which interfere with visual acuity and
other ocular assessments, including SD-OCT, during the study period.
- Presence of any systemic or ocular diseases, or pathologies, other than
non-syndromic RP, or their associated therapies, that can cause or have the
potential to cause vision loss.
- Prior vitrectomy or vitreomacular surgery.
- Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker
and macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations and
assessed by the investigator to significantly affect central vision.
- Current evidence of retinal detachment assessed by the investigator to significantly
affect central vision.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune
associated uveitis.
- Presence of an Active Implantable Medical Device.
- Subjects who have undergone thermal laser procedure to the retina within 3 months of
trial entry, or any prior thermal laser procedure to the macular region.