Contact SCGE

Gene Therapy Trial Report

Summary

A Phase 3 Trial for Intravitreal Injection of 4D-150 in Adults With Diabetic Macular Edema (DME).


NCTID NCT07867080 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication Diabetic Macular Edema (DME)
Disease Ontology Term 🔄 DOID:9191
Drug Product Name 🔄 4D-150
Sponsor 4D Molecular Therapeutics
Funder Type Industry
Recruitment Status
Enrollment Count 514 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 VEGF Trap + VEGF-C RNAi
Therapeutic Modality 🔄 Gene transfer
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Genetic delivery of therapeutic protein
Route of Administration 🔄 Intravitreal
Drug Product Type 🔄 Viral vector
Gene Delivery System Type 🔄 Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV R100
Dose 1 🔄 Phase 1/2 dose escalation: 5E9 vg/eye (n=1); 1E10 vg/eye (n=12); 3E10 vg/eye (n=9)
Dose 2 🔄 Phase 3 dose: 3E10 vg/eye

Study Record Dates


Current Phase Phase3
Submit Date 2026-10-05
Completion Date 2030-01
Last Update 🔄 2026-10-30

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
5.2 Inclusion Criteria Individuals eligible to participate in the trial must meet the following inclusion criteria: General Inclusion Criteria 1. Signed informed consent before any protocol-specified assessment is performed 2. Ability and willingness to comply with the protocol-specified procedures (including IVT injection) and visits, in the Investigator's judgment 3. ≥18 years of age at time of signing informed consent 4. Documented diagnosis of diabetes mellitus (Type 1 or Type 2) 5. WOCBP must agree to use highly effective contraception from the time of informed consent, through duration of trial participation 6. Agree to use a barrier method (e.g., condom) during intercourse for 3 months after Day 1 to prevent fluid transmission; sexually active males should not father a child or donate sperm during this period Ocular Inclusion Criteria 7. Macular thickening secondary to DME involving the center of the fovea (confirmed by the Reading Center) in the study eye 8. Study eye amenable to IVT injection identified 9. Presence of intraretinal fluid and/or subretinal fluid secondary to DME 10. Decreased visual acuity attributable primarily to DME, as determined by the Investigator 11. Demonstrated clinical response to aflibercept and functional stability in the study eye Exclusion Criteria: Individuals who meet any of the following exclusion criteria will not be eligible to participate: Ocular Condition 1. Macular conditions other than DME 2. History or presence of moderate Proliferative Diabetic Retinopathy or worse 3. History of retinal detachment in the study eye 4. History or presence of active inflammation or infection in either eye 5. History or presence of low Intraocular Pressure 6. Uncontrolled glaucoma or intraocular hypertension Ocular Treatments/Interventions: 7. Any prior concomitant treatment for DME 8. History of prior significant ocular surgery or treatment 9. Any prior investigational drug, agent, device, or gene therapy
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 87
Locations United States,China,Japan

Regulatory Information


Has US IND True
FDA Designations 🔄 Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates First patients have been enrolled in the phase 3 pivotal trial

Resources/Links