Trials Sponsors Conditions Drugs Pricing

Conditions

Leber Congenital Amaurosis LCA LCA1

Keywords

GUCY2D

Outcome Measures

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)
From Baseline to Week 52

Secondary Outcomes (7)

Change From Baseline of Best-Corrected Visual Acuity (BCVA) Between the Treated and Untreated Eyes
Baseline to Week 52
Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Light Adapted
Baseline to Week 52
Mean Change From Baseline of Sensitivity Between the Treated and Untreated Eye - Dark Adapted
Baseline to Week 52
Number of Participants With Adverse Events (AEs) From Baseline up to the End of the Observation Period
From Baseline to Week 260
Change From Baseline of Best-Corrected Visual Acuity (BCVA) Between the Treated and Untreated Eyes
Baseline to Week 260
+2 more secondary outcomes

Study Arms (1)

ATSN-101

EXPERIMENTAL

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

Drug: ATSN-101Drug: ATSN-101 Diluent SolutionDrug: PrednisoneDrug: Triamcinalone AcetonideDrug: 1% PrednisoloneDrug: Trimethoprim/polymyxin B

Interventions

ATSN-101Drug

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

ATSN-101

ATSN-101 Diluent SolutionDrug

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

ATSN-101

Registry intervention entries

These entries preserve how the registry describes the intervention. Their treatment roles are shown separately from molecular identities.

Grouped treatment entry
Trimethoprim/polymyxin B
The registry names several treatments. This does not confirm that participants received them together or that they form a single product.
View registry record

Eligibility Criteria

Age6 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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.

Sponsors & Collaborators

Atsena Therapeutics Inc.
Industrylead

Study Sites (2)

Casey Eye Institute - Oregon Health & Science University

Portland, Oregon, 97239, United States

Scheie Eye Institute, University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

MeSH Terms

Conditions

Leber Congenital AmaurosisRetinal cone dystrophy 2

Interventions

PrednisonePrednisoloneTrimethoprimPolymyxin B

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal Diseases

Intervention Hierarchy (Ancestors)

PregnadienediolsPregnadienesPregnanesSteroids

Results Point of Contact

Title: Dan Yoon, MD, Sr. Director of Clinical Development
Organization: Atsena Therapeutics

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: GT60
Restrictive Agreement: Yes

Enrollment

15 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

April 10, 2019

First Posted

April 18, 2019

Study Start

October 22, 2019

Actual (reported)

You may not qualify if:

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.
+3 more criteria

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Results Posted

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (7)

Study Arms (1)

ATSN-101

Interventions

Registry intervention entries

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Results Posted

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (7)

Study Arms (1)

ATSN-101

Interventions

Registry intervention entries

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations