Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03920007 · ClinicalTrials.gov registry record · Phase 1
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
A Phase 1 study of Leber Congenital Amaurosis and LCA, sponsored by Atsena Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 2
- Study locations
NCT03920007: Active Phase 1 study of Leber Congenital Amaurosis and LCA, sponsored by Atsena Therapeutics.
NCT03920007 is a Phase 1 study of Leber Congenital Amaurosis and LCA that is active but no longer recruiting, run by Atsena Therapeutics. The registered enrollment target is 15 participants, below the 26-participant average among 6 other Leber Congenital Amaurosis trials with a reported enrollment target (42% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03920007, a Phase 1 study of Leber Congenital Amaurosis and LCA, is active but no longer recruiting, sponsored by Atsena Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 2
- Study locations
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.
Primary Outcome
Number of participants with treatment-emergent AEs will be summarized in each cohort (this includes cohorts 1 - 5)
Conditions Studied
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
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2019-09-12 |
| Est. Completion | 2027-05-19 |
| Phase | Phase 1 |
What the registry record for NCT03920007 still lists
NCT03920007 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 26-participant average among 6 other Leber Congenital Amaurosis trials with a reported enrollment target (42% lower).
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.
NCT03920007 reports a single indexed study location in Oregon, Pennsylvania.
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.
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).
Who is sponsoring clinical trial NCT03920007?
This trial is sponsored by Atsena Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Leber Congenital Amaurosis
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
RECRUITING · Phase 1
-
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
ACTIVE NOT RECRUITING · Phase 1
-
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
ACTIVE NOT RECRUITING · Phase 1
-
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
RECRUITING · Phase 3
-
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
ACTIVE NOT RECRUITING · Phase 2
-
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
ACTIVE NOT RECRUITING · Phase 3
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01889381 · 15 participants · Phase 2
Human Craniomaxillofacial Allotransplantation
- NCT02530073 · 15 participants · NA
Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) for CDH
- NCT02535936 · 15 participants · NA
Cortical Plasticity in Spastic Diplegia After Selective Dorsal Rhizotomy
- NCT02918474 · 15 participants · NA
Decision Making Tool in Supporting Decision Making in Contralateral Prophylactic Mastectomy in Patients With Newly Diagnosed Breast Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI