Ignite Creation Date:
2024-05-06 @ 1:05 PM
Last Modification Date:
2024-10-26 @ 1:08 PM
Study NCT ID:
NCT03920007
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-02-20
First Post:
2019-04-10
Brief Title:
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Sponsor:
Atsena Therapeutics Inc
Organization:
Atsena Therapeutics Inc
Study Overview
Official Title:
A Phase 12 Dose Escalation Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
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
Detailed Description:
Study duration per participant is approximately 112 weeks including an approximately 56-day screeningbaseline 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
After completion of the main study ATSN-101-1 participants may have the option to enroll in a separate long-term follow-up study in which case they would no longer continue in ATSN-101-1 and their end of study visit would be conducted at Week 52
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
After completion of the main study ATSN-101-1 participants may have the option to enroll in a separate long-term follow-up study in which case they would no longer continue in ATSN-101-1 and their end of study visit would be conducted at Week 52
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| U1111-1200-1308 | OTHER | UTN | None |