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      "canonicalId": "NCT06467344",
      "title": "Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)",
      "scientificTitle": "ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy",
      "status": "recruiting",
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        "PHASE2"
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        "Cone Rod Dystrophy",
        "Juvenile Macular Degeneration",
        "Stargardt Disease 1"
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          "name": "ACDN-01",
          "type": "DRUG",
          "description": "ACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection."
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        "name": "Ascidian Therapeutics, Inc",
        "type": "INDUSTRY"
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      "locations": [
        {
          "city": "San Francisco",
          "country": "United States",
          "facility": "University of San Francisco"
        },
        {
          "city": "Gainesville",
          "country": "United States",
          "facility": "Vitreo Retinal Associates"
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        {
          "city": "Miami",
          "country": "United States",
          "facility": "Bascom Palmer Eye Institute"
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        {
          "city": "Baltimore",
          "country": "United States",
          "facility": "Wilmer Eye Institute at John Hopkins"
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          "country": "United States",
          "facility": "Massachusetts Eye and Ear"
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          "country": "United States",
          "facility": "University of Michigan Kellogg Eye Center"
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          "city": "Cincinnati",
          "country": "United States",
          "facility": "Cincinnati Eye Institute"
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          "city": "Portland",
          "country": "United States",
          "facility": "Casey Eye Institute OHSU"
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          "city": "Philadelphia",
          "country": "United States",
          "facility": "Children's Hospital of Philadelphia"
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          "city": "Dallas",
          "country": "United States",
          "facility": "Retina Foundation of the Southwest"
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          "city": "Houston",
          "country": "United States",
          "facility": "Retina Consultants of Texas"
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          "To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.",
          "To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.",
          "To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.",
          "To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.",
          "To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.",
          "To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry."
        ],
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        ],
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          "Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.",
          "To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).",
          "To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT)."
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