Characteristics of Ongoing Clinical Trials for Cocaine Use Disorder Registered on Global Clinical Trial Databases

Cocaine use disorder (CUD) affects 1.4 million people in the United States, yet no FDA-approved treatments exist. In 2023, the Food and Drug Administration (FDA) released a draft guideline on treatments for stimulant use disorders, providing direction for trial design, outcomes, and population selection. In this study, we aimed to review ongoing clinical trials for CUD and assess their alignment with the FDA's recommendations.

We conducted a systematic search of the 6 major clinical trial databases (United States, Australia, Canada, Iran, Netherlands, and Switzerland) to identify ongoing interventional studies for CUD. We included trials evaluating pharmacological, behavioral, device-based, and mixed treatments. We extracted data on intervention type, target population, study design, duration, and primary outcomes. Trials were assessed for alignment with 5 key FDA recommendations, including trial duration, use of both self-reported and biological outcome measures, randomization, placebo control, and double blinding.

In total, 38 trials were identified, primarily from the United States (32). Most trials were randomized: 36 (94.7%), while 21 (55.3%) trials had combined endpoints or a 3-month minimum duration. Only 7 trials (18.4%) met all 5 key FDA recommendations. New treatment approaches were identified, including psilocybin and the dAd5GNE vaccine, as well as digital platforms for behavioral therapies.

A variety of promising treatments for CUD are under investigation. However, many trials fall short of current FDA design recommendations. Improved adherence to regulatory guidance and stronger collaboration between researchers and regulators will be essential to advance effective, scalable treatments for CUD.