Summary

Eligibility
for males ages 18-65 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Priscilla Hsue
Headshot of Priscilla Hsue
Priscilla Hsue

Description

Summary

This is a First in Human (FIH) study of EBT-101 administered IV to aviremic HIV-1 infected adults on stable antiretroviral therapy (ART).

Official Title

A Phase 1/2a, Sequential Cohort, Single Ascending Dose Study of the Safety, Tolerability, Biodistribution, and Pharmacodynamics of EBT 101 in Aviremic HIV-1 Infected Adults on Stable Antiretroviral Therapy

Details

This is a FIH, open-label, sequential cohort, single ascending dose (SAD) study of EBT-101 administered IV to aviremic HIV-1 infected adults on stable ART. Participants will be asked to attend several visits for screening to determine eligibility. On Day 1, eligible participants will receive a single IV dose of EBT-101. All participants will be assessed for eligibility for an analytical treatment interruption (ATI) of their background ART at Week 12. All participants will be followed through Week 48 (end of study). Participants are required to attend multiple study visits at the clinical site including daily visits for the first 14 days, followed by weekly visits from Week 12 to Week 48. Non-ATI participants are followed less frequently after Week 12. Eligible participants who are enrolled in the FIH study (EBT-101-001) will also be enrolled in a separate Long Term Follow Up (LTFU) study (EBT-101-002) for safety monitoring. The duration of the LTFU study will be up to 15 years.

Keywords

HIV-1-infection, CRISPR, Gene Therapy, AAV9, HIV, EBT-101

Eligibility

You can join if…

Open to males ages 18-65

(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 65 years (both inclusive).
  • Body weight ≥45 and ≤90 kg.
  • Cohort A will only enroll male subjects (sex at birth).
  • Chronic HIV-1 infection
  • On stable regimen defined as continuous ART treatment for >2 years prior to screening; no changes in regimen or missed doses for ≤90 days prior to the planned dosing date.
  • Plasma HIV-1 RNA levels below the limit of quantification: on all available results in past 12 months (isolated single values ≥20 but <200 copies/mL will be allowed if they were preceded and followed by undetectable viral load results); within 42 days prior to the planned dosing date
  • Peripheral blood CD4 T cell count >350 cells/mm3 within the last 2 years prior to screening
  • Normal laboratory values in hematology, hepatic, renal and other systems for lab safety 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.

You CAN'T join if...

(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.
  • Received in the preceding 12 months or HCP plans to prescribe long-acting injectable ART.
  • Pregnant or breastfeeding or planning to become pregnant (self or partner) or to breastfeed at any time through 48 weeks post dose.
  • 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.
  • History of one or more HIV-related opportunistic infections or within the past 2 years.
  • 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.
  • 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.

Locations

  • University of California San Francisco accepting new patients
    San Francisco California 94110 United States
  • Washington University accepting new patients
    Saint Louis Missouri 63110 United States

Lead Scientist at UCSF

  • Priscilla Hsue
    Professional Experience: Dr. Hsue trained in Internal Medicine in the Molecular Medicine Training Program at UC San Francisco and in Cardiovascular Medicine at UC San Francisco. She served as Chief Cardiology Fellow during this time. She has been on the faculty in the Department of Medicine at San Francisco General Hospital since 2002.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Excision BioTherapeutics
ID
NCT05144386
Phase
Phase 1 HIV/AIDS Research Study
Study Type
Interventional
Participants
Expecting 9 study participants
Last Updated