ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

Part of paid clinical trials in Los Angeles, California.

Sponsor
Atsena Therapeutics Inc.
Study ID
NCT05878860
Phase
PHASE3
Status
Recruiting
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Conditions

  • X-linked Retinoschisis

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • ATSN-201 — BIOLOGICAL
    AAV.SPR-hGRK1-hRS1syn

Study Details

This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).

Key Dates

Start date
Aug 22, 2023
Status verified
Apr 2026
Primary completion
Apr 30, 2028
Completion
Apr 30, 2033

Study Design

Enrollment
97 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1, Low Dose
  • Experimental: Cohort 2, High Dose
  • Experimental: Cohort 3, Mid Dose
  • Experimental: Cohort 4, Low Volume
  • Experimental: Cohort 4, High Volume
  • No Intervention: Cohort 4, Control
  • Experimental: Cohort 5, Pediatric
  • Experimental: Cohort 6, Treatment
  • No Intervention: Cohort 6, Control

Primary Outcome Measure

Part A (Dose Escalation) and Part B (Dose Expansion): Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events [ Time Frame: From baseline to week 52 ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Children's Hospital of Los AngelesLos AngelesCalifornia90027
Aaron Nagiel, MD, PhD (PRINCIPAL_INVESTIGATOR)
Bascom Palmer Eye InstituteMiamiFlorida33136
Byron Lam, MD (PRINCIPAL_INVESTIGATOR)
Oregon Health Sciences UniversityPortlandOregon97239
Lesley Everett, MD, PhD, MPhil (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Tomas Aleman, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By research site
Children's Hospital of Los Angeles· Los Angeles, CABascom Palmer Eye Institute· Miami, FLOregon Health Sciences University· Portland, ORChildren's Hospital of Philadelphia· Philadelphia, PA

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