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Gene Therapy Trial Report

Summary

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia


NCTID NCT04483440 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Choroideremia
Disease Ontology Term DOID:9821
Compound Name 4D-110
Compound Description adeno-associated virus serotype 2 capsid variant (4D R100) carrying a transgene encoding a codon-optimized human choroideremia (CHM) gene
Sponsor 4D Molecular Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 13 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant CHM
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV R100
Editor Type none
Dose 1 3E11 vg/eye
Dose 2 1E12 vg/eye

Study Record Dates


Current Stage Phase1
Submit Date 2020-07-20
Completion Date 2027-08-31
Last Update 2025-05-22

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
Key Inclusion Criteria: * Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing * Both eyes must have ≥ 34 ETDRS letters (\~20/200) Key Exclusion Criteria: * Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye * Patient has previously received any AAV treatment * Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates January 2025: Sponsor announced they would terminate development of this program

Resources/Links