Study of Subretinally Injected SAR439483 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
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|ClinicalTrials.gov Identifier: NCT03920007|
Recruitment Status : Active, not recruiting
First Posted : April 18, 2019
Last Update Posted : February 19, 2020
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).
To evaluate the efficacy of ascending doses of SAR439483 administered as a unilateral subretinal injection in patients with GUCY2D-LCA.
|Condition or disease||Intervention/treatment||Phase|
|Leber's Congenital Amaurosis||Drug: SAR439483 Drug: SAR439483 Diluent Solution Drug: Prednisone Drug: Triamcinalone Acetonide Drug: 1% Prednisolone Drug: Trimethoprim/polymyxin B||Phase 1 Phase 2|
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|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
SAR439483 single dose according to an ascending dose design (dose escalation phase) or SAR439483 single dose (dose expansion phase)
Pharmaceutical form:Solution for intraocular administration Route of administration: Subretinal injection
Drug: SAR439483 Diluent Solution
Pharmaceutical form:Solution for parenteral use Route of administration: Subretinal injection
Pharmaceutical form:Tablet Route of administration: Oral
Drug: Triamcinalone Acetonide
Pharmaceutical form:Suspension Route of administration: Peri-ocular injection
Drug: 1% Prednisolone
Pharmaceutical form:Suspension Route of administration: Drops
Drug: Trimethoprim/polymyxin B
Pharmaceutical form:Solution Route of administration: Topical
- Number of participants with adverse events (AEs) from baseline up to the end of the observation period [ Time Frame: From baseline to week 52 ]Number of participants with AEs will be summarized in each cohort and overall
- Number of participants with AEs from baseline up to the end of the safety follow-up period [ Time Frame: From baseline to week 104 ]Number of participants with AEs will be summarized in each cohort and overall
- Change in best -corrected visual acuity (BCVA) [ Time Frame: Baseline to week 52 and Baseline to week 104 ]Change in BCVA from baseline in the treated and untreated eye (control)
- Change in sensitivity [ Time Frame: Baseline to week 52 and Baseline to week 104 ]Change in sensitivity from baseline in the treated eye and untreated eye (control) as measured by the full-field stimulus testing
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03920007
|United States, Pennsylvania|
|Investigational Site Number 8400001|
|Philadelphia, Pennsylvania, United States, 19104|
|Study Director:||Clinical Sciences & Operations||Sanofi|