Cocaine Dependence, in RemissionPhase 1NCT04884594
AAV8-hCocH for Cocaine Use Disorder
A Phase 1 study testing AAV8-hCocH in people with Cocaine Dependence, in Remission, run by W. Michael Hooten at 1 site in United States.
Trial summary
The purpose of this study is to test the safety of a novel gene viral vector treatment for adults with cocaine use disorder-sustained remission. This gene regulates an enzyme (cocaine hydrolase) that breaks down cocaine into inactive substances, thereby decreasing the pleasurable feeling this drug usually provides. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Cocaine Dependence, in Remission
- What is being tested?
- AAV8-hCocH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Oct 2021to Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Non-treatment seeking male or females ages 18 to 65 years, inclusive.
- DSM-5 diagnosis of cocaine use disorder in sustained remission as confirmed by the PI's review of the medical record.
- Are motivated to abstain from cocaine use during the period of the study, as evidenced both by the judgment of the Investigator or designee and by compliance with the requirement to make regular clinic visits.
- In the opinion of the PI, be in good general health as determined by medical and psychiatric history, general clinical examination, vital signs, and laboratory tests.
- Have provided written informed consent. Subjects should be cooperative, willing and able to participate and adhere to the protocol requirements.
- Have hematology, chemistry, kidney and liver function laboratory tests that are within (+/- 10%) of the current Mayo Clinic standardized normal values.
- Show a baseline EKG that demonstrates normal sinus rhythm and conduction without clinically significant abnormalities or arrhythmias.
- Are willing to return to research area for follow-up.
Who cannot join
- They show detectable pre-existing immunity to the AAV8 capsid as measured by AAV8 transduction inhibition and AAV8 total antibodies.
- Evidence of HIV or hepatitis of any etiology.
- Creatinine ≥ 1.5 mg/dL.
- Any disease or mental health condition at the physician's discretion that would prevent the subject from fully complying with the requirements of the study. The physician may exclude subjects with active alcohol abuse, other substance abuse or positive urine toxicology screen for substances of abuse.
- Pregnant \&/or lactating. All lactating women will be excluded from study participation. Women of child-bearing potential must have a negative pregnancy test performed at screening visit, agree to use birth control throughout the study period, refrain from getting pregnant within the study period and consent to pregnancy testing throughout the study period. Men must agree to use barrier methods of birth control and refrain from fathering children within the next year.
- Morbid obesity (BMI > 40).
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: Intravenous Administration of AAV8-human Cocaine Hydrolase to Treat Cocaine Use Disorder
The purpose of this study is to test the safety of a novel gene viral vector treatment for adults with cocaine use disorder-sustained remission. This gene regulates an enzyme (cocaine hydrolase) that breaks down cocaine into inactive substances, thereby decreasing the pleasurable feeling this drug usually provides.
Read the detailed description
This is a phase-I dose escalation clinical trial testing the safety and MTD of IV administration of AAV8-hCocH to subjects with a history of cocaine use disorder-sustained remission. Subjects who provide written informed consent, meet entry criteria, and do not have transduction inhibition to AAV8 (pre-existing AAV8 antibodies) will be eligible. Subjects will be enrolled sequentially every 2-3 months or longer between cohorts. Dose escalation may be initiated after a single subject has been safely dosed; maximum enzyme expression is anticipated at week 3-4. This escalation paradigm is intended to minimize the number of subjects exposed to sub-therapeutic doses. The starting dose is based on the expression and safety of AAV8-CocH in mice, rats and NHP, and previous human experience using AAV8-FVIII IV in hemophilia patients. The starting dose has a large safety margin (15-fold) from the NOAEL in NHP. Approximately 7 weeks after an injection, a decision to escalate to the next dose level will be made based on a review of safety parameters and CocH levels by the investigative team.
- Conditions
- Cocaine Dependence, in Remission
- Being tested
- AAV8-hCocH
- Medical subject headings
- Cocaine-Related Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV8-hCocHDrug
2e12 vg/kg single infusion intravenous
AAV8-hCocHDrug
6e12 vg/kg single infusion intravenous
AAV8-hCocHDrug
4e12 vg/kg single infusion intravenous
| Study group | Role | What they receive |
|---|---|---|
| AAV8-hCocH dose level 1: 2e12 vg/kg | Experimental | Cohort 1: Participant receives one-time IV administration of low dose 2e12 vg/kg of AAV8-hCocH, with 7 week of follow-up after dose Receives: Drug: AAV8-hCocH |
| AAV8-hCocH dose level 3: 6e12vg/kg | Experimental | Cohort 3: Participant receives one-time IV administration of medium dose 6e12vg/kg of AAV8-hCocH, with 7 week of follow-up after dose Receives: Drug: AAV8-hCocH |
| AAV8-hCocH dose level 2: 4e12vg/kg | Experimental | Cohort 2: Participant receives one-time IV administration of medium dose 4e12vg/kg of AAV8-hCocH, with 7 week of follow-up after dose Receives: Drug: AAV8-hCocH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of participants with treatment-related adverse events
Measured: 60 months
Change in enzyme expression profile
Serum level of AAV8-hCocH gene expression
Measured: Baseline, 24 months
Secondary outcomes (4)
Maximum Observed Plasma Concentration (Cmax)
Cmax is measured as the peak concentration of AAV8 in the blood after intravenous infusion
Measured: 24 months
Time of peak concentration (tmax)
The time to maximum plasma concentration of AAV8 in the blood after intravenous infusion
Measured: 24 months
Half-Life (t1/2)
The time for plasma concentration of AAV8 in the blood to be reduced to exactly half of starting concentration
Measured: 24 months
Area under the Concentration-Time Curve (AUC)
AUC is a measure of the AAV8 serum concentration over time. Used to characterize drug absorption.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2021
- Study start8 Oct 2021
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cocaine Dependence, in RemissionStudies 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
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- W. Michael Hooten
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute on Drug Abuse (NIDA)
- Responsible party
- Sponsor-investigator, W. Michael Hooten, Mayo Clinic
- Investigators
- W. Michael Hooten, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04884594
- Sponsor's protocol ID
- 20-012225
- Other IDs
- 5UH3DA042492
- Status last verified
- May 2026
- First submitted
- 7 May 2021
- Last updated
- 26 May 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 26 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intravenous Administration of AAV8-human Cocaine Hydrolase to Treat Cocaine Use Disorder
- ClinicalTrials.gov ID
- NCT04884594
- Acronym / sponsor ID
- 20-012225
- Conditions
- Cocaine Dependence, in Remission
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- W. Michael HootenUniversity, hospital or other
- Collaborators
- National Institute on Drug Abuse (NIDA)
- Study start
- 8 Oct 2021 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 13 May 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.
The purpose of this study is to test the safety of a novel gene viral vector treatment for adults with cocaine use disorder-sustained remission. This gene regulates an enzyme (cocaine hydrolase) that breaks down cocaine into inactive substances, thereby decreasing the pleasurable feeling this drug usually provides.
Detailed description
This is a phase-I dose escalation clinical trial testing the safety and MTD of IV administration of AAV8-hCocH to subjects with a history of cocaine use disorder-sustained remission. Subjects who provide written informed consent, meet entry criteria, and do not have transduction inhibition to AAV8 (pre-existing AAV8 antibodies) will be eligible. Subjects will be enrolled sequentially every 2-3 months or longer between cohorts. Dose escalation may be initiated after a single subject has been safely dosed; maximum enzyme expression is anticipated at week 3-4. This escalation paradigm is intended to minimize the number of subjects exposed to sub-therapeutic doses. The starting dose is based on the expression and safety of AAV8-CocH in mice, rats and NHP, and previous human experience using AAV8-FVIII IV in hemophilia patients. The starting dose has a large safety margin (15-fold) from the NOAEL in NHP. Approximately 7 weeks after an injection, a decision to escalate to the next dose level will be made based on a review of safety parameters and CocH levels by the investigative team.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| AAV8-hCocH dose level 1: 2e12 vg/kg | Experimental | Drug: AAV8-hCocHCohort 1: Participant receives one-time IV administration of low dose 2e12 vg/kg of AAV8-hCocH, with 7 week of follow-up after dose |
| AAV8-hCocH dose level 3: 6e12vg/kg | Experimental | Drug: AAV8-hCocHCohort 3: Participant receives one-time IV administration of medium dose 6e12vg/kg of AAV8-hCocH, with 7 week of follow-up after dose |
| AAV8-hCocH dose level 2: 4e12vg/kg | Experimental | Drug: AAV8-hCocHCohort 2: Participant receives one-time IV administration of medium dose 4e12vg/kg of AAV8-hCocH, with 7 week of follow-up after dose |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Non-treatment seeking male or females ages 18 to 65 years, inclusive.
- DSM-5 diagnosis of cocaine use disorder in sustained remission as confirmed by the PI's review of the medical record.
- Are motivated to abstain from cocaine use during the period of the study, as evidenced both by the judgment of the Investigator or designee and by compliance with the requirement to make regular clinic visits.
- In the opinion of the PI, be in good general health as determined by medical and psychiatric history, general clinical examination, vital signs, and laboratory tests.
- Have provided written informed consent. Subjects should be cooperative, willing and able to participate and adhere to the protocol requirements.
Key exclusion criteria (6 listed)
- They show detectable pre-existing immunity to the AAV8 capsid as measured by AAV8 transduction inhibition and AAV8 total antibodies.
- Evidence of HIV or hepatitis of any etiology.
- Creatinine ≥ 1.5 mg/dL.
- Any disease or mental health condition at the physician's discretion that would prevent the subject from fully complying with the requirements of the study. The physician may exclude subjects with active alcohol abuse, other substance abuse or positive urine toxicology screen for substances of abuse.
- Pregnant \&/or lactating. All lactating women will be excluded from study participation. Women of child-bearing potential must have a negative pregnancy test performed at screening visit, agree to use birth control throughout the study period, refrain from getting pregnant within the study period and consent to pregnancy testing throughout the study period. Men must agree to use barrier methods of birth control and refrain from fathering children within the next year.
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 |
|---|---|---|
| AAV8-hCocH2e12 vg/kg single infusion intravenous | Drug | AAV8-hCocH dose level 1: 2e12 vg/kg |
| AAV8-hCocH6e12 vg/kg single infusion intravenous | Drug | AAV8-hCocH dose level 3: 6e12vg/kg |
| AAV8-hCocH4e12 vg/kg single infusion intravenous | Drug | AAV8-hCocH dose level 2: 4e12vg/kg |
Primary outcome
Adverse Events
Time frame: 60 months
Number of participants with treatment-related adverse events
Change in enzyme expression profile
Time frame: Baseline, 24 months
Serum level of AAV8-hCocH gene expression
4 secondary outcomes
Maximum Observed Plasma Concentration (Cmax)
Time frame: 24 months
Time of peak concentration (tmax)
Time frame: 24 months
Half-Life (t1/2)
Time frame: 24 months
Area under the Concentration-Time Curve (AUC)
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2026.
- Individual participant dataSharing planned: No
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.
W. Michael Hooten, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AAV8-hCocH in people with Cocaine Dependence, in Remission, run by W. Michael Hooten at 1 site 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 8 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 26 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
This study is still under way. Results are not available in the source record yet.
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?
Adverse Events (time frame: 60 months). The study lists 2 primary outcomes in all. 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 26 May 2026.