Trial · NCT04442295
An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome
NCT04442295, Phase 1, Phase 2, Completed, Stoke Therapeutics, Inc
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome. Trials, record NCT04442295. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04442295
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome
- NCT number
- NCT04442295
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 62 participants, actual
- Lead sponsor
- Stoke Therapeutics, Inc
- Conditions
- Dravet Syndrome
- Interventions
- STK-001 - Single Ascending Doses, STK-001 - Multiple Ascending Doses
- First posted
- 2020-06-22
- Last updated
- 2025-05-18
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety and Tolerability of single and multiple doses of STK-001 with respect to: | Screening (Day -28) until 6 months after single and multiple drug dosing | 1. Incidence of adverse events 2. incidence of abnormal vital signs 3. Abnormal physical examination findings 4. Abnormal 12-lead electrocardiogram (ECG) 5. Abnormal laboratory parameters |
| Primary | Pharmacokinetic (PK) Parameters | Day 1 (Dosing) until 6 months after single and multiple drug dosing | Analysis of plasma concentrations of STK-001 |
| Primary | Exposure of STK-001 in Cerebrospinal Fluid (CSF) | Day 1 (Dosing) until 6 months after single and multiple drug dosing | Measurement of STK-001 concentrations |
| Secondary | Measurement of seizure frequency | Screening (Day -28) until 6 months after single and multiple drug dosing | Measured by paper diary |
| Secondary | Change in Caregiver Global Impression of Change Scale | Baseline (Day -1) until 6 months after single and multiple drug dosing | Change from baseline in overall clinical status as measured by the Clinical Global Impression of Change (CGIC). Values of scales: 1. Very much improved 2. Much improved 3. Minimally improved 4. No change 5. Minimally worse 6. Much worse 7. Very much worse |
| Secondary | Change in Clinician-assessed Global Impression of Change Scale | Baseline (Day -1) until 6 months after single and multiple drug dosing | Change from baseline in overall clinical status as measured by the Caregiver Global Impression of Change (CaGIC) Values of scales: 1. Very much improved 2. Much improved 3. Minimally improved 4. No change 5. Minimally worse 6. Much worse 7. Very much worse |
| Secondary | Measurement of Quality of Life | Baseline (Day -1) until 6 months after single and multiple drug dosing | Change in quality of life as measured by the EuroQoL-five dimensions, youth version (EQ-5D-Y) instrument. The scale is scored from 0-100. The reference to a high score indicates a better outcome of quality of life. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41780062 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04442295
GET https://conductscience.org/api/trials/v1/NCT04442295/registered