Study of Subretinally Injected SAR439483 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Study of Subretinally Injected SAR439483 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Brief Summary:
Primary Objective:
To evaluate the safety and tolerability of ascending doses of SAR439483 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 SAR439483 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 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 104 weeks after the Investigational Medicinal Product (IMP) administration.
After completion of the main study (DFI14738), participants may have the option to enroll in a separate long-term follow-up study, in which case they would no longer continue in DFI14738 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 Type: Interventional (Clinical Trial)
Estimated Enrollment: 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Dose Escalation Study of Subretinally Injected SAR439483 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Actual Study Start Date: September 12, 2019
Estimated Primary Completion Date: February 2021
Estimated Study Completion Date: February 2022
Arm:
- Experimental: SAR439483
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Estimated enrolment | 15 |
| Actual Study start date | 12 September 2019 |
| Estimated Study Completion Date | 01 February 2022 |