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Completed

NCT Number: NCT06341127

Fluoxetine in KCNC1-related Disorder

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.

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Key information

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M5G1R8, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Consent provided by substitute decision maker.
  • In good general health as evidenced by medical history
  • Screening baseline bloodwork (or availability of clinical bloodwork within 3 months of trial start) with values below relevant cut-offs for adequate hepatic and renal function, and baseline electrolytes including potassium within normal range.
  • Ability to take oral medication and be willing to adhere to the daily oral medication regimen

Exclusion criteria

  • Current use of monoamine oxidase inhibitors, other selective serotonin reuptake inhibitors, tricyclic antidepressants, or agents that strongly affect metabolism via CYP2D6, CYP2C9 or CYP3A4.
  • Hypersensitivity to fluoxetine or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container
  • Treatment with another investigational drug or other medication intervention within 8 weeks of starting the trial.
  • 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.
  • Long QT syndrome including acquired long QT syndrome (e.g., due to concomitant use of a drug that prolongs the QT); a family history of QT prolongation; or other clinical conditions that predispose to arrhythmias.

Treatment and study plan

Fluoxetine

Drug

Oral fluoxetine daily, 2.5 to 5 mg.

Primary outcomes

  1. Motor development

    Time frame: Weekly from date of randomization to up to 42 weeks

    Parent report on the Early Motor Questionnaire

  2. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

    Time frame: From date of randomization to up to 42 weeks

    Adverse event reporting

Secondary outcomes

  1. Adaptive skills

    Time frame: Week 1, 13, 29 and 37

    Vineland Adaptive Behavior Scale

  2. Cognitive skills

    Time frame: Week 1, 13, 29 and 37

    Mullen Scales of Early Learning

  3. Family priority outcome targets

    Time frame: Weekly from date of randomization to up to 42 weeks

    Measure Your Own Medical Profile- 2

  4. Clinical Global Impression- Improvement Scale (CGI-I)

    Time frame: Every 4 weeks from date of randomization to up to 42 weeks

    Clinician assessment of overall development

Sponsors and collaborators

Lead sponsor

Holland Bloorview Kids Rehabilitation Hospital

Other

Collaborators

  • The Hospital for Sick Children

Registry information

Official study title

A Single Patient Trial of Fluoxetine in KCNC1-related Disorder

Important dates

Study start
2024
Primary completion
2024
Study completion
2024
First posted
Apr 2, 2024
Registry last updated
Jul 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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