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Active, not recruitingNCT03920007Updated Aug 3, 2026Results posted

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

A Phase 1/2 interventional study of ATSN-101 and ATSN-101 Diluent Solution in Leber Congenital Amaurosis, LCA and LCA1, sponsored by Atsena Therapeutics Inc.. Active, not recruiting at 2 sites in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-08-03.

Sponsored by Atsena Therapeutics Inc. · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
15
Allocation
Not applicable
Ages
6 Years and older
Sex
All
01

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.

Read the detailed description

Study duration per participant is approximately 112 weeks including: an approximately 56-day screening/baseline period, an approximately 52-week study observation period including 1 treatment day, and an approximately 52-week safety follow-up period. The end of study visit will be approximately 260 weeks after the Investigational Medicinal Product (IMP) administration.

The study is separated into 2 parts including a dose escalation phase (Part A) and a dose expansion phase (Part B). In Part B participants will be treated at the maximum tolerated dose (MTD) or maximum administered dose (MAD) determined from Part A.

02

Conditions studied

  • Leber Congenital Amaurosis
  • LCA
  • LCA1

Keywords

  • GUCY2D
03

In context

Leber Congenital Amaurosis

31 studies on the registry are indexed under Leber Congenital Amaurosis; 4 are open to participants now.

This study's enrollment of 15 is close to the median of 15 across 21 interventional studies indexed under Leber Congenital Amaurosis.

Browse Leber Congenital Amaurosis studies →

Lead sponsor

Atsena Therapeutics Inc. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Male or female participant with clinical diagnosis of Leber congenital amaurosis caused by biallelic mutations in the GUCY2D (retinal guanylate cyclase) gene with all of the following: a) Documented mutations in both alleles of the GUCY2D gene per testing in a CLIA-approved laboratory, b) For Cohort 1-3, best corrected visual acuity (BCVA) of 20/200 or worse in the eye to be injected; subsequent cohorts may include BCVA of 20/80 or worse in the eye to be injected, c) Photoreceptor (outer nuclear) layer structure identifiable on an optical coherence tomography (OCT) scan across the central retina.
  • Age ≥18 years for Cohorts 1 through 4, and age ≥ 6 years and \<18 years for Cohort 5.
  • Male and female participants must follow the contraception requirements of the trial.
  • Participants must agree to not donate blood, organs, tissues, cells or sperm for at least three months following ATSN-101 administration.

Exclusion criteria

Exclusion criteria:

  • Complicating systemic diseases (such as medical conditions causing immunosuppression) that would preclude the gene transfer, ocular surgery or planned study procedures.
  • History of human immunodeficiency virus (HIV) infection.
  • Pre-existing eye conditions in the study eye that would preclude the planned surgery or interfere with the assessment and interpretation of study endpoints: for example, glaucoma or optic neuropathy that has resulted in significant visual loss, corneal or lenticular abnormalities or opacities that would preclude view of the fundus or performance of the outcome measures, uveitis, retinopathy and maculopathy that in the opinion of the Investigator are causing significant visual loss.
  • Presence of significant ocular abnormalities in the study eye that in the opinion of the Investigator would preclude the planned surgery, effective safety follow-up, or interfere with the interpretation of study endpoints (eg, glaucoma, corneal or significant lens abnormalities or opacities, pre-existing uveitis, intraocular infection, choroidal neovascularization).
  • Any contraindication to the planned surgical procedure, such as contraindications to the use of anaesthesia or allergy to medications planned in the peri-operative period.
  • Known allergy or hypersensitivity to any component of the investigational medicinal product (IMP), diagnostic agents used during the study or medications planned for use in the peri-operative period, particularly corticosteroids.
  • Women who are pregnant (defined as positive beta-Human Chorionic Gonadotropin (HCG) blood or urine test), lactating or breastfeeding.
  • Any ocular procedure, either planned or performed within 6 months of Day 1, which would interfere with the planned surgery or the interpretation of study endpoints in the opinion of the Principal Investigator (PI).
  • Laboratory test abnormalities or abnormalities in electrocardiogram that in the opinion of the PI would make the participant unsuitable for participation in the study.
  • Significant intercurrent illness or infection during the 28 days prior to enrollment.
  • Current substance use disorder.
  • Use of any investigational agent administered within 5 times the elimination half-life of that investigational agent prior to ATSN-101 administration.
  • Enrollment in any other clinical treatment study, for any condition, including those relating to GUCY2D-LCA, throughout the duration of the ATSN-101 study participation.
  • Use of anticoagulation therapy within two weeks prior to surgery.
  • Use of immunosuppressive medications.
  • Current, planned during the course of this trial, or past (within 5 times the elimination half-life of that therapy prior to ATSN-101 administration) use of anti-viral therapy that would inactivate the investigational agent.
  • Received gene therapy within the last 15 years.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
15 participants (actual)

