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Open Clinical Trials and Research Studies

To fulfill our mission to save and restore vision through innovative research and treatment, the Retina Foundation seeks volunteers for clinical trials and research studies. Patients volunteer to further research, and they are never charged for any treatment or care they receive. The Retina Foundation is currently conducting a wide variety of clinical trials and research studies in macular degeneration, inherited retinal degeneration and pediatric disorders.
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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.

My Retina Tracker (MRT)

  • PI: Mark Pennesi
  • Description: Genetic Testing Study and Patient driven Registry for patients with Inherited Retinal Diseases (IRDs).

UNI-Rare (Foundation for Fighting Blindness)

  • PI: Mark Pennesi
  • Description: Natural History Universal Rare Gene Study of Retinal Dystrophies Associated with Rare Disease-Causing Genetic Variants.

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.

Inherited Retinal Diseases:

Landscape RPGR-005 (Ages 12+)

  • 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 (Ages 8+)

  • 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.

Polaris SB-CS-001 (Ages 12+)

  • 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.

Astra SB-007 CS-101 Part B (Ages 12+)

  • 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.

Astellas 2020-CL0102 (Ages 6+)

  • 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.

OPGx-LCA5-1001 Part B (Ages 4+)

  • PI: Mark Pennesi
  • Description: Natural History Study of Patients with LCA5-Associated Inherited Retinal Degeneration.

My Retina Tracker (All Ages)

  • PI: Mark Pennesi
  • Description: Genetic Testing Study and Patient driven Registry for patients with Inherited Retinal Diseases (IRDs).

UNI-Rare Foundation for Fighting Blindness (Ages 4+)

  • PI: Mark Pennesi
  • Description: Natural History Universal Rare Gene Study of Retinal Dystrophies Associated with Rare Disease-Causing Genetic Variants.

Amblyopia/Strabismus:

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).