Fluoxetine for Genetic Disorder

Recruiting in Palo Alto (17 mi)

Overseen ByDanielle Baribeau, MD, PhD

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Academic

Waitlist Available

Sponsor: Holland Bloorview Kids Rehabilitation Hospital

No Placebo Group

Approved in 2 jurisdictions

Trial Summary

What is the purpose of this trial?This is a single patient study of oral powdered fluoxetine to target developmental outcomes in a child with KCNC1-related disorder. This trial will be conducted at Holland Bloorview Kids Rehabilitation Hospital over 32 to 42 weeks, using a quasi experimental ABA phase design (placebo-fluoxetine-placebo) with randomized and blinded active treatment start and stop moments.

Eligibility Criteria

This trial is for a child with KCNC1-related disorder, which is a rare genetic condition that can cause epileptic encephalopathy. The study will take place at Holland Bloorview Kids Rehabilitation Hospital and aims to improve developmental outcomes.

Participant Groups

The trial tests oral powdered fluoxetine over approximately 8 to 10 months using an ABA phase design where the patient receives placebo, then fluoxetine, and back to placebo. The exact times when active treatment starts and stops are chosen randomly but kept secret.

1Treatment groups

Experimental Treatment

Group I: Placebo-Fluoxetine-PlaceboExperimental Treatment1 Intervention

placebo (medical grade cornstarch in a gel capsule, 1 capsule daily), followed by blinded randomized cross-over moment to active fluoxetine (2.5 then 5 mg po daily), followed by blinded randomized cross-over moment back to placebo

Fluoxetine is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Prozac for: