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| Name | Class |
|---|---|
| The Hospital for Sick Children | OTHER |
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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo-Fluoxetine-Placebo | Experimental | 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fluoxetine | Drug | Oral fluoxetine daily, 2.5 to 5 mg. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Motor development | Parent report on the Early Motor Questionnaire | Weekly from date of randomization to up to 42 weeks |
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | Adverse event reporting | From date of randomization to up to 42 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Adaptive skills | Vineland Adaptive Behavior Scale | Week 1, 13, 29 and 37 |
| Cognitive skills | Mullen Scales of Early Learning | Week 1, 13, 29 and 37 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Danielle Baribeau, MD, PhD | Holland Bloorview Kids Rehabilitation Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Holland Bloorview Kids Rehabilitation Hospital | Toronto | Ontario | M5G1R8 | Canada |
Single patient trial; would be challenging to de-identify.
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| Type | Date | Date Unknown |
|---|---|---|
| Release | Jun 5, 2026 | |
| Reset | Jun 30, 2026 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Jun 5, 2026 | Jun 30, 2026 |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D005473 | Fluoxetine |
| ID | Term |
|---|---|
| D011437 | Propylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
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This quasi experimental design involves masking of transition moments, but the investigator and participant are aware of the order of cross-over.
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| Family priority outcome targets | Measure Your Own Medical Profile- 2 | Weekly from date of randomization to up to 42 weeks |
| Clinical Global Impression- Improvement Scale (CGI-I) | Clinician assessment of overall development | Every 4 weeks from date of randomization to up to 42 weeks |