Study arms

  • Experimental
    ATSN-101

    ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)

    Drug: ATSN-101 · Drug: ATSN-101 Diluent Solution · Drug: Prednisone · Drug: Triamcinalone Acetonide · Drug: 1% Prednisolone · Drug: Trimethoprim/polymyxin B

Interventions

  • DrugATSN-101

    Pharmaceutical form:Solution for intraocular administration Route of administration: Subretinal injection

  • DrugATSN-101 Diluent Solution

    Pharmaceutical form:Solution for parenteral use Route of administration: Subretinal injection

  • DrugPrednisone

    Pharmaceutical form:Tablet Route of administration: Oral

  • DrugTriamcinalone Acetonide

    Pharmaceutical form:Suspension Route of administration: Peri-ocular injection

  • Drug1% Prednisolone

    Pharmaceutical form:Suspension Route of administration: Drops

  • DrugTrimethoprim/polymyxin B

    Pharmaceutical form:Solution Route of administration: Topical

06

What researchers measure

Primary outcomes

  1. Number of Participants With Adverse Events (AEs) From Baseline up to the End of the Observation Period

    Number of participants with treatment-emergent AEs will be summarized in each cohort (this includes cohorts 1 - 5)

    Time frame: From Baseline to Week 52

Secondary outcomes

  1. Change From Baseline of Best-Corrected Visual Acuity (BCVA) Between the Treated and Untreated Eyes

    Mean change from Baseline to Week 52 in BCVA (LogMAR) in the treated eye and untreated eye (control). In logMAR notation (logarithm of the minimum angle of resolution) is interpreted as follows: Lower LogMAR values indicates better vision. Higher LogMAR values indicates worse vision.

    Time frame: Baseline to Week 52

  2. Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Light Adapted

    Mean change from Baseline to Week 52 in sensitivity as measured by the full-field stimulus testing - Light Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

    Time frame: Baseline to Week 52

  3. Mean Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Dark Adapted

    Mean change from Baseline to Week 52 in sensitivity as measured by the full-field stimulus testing - Dark Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

    Time frame: Baseline to Week 52

  4. Number of Participants With Adverse Events (AEs) From Baseline up to the End of the Observation Period

    Number of participants with treatment-emergent AEs will be summarized in each cohort (this includes cohorts 1 - 5)

    Time frame: From Baseline to Week 260

  5. Change From Baseline of Best-Corrected Visual Acuity (BCVA) Between the Treated and Untreated Eyes

    Mean change from Baseline to Week 260 in BCVA (LogMAR) in the treated eye and untreated eye (control). In logMAR notation (logarithm of the minimum angle of resolution) is interpreted as follows: Lower LogMAR values indicates better vision. Higher LogMAR values indicates worse vision.

    Time frame: Baseline to Week 260

  6. Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Light Adapted

    Mean change from Baseline to Week 260 in sensitivity as measured by the full-field stimulus testing - Light Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

    Time frame: Baseline to Week 260

  7. Mean Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Dark Adapted

    Mean change from Baseline to Week 260 in sensitivity as measured by the full-field stimulus testing - Dark Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

    Time frame: Baseline to Week 260

07

Results

Posted Aug 3, 2026

Participant flow

Study screening and enrollment occurred between October 2019 through May 2022. A total of 23 patients were screened, resulting in 2 enrollments at the Casey Eye Institute - Oregon Health and Science University and 13 enrollments at the Scheie Eye Institute - University of Pennsylvania to reach the study's goal of 15 enrolled patients across all cohorts.

