HIV-1-infectionPhase 1NCT05144386
Study of EBT-101 in Aviremic HIV-1 Infected Adults on Stable ART
A Phase 1 study testing EBT-101 in people with HIV-1-infection, run by Excision BioTherapeutics at 3 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 70 years, men only
- Conditions the study is for
- HIV-1-infection
- What is being tested?
- EBT-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 3 sitesUnited States
- Study dates
- 24 Jan 2022to 14 Nov 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cooper Health
Camden, New Jersey, 08103
Quest Clinical Research
San Francisco, California, 94115
Washington University
St Louis, Missouri, 63110
About this study
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
This is a First in Human (FIH) study of EBT-101 administered IV to aviremic HIV-1 infected adults on stable antiretroviral therapy (ART).
Read the detailed description
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 after Week 12 for ATI participants. Non-ATI participants are followed monthly 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 is 15 years.
- Conditions
- HIV-1-infection
- Being tested
- EBT-101
- Keywords
- CRISPR, Gene Therapy, AAV9, HIV, SaCas9
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EBT-101Biological
EBT-101 is a HIV-1-specific clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 gene editing system delivered by adenovirus-associated virus vector serotype 9 (AAV9) for intravenous (IV) administration
| Study group | Role | What they receive |
|---|---|---|
| EBT-101 Dose-Level 1 | Experimental | Cohort A: Participants will be administered dose-level 1 of EBT-101 Receives: Biological: EBT-101 |
| EBT-101 Dose-Level 2 | Experimental | Cohort B: Participants will be administered dose-level 2 of EBT-101 Receives: Biological: EBT-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability of EBT-101
Safety and tolerability of EBT-101 will be assessed based on the incidence and severity of adverse events (AEs) according to Division of AIDS (DAIDS) 2017 over 48 weeks
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2021
- Study start24 Jan 2022
- Primary completion14 Nov 2024
- Study completion14 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HIV-1-infectionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Excision BioTherapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- William Kennedy, MD, Study director, Excision BioTherapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05144386
- Sponsor's protocol ID
- EBT-101-001
- Status last verified
- Jul 2024
- First submitted
- 22 Nov 2021
- Last updated
- 2 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05144386
- Acronym / sponsor ID
- EBT-101-001
- Conditions
- HIV-1-infection
- Keywords
- CRISPR, Gene Therapy, AAV9, HIV, SaCas9
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Excision BioTherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 24 Jan 2022 (actual)
- Primary completion
- 14 Nov 2024 (actual)
- Study completion
- 14 Nov 2024 (actual)
- First posted
- 3 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a First in Human (FIH) study of EBT-101 administered IV to aviremic HIV-1 infected adults on stable antiretroviral therapy (ART).
Detailed description
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 after Week 12 for ATI participants. Non-ATI participants are followed monthly 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 is 15 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EBT-101 Dose-Level 1 | Experimental | Biological: EBT-101Cohort A: Participants will be administered dose-level 1 of EBT-101 |
| EBT-101 Dose-Level 2 | Experimental | Biological: EBT-101Cohort B: Participants will be administered dose-level 2 of EBT-101 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EBT-101EBT-101 is a HIV-1-specific clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 gene editing system delivered by adenovirus-associated virus vector serotype 9 (AAV9) for intravenous (IV) administration | Biological | EBT-101 Dose-Level 1, EBT-101 Dose-Level 2 |
Primary outcome
Safety and Tolerability of EBT-101
Time frame: 48 weeks
Safety and tolerability of EBT-101 will be assessed based on the incidence and severity of adverse events (AEs) according to Division of AIDS (DAIDS) 2017 over 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
William Kennedy, MD study director
Excision BioTherapeutics
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EBT-101 in people with HIV-1-infection, run by Excision BioTherapeutics at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability of EBT-101 (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Dec 2024.