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Gene Therapy Trial Report

Summary

ADVM-022 Intravitreal Gene Therapy for DME


NCTID NCT04418427 (View at clinicaltrials.gov)
Description
Development Status 🔄 Inactive
Indication Diabetic Macular Edema, Diabetic Retinopathy
Disease Ontology Term 🔄 DOID:9191
Drug Product Name 🔄 Ixoberogene soroparvovec
Drug Product Alias 🔄 ADVM-022
Drug Product Description 🔄 AAV.7m8-aflibercept
Sponsor Adverum Biotechnologies, Inc.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 36 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant 🔄 Codon optimized aflibercept
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 AAV2.7m8
Dose 1 🔄 2E11 vg/eye (n=9)
Dose 2 🔄 6E11 vg/eye (n=12), DLT observed at this level

Study Record Dates


Current Phase Phase2
Submit Date 2020-05-28
Completion Date 2023-06-14
Last Update 2025-07-10

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Age ≥ 18 * Type 1 or Type 2 diabetes mellitus * Willing and able to provide informed consent * Vision impairment due to center involving diabetic macular edema Exclusion Criteria: * Uncontrolled diabetes defined as HbA1C \>10%, or history of diabetic ketoacidosis within 3 months prior to randomization; or subjects who, within the last 3 months, initiated intensive insulin treatment (a pump or multiple daily injection) or plan to do so in the next 3 months. * Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months * Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg * Known severe renal impairment * High risk Proliferative Diabetic Retinopathy * History of retinal disease in the study eye other than diabetic retinopathy * History of retinal detachment (with or without repair) in the study eye * History of vitrectomy, trabeculectomy, or other filtration surgery in the study eye * Any prior focal or grid laser photocoagulation or any prior PRP in the study eye * Current or planned pregnancy or breastfeeding
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 14
Locations Puerto Rico,United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Development of ADVM-022 for DME indication was halted after a dose-limited toxicity event in the high dose group, Adverum continues to develop this drug product for use in nAMD

Resources/Links