Enrollment to Week 52
Participant flow — Enrollment to Week 52
MilestoneATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)
Started33333
Completed33333
Not completed00000
Enrollment to Week 260
Participant flow — Enrollment to Week 260
MilestoneATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)
Started33333
Completed00000
Not completed33333

Outcome measures

PrimaryNumber of Participants With Adverse Events (AEs) From Baseline up to the End of the Observation Period

Number of participants with treatment-emergent AEs will be summarized in each cohort (this includes cohorts 1 - 5)

Time frame:
From Baseline to Week 52
Reported as:
Count of participants · Participants
Number of Participants With Adverse Events (AEs) From Baseline up to the End of the Observation Period
ParticipantsATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)
Number of Participants With Adverse Events (AEs) From Baseline up to the End of the Observation Period33333
SecondaryChange From Baseline of Best-Corrected Visual Acuity (BCVA) Between the Treated and Untreated Eyes

Mean change from Baseline to Week 52 in BCVA (LogMAR) in the treated eye and untreated eye (control). In logMAR notation (logarithm of the minimum angle of resolution) is interpreted as follows: Lower LogMAR values indicates better vision. Higher LogMAR values indicates worse vision.

Time frame:
Baseline to Week 52
Reported as:
Mean · LogMAR
Change From Baseline of Best-Corrected Visual Acuity (BCVA) Between the Treated and Untreated Eyes
LogMARTreated EyeUntreated Eye
ATSN-101 Cohort 1-0.27 ± 0.30-0.19 ± 0.35
ATSN-101 Cohort 2-0.08 ± 0.09-0.04 ± 0.04
ATSN-101 Cohort 3-0.37 ± 0.470.00 ± 0.00
ATSN-101 Cohort 4 (Adult Expansion)-0.12 ± 0.110.03 ± 0.03
ATSN-101 Cohort 5 (Pediatric Expansion)-0.05 ± 0.18-0.01 ± 0.02
ATSN-101 High Dose-0.16 ± 0.250.01 ± 0.03
Statistical analysis
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.42 · Mean difference (final values): -0.07
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.32 · Mean difference (final values): -0.04
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.46 · Mean difference (final values): -0.37
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.10 · Mean difference (final values): -0.15
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.77 · Mean difference (final values): -0.03
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.11 · Mean difference (final values): -0.16
SecondaryChange From Baseline of Sensitivity Between the Treated and Untreated Eye - Light Adapted

Mean change from Baseline to Week 52 in sensitivity as measured by the full-field stimulus testing - Light Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

Time frame:
Baseline to Week 52
Reported as:
Mean · dB
Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Light Adapted
dBTreated EyeUntreated Eye
ATSN-101 Cohort 1 - Blue0.81 ± 3.09-1.11 ± 3.10
ATSN-101 Cohort 2 - Blue1.44 ± 3.103.14 ± 4.21
ATSN-101 Cohort 3 - Blue1.00 ± 0.944.83 ± 2.12
ATSN-101 Cohort 4 (Adult Expansion) - Blue-0.87 ± 6.09-1.51 ± 3.74
ATSN-101 Cohort 5 (Pediatric Expansion) - Blue5.53 ± 14.296.92 ± 10.31
ATSN-101 High Dose - Blue2.00 ± 8.843.24 ± 7.15
ATSN-101 Cohort 1 - Red2.06 ± 2.710.78 ± 2.04
ATSN-101 Cohort 2 - Red3.12 ± 5.721.71 ± 2.89
ATSN-101 Cohort 3 - Red14.20 ± 1.906.67 ± 5.66
ATSN-101 Cohort 4 (Adult Expansion) - Red2.67 ± 2.36-0.17 ± 4.01
ATSN-101 Cohort 5 (Pediatric Expansion) - Red-3.67 ± 0.470.17 ± 2.59
ATSN-101 High Dose - Red4.40 ± 8.222.22 ± 4.78
ATSN-101 Cohort 1 - White1.13 ± 2.801.67 ± 3.33
ATSN-101 Cohort 2 - White1.70 ± 2.520.90 ± 3.73
ATSN-101 Cohort 3 - White2.50 ± 1.413.00 ± 3.54
ATSN-101 Cohort 4 (Adult Expansion) - White3.10 ± 4.000.32 ± 1.74
ATSN-101 Cohort 5 (Pediatric Expansion) - White4.60 ± 13.87-2.00 ± 4.24
ATSN-101 High Dose - White3.51 ± 7.790.42 ± 3.20
Statistical analysis
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.41 · Mean difference (final values): 1.92
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.13 · Mean difference (final values): -1.70
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.14 · Mean difference (final values): -3.83
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.85 · Mean difference (final values): 0.64
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.66 · Mean difference (net): -1.39
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.43 · Mean difference (final values): -1.24
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.32 · Mean difference (final values): 1.28
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.61 · Mean difference (final values): 1.42
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.39 · Mean difference (final values): 7.53
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.25 · Mean difference (final values): 2.83
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.33 · Mean difference (final values): -3.83
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.44 · Mean difference (final values): 2.18
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.76 · Mean difference (final values): -0.53
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.51 · Mean difference (final values): 0.80
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.80 · Mean difference (final values): -0.50
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.19 · Mean difference (final values): 2.78
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.86 · Mean difference (final values): -1.25
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.68 · Mean difference (final values): 0.69
SecondaryMean Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Dark Adapted

