Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT05144386)

Disease info:

Human immunodeficiency virus (HIV) is a virus that attacks the immune system by infecting and killing white blood cells known as CD4+ T-cells. CD4+ T-cells represent a vital part of the immune system and untreated HIV infections render patients more vulnerable to dangerous infections by other pathogens, including bacteria and fungi. If the virus remains untreated it can lead to patients developing the life-threatening disease acquired immunodeficiency syndrome (AIDS). Untreated HIV can leave people vulnerable to life-threatening infections. HIV symptoms often manifest as flu-like symptoms such as fever, chills, rash, night sweats, muscle aches, sore throat, and fatigue. Today, antiviral medications can allow people living with HIV to live healthy lives.

Frequency:
In 2021, 36,136 people received an HIV diagnosis in the United States and dependent areas. An estimated 1.2 million people in the United States had HIV at the end of 2021.
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
Who:

Contact

Name: William Kennedy, MD

Phone: 833-214-2241 

Email: clinicalscience@excisionbio.com

 

Sponsor:

Excision BioTherapeutics 

Partners:
Locations:

United States, California

University of California San Francisco, San Francisco, California, United States, 94110

 

United States, Missouri

Washington University, Saint Louis, Missouri, United States, 63110

 

United States, New Jersey

Cooper Health, Camden, New Jersey, United States, 08103

Study start:
Jan. 24, 2022
Enrollment:
9 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene disruption
Gene:
Three undisclosed genomic sites in the HIV DNA
Delivery method:
AAV9 - In-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/NCT05144386
Safety updates:

(20-07-2023) Excision BioTherapeutics Receives FDA Fast Track Designation for EBT-101, a First-in-Class CRISPR-Based Gene Therapy Candidate to Functionally Cure HIV-1

(22-11-2022) First patient dosed with HIV gene therapy

(27-01-2022) Excision BioTherapeutics Initiates Phase 1/2 Trial Evaluating EBT-101 as a Potential Cure for HIV

(15-09-2021) Excision Receives FDA Clearance of IND for Phase 1/2 Trial of EBT-101 CRISPR-Based Therapeutic for Treatment of HIV

IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

This is a First in Human (FIH), open-label, sequential cohort, single ascending dose (SAD) study of EBT-101 administered IV to aviremic HIV-1 infected adults on stable antiretroviral therapy (ART).

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; participants who undergo ATI will have more frequent study visits than non-ATI participants.

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.

Last updated: Dec. 28, 2023
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