Long-Term Follow-Up of EBT-101 for HIV

Enrolling by invitation at 8 trial locations

Overseen By Study Lead at ExcisionBio

Age: 18+

Sex: Male

Trial Phase: Phase 1

Sponsor: Excision BioTherapeutics

Disqualifiers: Did not receive EBT-101

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to monitor the long-term effects of EBT-101, a treatment for HIV. The focus is on observing how participants who received EBT-101 in a previous study are doing over time. This follow-up is crucial for understanding the ongoing impact and safety of the treatment. Those who participated in the original study and received EBT-101 might be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications.

Is there any evidence suggesting that EBT-101 is likely to be safe for humans?

Research has shown that EBT-101 has undergone safety testing in earlier studies. In one study, participants with HIV received EBT-101, and it met its main safety goals, indicating it was generally well-tolerated. Reports from that study confirmed that most participants did not experience serious side effects. Some minor side effects occurred, but they were not severe enough to halt the trial. This early evidence suggests EBT-101 might be safe for humans, but further research is necessary to confirm this. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

EBT-101 is unique because it uses a novel gene-editing approach to target HIV at its genetic source. Unlike standard antiretroviral therapies that aim to suppress the virus, EBT-101 is designed to potentially eliminate it by cutting out sections of the HIV DNA integrated into human cells. Researchers are excited about this treatment because it offers the possibility of a one-time intervention that could lead to long-term remission or even a cure, in contrast to the lifelong daily medications currently required to manage HIV.

What evidence suggests that EBT-101 might be an effective treatment for HIV?

Research has shown that EBT-101, a gene therapy using CRISPR technology, offers promising early results for managing HIV. In one study, a patient maintained virus control for 16 weeks after stopping regular treatment, which exceeded the usual duration. However, for most participants, EBT-101 did not prevent the HIV virus from returning. The therapy has been proven safe, but its ability to completely stop the virus remains under investigation. These early findings suggest potential, but further research is needed to confirm long-term benefits. Participants in this trial will undergo long-term follow-up to evaluate the effects of EBT-101 further. ^{1 2 4 6 7}

Who Is on the Research Team?

William Kennedy, MD

Principal Investigator

Excision BioTherapeutics

Are You a Good Fit for This Trial?

This trial is for adults with HIV who have previously received the investigational treatment EBT-101 in an earlier study. They must have signed a consent form agreeing to participate and be monitored over time.

Inclusion Criteria

Participants who received the investigational study intervention EBT-101 in a parent study

Willing and able to provide written, signed informed consent

Exclusion Criteria

I have not previously received EBT-101 in any study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Long-Term Follow-Up

Participants will have follow-up visits every six months until Year 5 post EBT-101 administration

5 years

Follow-up visits every six months

Extended Follow-Up

Participants will have annual follow-up visits on the anniversary of EBT-101 administration until study completion

10 years

Annual follow-up visits

What Are the Treatments Tested in This Trial?

Interventions

• EBT-101

Trial Overview The focus of this study is on long-term follow-up of patients who were treated with EBT-101, which is a new intervention for HIV. The goal is to observe the extended effects and outcomes after initial treatment.

How Is the Trial Designed?

Group I: Long Term Follow Up Experimental Treatment 1 Intervention

Participants who received EBT-101 in a parent study will undergo long term follow up

Who Is Running the Clinical Trial?

Excision BioTherapeutics

Lead Sponsor

Trials

2

Recruited

20+

Citations

1. excision.bio excision.bio/news/press-releases/detail/43/excision-biotherapeutics-announces-data-from-the-phase-12

Press Releases EBT-101 successfully met the primary endpoint of safety and secondary endpoint of biodistribution/immunogenicity in a Phase 1/2 study ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05144386

NCT05144386 | Study of EBT-101 in Aviremic HIV-1 ... 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.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11697457/

The role of genetic diversity, epigenetic regulation, and sex ... The first-in-human clinical trial of Excision BioTherapeutics' CRISPR-based HIV cure, EBT-101, demonstrated safety but failed to prevent viral ...

4. aidsmap.com aidsmap.com/news/may-2024/crispr-gene-therapy-ebt-101-does-not-prevent-hiv-viral-rebound

CRISPR gene therapy EBT-101 does not prevent HIV viral ... One EBT-101 recipient was able to maintain viral suppression for 16 weeks after treatment discontinuation, considerably longer than it typically ...

5. eatg.org eatg.org/hiv-news/crispr-hiv-gene-therapy-disappoints-in-early-study/

CRISPR HIV gene therapy disappoints in early study One EBT-101 recipient was able to maintain viral suppression for four months after treatment discontinuation—considerably longer than it ...

6. eatg.org eatg.org/hiv-news/excision-biotherapeutics-initiates-phase-1-2-trial-evaluating-ebt-101-as-a-potential-cure-for-hiv/

Excision BioTherapeutics initiates Phase 1/2 trial ... The FIH study is a multicenter, open-label, single ascending dose study designed to evaluate the safety, tolerability and efficacy of EBT-101. The trial is ...

7. crisprmedicinenews.com crisprmedicinenews.com/news/excisions-ebt-101-demonstrates-safety-in-clinical-trial-but-does-not-cure-hiv/

Excision's EBT-101 Demonstrates Safety in Clinical Trial ... The objectives of the Phase 1/2 trial are to evaluate the safety and pharmacodynamics of EBT-101 in individuals living with HIV type 1 (HIV-1), ...