Mean change from Baseline to Week 52 in sensitivity as measured by the full-field stimulus testing - Dark Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

Time frame:
Baseline to Week 52
Reported as:
Mean · dB
Mean Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Dark Adapted
dBTreated EyeUntreated Eye
ATSN-101 Cohort 1 - Blue6.89 ± 6.192.78 ± 3.91
ATSN-101 Cohort 2 - Blue2.11 ± 3.34-0.78 ± 3.10
ATSN-101 Cohort 3 - Blue29.92 ± 11.67-2.49 ± 1.63
ATSN-101 Cohort 4 (Adult Expansion) - Blue25.53 ± 21.881.60 ± 6.57
ATSN-101 Cohort 5 (Pediatric Expansion) - Blue6.54 ± 5.952.59 ± 6.27
ATSN-101 High Dose - Blue21.36 ± 17.160.95 ± 5.35
ATSN-101 Cohort 1 - Red6.22 ± 6.051.33 ± 1.76
ATSN-101 Cohort 2 - Red3.33 ± 9.43-4.00 ± 3.77
ATSN-101 Cohort 3 - Red28.67 ± 14.610.17 ± 4.01
ATSN-101 Cohort 4 (Adult Expansion) - Red18.49 ± 11.162.89 ± 4.73
ATSN-101 Cohort 5 (Pediatric Expansion) - Red12.18 ± 10.97-3.69 ± 15.73
ATSN-101 High Dose - Red18.67 ± 12.13-0.26 ± 9.42
ATSN-101 Cohort 1 - White5.37 ± 7.161.22 ± 2.91
ATSN-101 Cohort 2 - White0.89 ± 5.50-0.78 ± 7.30
ATSN-101 Cohort 3 - White28.50 ± 21.21-2.00 ± 4.95
ATSN-101 Cohort 4 (Adult Expansion) - White24.94 ± 20.181.04 ± 5.13
ATSN-101 Cohort 5 (Pediatric Expansion) - White7.17 ± 6.604.20 ± 6.65
ATSN-101 High Dose - White20.88 ± 17.561.08 ± 5.16
Statistical analysis
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.09 · Mean difference (final values): 4.11
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.49 · Mean difference (final values): 2.89
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.14 · Mean difference (final values): 32.40
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.17 · Mean difference (final values): 23.93
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.02 · Mean difference (final values): 1.29
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.03 · Mean difference (final values): 19.88
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.20 · Mean difference (final values): 4.89
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.32 · Mean difference (final values): 7.33
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.16 · Mean difference (final values): 28.50
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.09 · Mean difference (final values): 15.60
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.39 · Mean difference (final values): 15.87
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.01 · Mean difference (final values): 18.93
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.29 · Mean difference (final values): 4.14
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.69 · Mean difference (final values): 1.67
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.23 · Mean difference (final values): 30.50
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.15 · Mean difference (final values): 23.90
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.01 · Mean difference (final values): 2.97
  • Treated Eye vs Untreated Eye · t-test, 2 sided · p = 0.02 · Mean difference (final values): 19.80
SecondaryNumber of Participants With Adverse Events (AEs) From Baseline up to the End of the Observation Period

Number of participants with treatment-emergent AEs will be summarized in each cohort (this includes cohorts 1 - 5)

Time frame:
From Baseline to Week 260

Results for this outcome have not been posted.

SecondaryChange From Baseline of Best-Corrected Visual Acuity (BCVA) Between the Treated and Untreated Eyes

Mean change from Baseline to Week 260 in BCVA (LogMAR) in the treated eye and untreated eye (control). In logMAR notation (logarithm of the minimum angle of resolution) is interpreted as follows: Lower LogMAR values indicates better vision. Higher LogMAR values indicates worse vision.

Time frame:
Baseline to Week 260

Results for this outcome have not been posted.

SecondaryChange From Baseline of Sensitivity Between the Treated and Untreated Eye - Light Adapted

Mean change from Baseline to Week 260 in sensitivity as measured by the full-field stimulus testing - Light Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

Time frame:
Baseline to Week 260

Results for this outcome have not been posted.

