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ACTIVE NOT RECRUITING Phase 1

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

NCT03920007 · View on ClinicalTrials.gov ↗

Study Summary

Primary Objective: To evaluate the safety and tolerability of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate cyclase 2D (GUCY2D) mutations (GUCY2D-LCA). Secondary Objective: To evaluate the efficacy of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with GUCY2D-LCA.

Interventions

  • DRUG Prednisone
  • DRUG ATSN-101
  • DRUG ATSN-101 Diluent Solution
  • DRUG Triamcinalone Acetonide
  • DRUG 1% Prednisolone

Study Locations (2)

Oregon

  • Casey Eye Institute - Oregon Health & Science University — Portland

Pennsylvania

  • Scheie Eye Institute, University of Pennsylvania — Philadelphia

Trial Details

FieldValue
Enrollment Target 15 participants
Start Date 2019-09-12
Est. Completion 2027-05-19
Phase Phase 1

Sponsor

Atsena Therapeutics

2 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03920007

The ClinicalTrials.gov registry entry for NCT03920007 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Atsena Therapeutics, which has 2 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 3 conditions, with Leber Congenital Amaurosis appearing as the primary indexed condition, and to 5 interventions — of which Prednisone is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03920007 reports 2 study locations spanning 2 distinct geographic areas — top geographies include Oregon, Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT03920007 about?

NCT03920007 is a clinical study titled "Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D". Primary Objective: To evaluate the safety and tolerability of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate cyclase 2D (GUCY2D) mutations (GUCY2D-LCA). Secondary Objective: To...

What is the current status of trial NCT03920007?

This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2019-09-12. Estimated completion is 2027-05-19.

What conditions does trial NCT03920007 study?

This clinical trial studies the following conditions: Leber Congenital Amaurosis, LCA, LCA1. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03920007?

The interventions under investigation include: Prednisone (DRUG), ATSN-101 (DRUG), ATSN-101 Diluent Solution (DRUG), Triamcinalone Acetonide (DRUG), 1% Prednisolone (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03920007?

This trial is sponsored by Atsena Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT03920007 being conducted?

This trial has 2 study locations across Oregon, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial