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Gene Therapy Trial Report

Summary

Study of EBT-101 in Aviremic HIV-1 Infected Adults on Stable ART


NCTID NCT05144386 (View at clinicaltrials.gov)
Description
Development Status Active
Indication HIV-1-infection
Disease Ontology Term DOID:526
Compound Name EBT-101
Compound Description AAV9-SaCas9-GagD-LTR1
Sponsor Excision BioTherapeutics
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 6 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant HIV genes
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene excision
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Lymphoid tissues
Delivery System Viral transduction
Vector Type AAV9
Editor Type Cas9
Dose 1 0.9E12 vg/kg
Dose 2 3.0E12 vg/kg

Study Record Dates


Current Stage Phase1
Submit Date 2021-11-22
Completion Date 2024-11-14
Last Update 2024-12-02

Participation Criteria


Eligible Age 18 Years - 70 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria (abbreviated): * Willing to enroll and sign the written informed consent for EBT-101-001 (current study) and EBT-101-002, the LTFU study. * Age between 18 and 70 years (both inclusive). * Weight: Cohort A ≥55 and ≤110 kg, Cohort B ≥55 and ≤90 kg. * Cohorts A and B will only enroll male subjects (sex at birth). * Chronic HIV-1 with known subtype B infection * On stable regimen defined as continuous ART suppressive treatment with HIV RNA level below the level of quantitation for \>1 years prior to screening * Plasma HIV-1 RNA levels below the limit of quantitation during screening. * Peripheral blood CD4 T cell count ≥500 cells/mm3 for at least 2 years prior to screening * Willing and able to comply, as assessed by the Investigator, with all study-related procedures. * Have previously been vaccinated for N. meningitidis with documented history and/or received a N. meningitidis vaccination prior to dosing. * Willing to stop ART if eligible for analytical treatment interruption. * Willing to comply with the measures to prevent HIV transmission and reinfection required by the protocol. * Must have received a COVID-19 vaccination series and boosters as specified by current federal (CDC) recommendations, with the last dose ≥30 days prior to dosing. Exclusion Criteria (abbreviated): * Documented prior HIV-1 drug resistance to ≥2 or more classes of ART defined as single key mutations or an accumulation of minor mutations that result in resistance to entire respective drug classes within the past 5 years. * History of \>1 change in ART due to virologic failure during preceding 2 years prior to screening. * Received in the preceding 12 months or HCP plans to prescribe long-acting injectable ART. * History of HIV dementia. * History of progressive multifocal leukoencephalopathy. * History of significant cardiac disease in last 2 years. * History of HIV-related kidney disease with abnormal renal function. * Known history and/or documented: pre-HIV treatment nadir CD4+ T cell count \<200 cells/mm3 or post-suppressive HIV treatment confirmed CD4+ T cell \<200 cells/mm3 prior to screening * History of AIDS-defining opportunistic infection prior to screening. * Evidence of acute or chronic hepatitis B and/or hepatitis C. * Known history or diagnosis of liver cirrhosis. * Diagnosis of nonalcoholic fatty liver or advanced nonalcoholic steatohepatitis. * Predefined abnormal laboratory values within 42 days of dosing per protocol * Known history of positive tuberculin skin test. * Receipt of any investigational HIV vaccine (prophylactic and/or therapeutic) within the year prior to screening. * Receipt of any gene therapy product approved or experimental, at any time. * Anti-AAV9 serum neutralizing antibodies (Nabs) \>1:20 titer. * Known positive SARS-CoV-2 test within 48 hours prior to planned dosing date.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Regenerative Medicine Advanced Therapy
Recent Updates Safe, but does not provide long-term viral suppression

Resources/Links