SecondaryMean Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Dark Adapted

Mean change from Baseline to Week 260 in sensitivity as measured by the full-field stimulus testing - Dark Adapted in the treated eye and untreated eye. Full-field stimulus testing was performed for three stimulus colors - blue, red, and white.

Time frame:
Baseline to Week 260

Results for this outcome have not been posted.

Adverse events

Collected over From Baseline through Week 52. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
ATSN-101 Cohort 10/3 (0%)0/3 (0%)3/3 (100%)
ATSN-101 Cohort 20/3 (0%)1/3 (33.3%)3/3 (100%)
ATSN-101 Cohort 30/3 (0%)0/3 (0%)3/3 (100%)
ATSN-101 Cohort 4 (Adult Expansion)0/3 (0%)0/3 (0%)3/3 (100%)
ATSN-101 Cohort 5 (Pediatric Expansion)0/3 (0%)1/3 (33.3%)3/3 (100%)
Most frequent serious events
Most frequent serious events
EventATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)
Macular HoleEye disorders0/31/30/30/30/3
EndopthalmitisInfections and infestations0/30/30/30/31/3
Retinal DetachmentEye disorders0/30/30/30/31/3
Most frequent other events
Showing 10 of 23
Most frequent other events
EventATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)
Conjunctival HaemorrhageEye disorders3/33/32/32/33/3
Ocular DiscomfortEye disorders3/33/32/32/32/3
Hypotony Of EyeEye disorders2/32/31/32/31/3
Retinal TearEye disorders1/30/30/30/30/3
Vitreal CellsEye disorders1/30/30/31/31/3
Ocular HypertensionEye disorders1/30/30/30/30/3
NasopharyngitisInfections and infestations0/31/31/31/31/3
Eye InflammationEye disorders0/30/31/31/30/3
Eye PainEye disorders0/30/31/30/31/3
Conjunctival HyperaemiaEye disorders0/30/31/31/31/3

Baseline characteristics

The baseline for a given parameter is defined as the last assessment for this parameter before first administration of the investigational medicinal product (IMP).

Age, Continuous
Age, Continuous(Years)ATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)Total
Mean33.7 ± 11.0623.3 ± 7.5723.7 ± 7.3739.0 ± 32.0814.0 ± 1.7326.7 ± 16.21
Sex: Female, Male
Sex: Female, Male(Participants)ATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)Total
Female2132210
Male120115
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)ATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)Total
Race — American Indian or Alaska Native000000
Race — Asian101103
Race — Black or African American000000
Race — Native Hawaiian or Other Pacific Islander000000
Race — White2322110
Race — Not Reported000022
Race — Unknown000000
Ethnicity
Ethnicity(Participants)ATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)Total
Hispanic or Latino000022
Not Hispanic or Latino3333113
Not Reported000000
Unknown000000
Baseline Height (cm)
Baseline Height (cm)(cm)ATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)Total
Mean165.0 ± 11.27174.7 ± 7.57165.7 ± 1.15163.7 ± 8.33158.0 ± 10.15165.4 ± 9.05
Baseline Weight (kg)
Baseline Weight (kg)(kg)ATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)Total
Mean77.9 ± 26.2969.3 ± 15.6160.5 ± 11.0365.7 ± 18.3454.5 ± 1.4665.6 ± 16.32
Baseline BMI (kg/m^2)
Baseline BMI (kg/m^2)(kg/m^2)ATSN-101 Cohort 1ATSN-101 Cohort 2ATSN-101 Cohort 3ATSN-101 Cohort 4 (Adult Expansion)ATSN-101 Cohort 5 (Pediatric Expansion)Total
Mean28.0 ± 5.4422.6 ± 3.9422.0 ± 3.7024.4 ± 5.6922.1 ± 3.5023.8 ± 4.51
08

Study locations

2 sites
  • Casey Eye Institute - Oregon Health & Science University
    Portland, Oregon 97239, United States
  • Scheie Eye Institute, University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
09

References and documents

Study documents

  • Study protocol · Sep 13, 2023
  • Statistical analysis plan · Jan 8, 2024
  • Informed consent form · Aug 27, 2025

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 3, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03920007
Lead sponsor
Atsena Therapeutics Inc.
Responsible party
Sponsor
First posted
Apr 18, 2019
Start date
Oct 22, 2019
Primary completion
May 19, 2023
Completion
May 19, 2027 (estimated)
Results posted
Aug 3, 2026
Last update
Aug 3, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

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