Description: Observational Study in Children and Adults with Stargardt Disease Related Atrophy Secondary to Biallelic Mutations in the ABCA4 Gene.
Pre-Gain (IGS02)
PI: Karl Csaky
Description: A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
ABID-2024
PI: Karl Csaky
Description: Observational study to collect and develop a meticulously curated and diverse Age-Related Macular Degeneration (AMD) benchmark dataset.
Verdant (1497-002)
PI: Karl Csaky
Description: Study to evaluate two regimens of intravitreal BI 771716 against Pegcetacoplan in participants with Geographic Atrophy secondary to Age-related Macular Degeneration.
Jade (1484-0005)
PI: Karl Csaky
Description: Phase II study to evaluate oral BI 1584862 in patients with Geographic Atrophy.
Sienna (R3918-AMD-2326)
PI: Karl Csaky
Description: Phase 3 study to evaluate subcutaneously administered Pozelimab in combination with Cemdisiran or Cemdisiran alone on patients with Geographic Atrophy Secondary to Dry Age-related Macular Degeneration.
Abbvie (M24-204)
PI: Karl Csaky
Description: Exploratory Efficacy of intravitreal ABBV-6628 in Subjects with Geographic Atrophy Secondary to Age-Related Macular Degeneration.
Sanofi (DFI18231)
PI: Karl Csaky
Description: Phase 1/2 Study to Evaluate the One-Time intravitreal dose of SAR446597 in Participants with Geographic Atrophy Secondary To Age-Related Macular Degeneration.
Stellar (ACDN-01-001)
PI: Karl Csaky
Description: Study to Evaluate Subretinal ACDN-01 in Participants with ABCA4- related Retinopathy.
Astellas (7317-CL-003)
PI: Karl Csaky
Description: Phase 1b study to evaluate Subretinal ASP7317 in Senior Adults with Geographic Atrophy Secondary to Dry Age-related Macular Degeneration.
GARDian3 (OCU410ST-101301)
PI: Karl Csaky
Description: A Phase 2/3 pivotal confirmatory study to assess Subretinal OCU410ST in Participants with Stargardt Disease.
Celest (039-101)
PI: Karl Csaky
Description: Two Part, Ascending Dose Followed by a Controlled Trial to assess the Subretinal Administration of AAVB-039 in Participants with Stargardt Disease.
Opti-Gain (CTX001-01)
PI: Karl Csaky
Description: Phase I/II Study to Evaluate CTx001 Administered via a Single Subretinal Injection in Patients with Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Binocular Amblyopia Treatment (Ages 4-10)
PI: Eileen Birch
Description: A study to evaluate the effectiveness of enhanced binocular amblyopia treatments in children with amblyopia
Patch-free Occlusion Therapy (Ages 3-12)
PI: Eileen Birch
Description: Study to evaluate patch-free occlusion therapy compared to standard-of-care occlusion therapy in young children with amblyopia
Video Treatment for Amblyopia (Ages 3-7)
PI: Eileen Birch
Description: Study is to evaluate asynchronous movies compared to standard-of-care occlusion therapy in young children with amblyopia.
Amblyopia Treatment for Children Aged 8 to 12 Years
PI: Eileen Birch
Description: Study to assess the effect of at-home amblyopia treatment with Curesight™ for older children with amblyopia aged 8 to 12 years.
Improving Control of Intermittent Exotropia (Ages 4-10)
PI: Eileen Birch
Description: Study to evaluate the use of dichoptic movies for 8 weeks to improve control alignment in children with intermittent exotropia (IXT).
Landscape (RPGR-005)
PI: Mark Pennesi
Description: A Phase 2 Study to Evaluate Subretinal Administration of Laruparetigene Zovaparvovec Administered Bilaterally In Male Participants with X-Linked Retinitis Pigmentosa.
Luna (SB-421a-006)
PI: Mark Pennesi
Description: A Study to Evaluate intravitreal Ultevursen in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene.
Blue Rock (OpCT-001-101)
PI: Mark Pennesi
Description: A Phase 1/2a study of subretinal administration of OpCT-001 photoreceptor precursor cells derived from iPSCs in patients with primary photoreceptor disease.
Amblyopia Treatment for Children Aged 8 to 12 Years
PI: Mark Pennesi
Description: Study to assess the effect of at-home amblyopia treatment with Curesight™ for older children with amblyopia aged 8 to 12 years.
Improving Control of Intermittent Exotropia (Ages 4-10)
PI: Mark Pennesi
Description: Study to evaluate the use of dichoptic movies for 8 weeks to improve control alignment in children with intermittent exotropia (IXT).
Polaris (SB-CS-001)
PI: Mark Pennesi
Description: An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene.
Stella-1 (NHS-OBS-01)
PI: Karl Csaky
Description: Observational Study in Children and Adults with Stargardt Disease Related Atrophy Secondary to Biallelic Mutations in the ABCA4 Gene.
Astra (SB-007 CS-101)
PI: Mark Pennesi
Description: Phase 1/2 Study to Evaluate a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene.
Stellar (ACDN-01-001)
PI: Karl Csaky
Description: Study to Evaluate Subretinal ACDN-01 in Participants with ABCA4- related Retinopathy.
GARDian3 (OCU410ST-101301)
PI: Karl Csaky
Description: A Phase 2/3 pivotal confirmatory study to assess Subretinal OCU410ST in Participants with Stargardt Disease.
Celest (039-101)
PI: Karl Csaky
Description: Two Part, Ascending Dose Followed by a Controlled Trial to assess the Subretinal Administration of AAVB-039 in Participants with Stargardt Disease.
Binocular Amblyopia Treatment (Ages 4-10)
PI: Eileen Birch
Description: A study to evaluate the effectiveness of enhanced binocular amblyopia treatments in children with amblyopia
Patch-free Occlusion Therapy (Ages 3-12)
PI: Eileen Birch
Description: Study to evaluate patch-free occlusion therapy compared to standard-of-care occlusion therapy in young children with amblyopia
Video Treatment for Amblyopia (Ages 3-7)
PI: Eileen Birch
Description: Study is to evaluate asynchronous movies compared to standard-of-care occlusion therapy in young children with amblyopia.
Amblyopia Treatment for Children Aged 8 to 12 Years
PI: Eileen Birch
Description: Study to assess the effect of at-home amblyopia treatment with Curesight™ for older children with amblyopia aged 8 to 12 years.
Improving Control of Intermittent Exotropia (Ages 4-10)
PI: Eileen Birch
Description: Study to evaluate the use of dichoptic movies for 8 weeks to improve control alignment in children with intermittent exotropia (IXT).
Astellas (2020-CL0102)
PI: Mark Pennesi
Description: An Observational Study of Natural Disease Progression in Pediatric and Adult Participants with Macular Dystrophies Expressing a Stargardt-Type Clinical Presentation.
BEST1 (OPGx-BEST1 DUO-1001)
PI: Mark Pennesi
Description: A Phase 1b/2a, Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-BEST1 Administered in Patients with Either Autosomal-Dominant or Autosomal-Recessive BEST1 Disease.
OPGx-LCA5-1001
PI: Mark Pennesi
Description: Two part, natural history and Phase 1b/2a, Study to Investigate Subretinally Injected OPGx-001 Administered in Patients with LCA5-Associated Inherited Retinal Degeneration.
Lighthouse (ATSN-201-01)
PI: Mark Pennesi
Description: A Phase 1/2/3 Study to Evaluate Subretinal Administration of ATSN-201 Gene Therapy in Subjects with RS1-Associated X-linked Retinoschisis.
ABID-2024
PI: Karl Csaky
Description: Observational study to collect and develop a meticulously curated and diverse Age-Related Macular Degeneration (AMD) benchmark dataset.
Pre-Gain (IGS02)
PI: Karl Csaky
Description: A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD).
Verdant (1497-002)
PI: Karl Csaky
Description: Study to evaluate two regimens of intravitreal BI 771716 against Pegcetacoplan in participants with Geographic Atrophy secondary to Age-related Macular Degeneration.
Jade (1484-0005)
PI: Karl Csaky
Description: Phase II study to evaluate oral BI 1584862 in patients with Geographic Atrophy.
Sienna (R3918-AMD-2326)
PI: Karl Csaky
Description: Phase 3 study to evaluate subcutaneously administered Pozelimab in combination with Cemdisiran or Cemdisiran alone on patients with Geographic Atrophy Secondary to Dry Age-related Macular Degeneration.
Abbvie (M24-204)
PI: Karl Csaky
Description: Exploratory Efficacy of intravitreal ABBV-6628 in Subjects with Geographic Atrophy Secondary to Age-Related Macular Degeneration.
Sanofi (DFI18231)
PI: Karl Csaky
Description: Phase 1/2 Study to Evaluate the One-Time intravitreal dose of SAR446597 in Participants with Geographic Atrophy Secondary To Age-Related Macular Degeneration.
Astellas (7317-CL-003)
PI: Karl Csaky
Description: Phase 1b study to evaluate Subretinal ASP7317 in Senior Adults with Geographic Atrophy Secondary to Dry Age-related Macular Degeneration.
Opti-Gain (CTX001-01)
PI: Karl Csaky
Description: Phase I/II Study to Evaluate CTx001 Administered via a Single Subretinal Injection in Patients with Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Vienna (R3918-AMD-2501)
PI: Karl Csaky
Description: A Phase 1 Study Intravitreal Pozelimab in Participants with Geographic Atrophy.
Description: A Phase 2 Study to Evaluate Subretinal Administration of Laruparetigene Zovaparvovec Administered Bilaterally In Male Participants with X-Linked Retinitis Pigmentosa.
Description: An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene.
Description: Phase 1/2 Study to Evaluate a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene.
Description: An Observational Study of Natural Disease Progression in Pediatric and Adult Participants with Macular Dystrophies Expressing a Stargardt-Type Clinical Presentation.
