Fluoxetine in KCNC1-related Disorder

Genetic Disease Clinical Trial

Official title:

A Single Patient Trial of Fluoxetine in KCNC1-related Disorder

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06341127
• Other study ID #: 2023-01
• Status: Enrolling by invitation
• Phase: N/A
• Start date: January 17, 2024
• Est. completion date: December 31, 2025

Study information

• Verified date: February 2024
• Source: Holland Bloorview Kids Rehabilitation Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 1
• Est. completion date: December 31, 2025
• Est. primary completion date: December 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

1. Consent provided by substitute decision maker.

2. In good general health as evidenced by medical history

3. 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.

4. Ability to take oral medication and be willing to adhere to the daily oral medication regimen

Exclusion Criteria:

1. Current use of monoamine oxidase inhibitors, other selective serotonin reuptake inhibitors, tricyclic antidepressants, or agents that strongly affect metabolism via CYP2D6, CYP2C9 or CYP3A4.

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

3. Treatment with another investigational drug or other medication intervention within 8 weeks of starting the trial.

4. 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.

5. 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.

Related Conditions & MeSH terms

• Genetic Disease
• Genetic Diseases, Inborn
• Rare Diseases

Intervention

Drug:
• Fluoxetine
Oral fluoxetine daily, 2.5 to 5 mg.

Locations

Country	Name	City	State
Canada	Holland Bloorview Kids Rehabilitation Hospital	Toronto	Ontario

Sponsors (2)

Lead Sponsor	Collaborator
Holland Bloorview Kids Rehabilitation Hospital	The Hospital for Sick Children

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Motor development	Parent report on the Early Motor Questionnaire	Weekly from date of randomization to up to 42 weeks
Primary	Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	Adverse event reporting	From date of randomization to up to 42 weeks
Secondary	Adaptive skills	Vineland Adaptive Behavior Scale	Week 1, 13, 29 and 37
Secondary	Cognitive skills	Mullen Scales of Early Learning	Week 1, 13, 29 and 37
Secondary	Family priority outcome targets	Measure Your Own Medical Profile- 2	Weekly from date of randomization to up to 42 weeks
Secondary	Clinical Global Impression- Improvement Scale (CGI-I)	Clinician assessment of overall development	Every 4 weeks from date of randomization to up to 42 weeks