Fluoxetine for Genetic Disorder

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.

Research Team

Danielle Baribeau, MD, PhD

Principal Investigator

Holland Bloorview Kids Rehabilitation Hospital

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.

Inclusion Criteria

Consent provided by substitute decision maker

In good general health as evidenced by medical history

My recent blood tests show my liver, kidneys, and potassium levels are normal.

Exclusion Criteria

Hypersensitivity to fluoxetine or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container

Any condition or diagnosis, that could in the opinion of the Principal Investigator or delegate interfere with the participant's ability to comply with study instructions, might confound the interpretation of the study results, or put the participant at risk

I am currently taking certain antidepressants or drugs that affect my body's drug processing enzymes.

Treatment Details

Interventions

• Fluoxetine (Selective Serotonin Reuptake Inhibitor)

Trial Overview 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.

Participant Groups

Group I: Placebo-Fluoxetine-Placebo Experimental Treatment 1 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

Who Is Running the Clinical Trial?

Holland Bloorview Kids Rehabilitation Hospital

Lead Sponsor

Trials

69

Recruited

14,100+

The Hospital for Sick Children

Collaborator

Trials

724

Recruited

6,969,000+