AAV8-hCocH for Cocaine Use Disorder

NCT04884594 | Active Not Recruiting Phase 1 FDA Drug

• 2 other identifiers: 20-0122255UH3DA042492
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Cocaine Dependence, in Remission

Outcome Measures

Primary Outcomes (2)

• Adverse Events

  Number of participants with treatment-related adverse events

  60 months

• Change in enzyme expression profile

  Serum level of AAV8-hCocH gene expression

  Baseline, 24 months

Secondary Outcomes (4)

• Maximum Observed Plasma Concentration (Cmax)

  24 months

• Time of peak concentration (tmax)

  24 months

• Half-Life (t1/2)

  24 months

• Area under the Concentration-Time Curve (AUC)

  24 months

Study Arms (3)

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

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

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

Drug: AAV8-hCocH

Interventions

AAV8-hCocH DRUG

2e12 vg/kg single infusion intravenous

AAV8-hCocH dose level 1: 2e12 vg/kg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• W. Michael Hooten: lead
• National Institute on Drug Abuse (NIDA): collaborator

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Cocaine-Related Disorders

Condition Hierarchy (Ancestors)

Substance-Related Disorders; Chemically-Induced Disorders; Mental Disorders

Study Officials

• W. Michael Hooten, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: May 7, 2021
• First Posted: May 13, 2021
• Study Start: October 8, 2021
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: May 26, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)