Gene therapy for cocaine use disorder

Intravenous Administration of AAV8-human Cocaine Hydrolase to Treat Cocaine Use Disorder

This study is testing a new gene therapy to see if it can help adults with a history of cocaine use disorder by safely breaking down cocaine in their bodies.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Mayo Clinic (other)
• Locations: 1 site (Rochester, Minnesota)
• Trial ID: NCT04884594 on ClinicalTrials.gov

What this trial studies

This phase-I clinical trial aims to evaluate the safety of a novel gene therapy using AAV8-hCocH, which regulates an enzyme that breaks down cocaine into inactive substances. The trial will involve adults aged 18 to 65 with a history of cocaine use disorder who are in sustained remission. Participants will receive intravenous administration of the treatment, with dose escalation based on safety and enzyme expression levels monitored over several weeks. The study seeks to minimize exposure to sub-therapeutic doses while ensuring participant safety throughout the process.

Who should consider this trial

Good fit: Ideal candidates are non-treatment seeking adults aged 18 to 65 with a DSM-5 diagnosis of cocaine use disorder in sustained remission.

Not a fit: Patients actively seeking treatment for cocaine use or those with significant health issues may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for individuals with cocaine use disorder, potentially reducing cravings and relapse rates.

How similar studies have performed: While gene therapy approaches are emerging, this specific application of AAV8-hCocH for cocaine use disorder is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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).

Where this trial is running

Rochester, Minnesota

• Mayo Clinic in Rochester — Rochester, Minnesota, United States (RECRUITING)

Study contacts

• Principal investigator: W. Michael Hooten, MD — Mayo Clinic
• Study coordinator: Beth Martin, RRT

Conditions: Cocaine Dependence, in Remission