Trial · NCT03861871
Fenfluramine in CDKL5 Deficiency Disorder (CDD)
NCT03861871, Phase 2, Completed, NYU Langone Health; results first posted 2023-10-04
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Fenfluramine in CDKL5 Deficiency Disorder (CDD). Trials, record NCT03861871. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03861871
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Fenfluramine in CDKL5 Deficiency Disorder (CDD)
- NCT number
- NCT03861871
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 7 participants, actual
- Lead sponsor
- NYU Langone Health
- Conditions
- CDD
- Interventions
- Fenfluramine Hydrochloride
- First posted
- 2019-03-04
- Last updated
- 2023-10-04
- Results first posted
- 2023-10-04
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Median Monthly Convulsive Seizure Frequency | Baseline, Week 14 | Change between baseline and Week 14 in the median number of monthly convulsive seizures. |
| Secondary | Change in Caregiver Global Impression of Change (CGIC) Score | Baseline, Week 14 | The CGIC is a 1-item, parent/caregiver-completed assessment used determine how much their child/care-recipient has improved with treatment. The instrument asks parents/caregivers to rate their child's/care-recipient's improvement as: 1) very much improved; 2) much improved; 3) minimally improved; 4) unchanged; 5) a little worse; 6) much worse; 7) very much worse; the total score correspondingly ranges from 1-7. |
| Secondary | Change in Investigator Global Impression of Change (IGIC) Score | Baseline, Week 14 | The IGIC is a 1-item, investigator-completed assessment used determine how much a patient has improved with treatment. The instrument asks the investigator to rate patients' improvement as: 1) very much improved; 2) much improved; 3) minimally improved; 4) unchanged; 5) a little worse; 6) much worse; 7) very much worse; the total score correspondingly ranges from 1-7. |
| Secondary | Change in Quality of Life in Childhood Epilepsy (QOLCE) Score | Baseline, Week 14 | Parent/caregiver-completed assessment assessing how epilepsy affects day-to-day functioning of their child/care-recipient in various life areas. Each item is ranked on a 5-point Likert scale from 1 (response correlated with the lowest possible quality of life) to 5 (response correlated with the highest possible quality of life). Item scores are then transformed to a 0-100 scale as follows: 1 = 0, 2 = 25, 3 = 50, 4=75, and 5=100. The total score is the average of all item scores and ranges from 0-100. Higher scores indicate greater quality of life; an increase in scores indicates quality of life increased during the observational period. |
| Secondary | Change in Pediatric Quality Of Life (PEDS-QL) Epilepsy Module Raw Score | Baseline, Week 14 | The PedsQL Epilepsy Module is a 29-item measure with five scales: Impact, Cognitive, Sleep, Executive Function, and Mood/Behavior. The Impact scale (nine items) assesses how epilepsy interferes with daily activities, interacting with peers, independence, and increased disease burden due to treatment. The Cognitive Scale (six items) assesses memory, ability to learn new materials, school-related difficulties, and reading difficulties. The Sleep Scale (three items) assesses fatigue and sleep difficulties. The Executive Function Scale (six items) assesses organization, task initiation, impulsivity, and inattention. The Mood/Behavior Scale (five items) assesses feelings of anger, sadness, worries, and frustration tolerance. Scores range from 0-100 for each subscale, with higher scores representing better quality of life. The raw score is the sum of each subscale score and ranges from 0-500. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Median Monthly Convulsive Seizure Frequency | Baseline, Week 14 | Number of monthly seizures | MEDIAN | |
| SECONDARY | Change in Caregiver Global Impression of Change (CGIC) Score | Baseline, Week 14 | score on a scale | MEAN | |
| SECONDARY | Change in Investigator Global Impression of Change (IGIC) Score | Baseline, Week 14 | score on a scale | MEAN | |
| SECONDARY | Change in Quality of Life in Childhood Epilepsy (QOLCE) Score | Baseline, Week 14 | score on a scale | MEAN | |
| SECONDARY | Change in Pediatric Quality Of Life (PEDS-QL) Epilepsy Module Raw Score | Baseline, Week 14 | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group5
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Median Monthly Convulsive Seizure Frequency | Fenfluramine Hydrochloride | 88.429 | Standard Deviation 122.44 | Number of monthly seizures | |
| Change in Caregiver Global Impression of Change (CGIC) Score | Fenfluramine Hydrochloride | -2.429 | Standard Deviation 1.988 | score on a scale | |
| Change in Investigator Global Impression of Change (IGIC) Score | Fenfluramine Hydrochloride | 1.571 | Standard Deviation 0.787 | score on a scale | |
| Change in Quality of Life in Childhood Epilepsy (QOLCE) Score | Fenfluramine Hydrochloride | -0.429 | Standard Deviation 1.512 | score on a scale | |
| Change in Pediatric Quality Of Life (PEDS-QL) Epilepsy Module Raw Score | Fenfluramine Hydrochloride | -103.571 | Standard Deviation 424.124 | score on a scale |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Fenfluramine Hydrochloride | 7 | |
| Overall Study | COMPLETED | Fenfluramine Hydrochloride | 7 | |
| Overall Study | NOT COMPLETED | Fenfluramine Hydrochloride | 0 |
Source ClinicalTrials.gov
Adverse events30
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhea | Gastrointestinal disorders | Fenfluramine Hydrochloride | 2 | 7 | |
| other | Lethargy | General disorders | Fenfluramine Hydrochloride | 6 | 7 | |
| other | Crying episode | General disorders | Fenfluramine Hydrochloride | 2 | 7 | |
| other | Screaming episode | General disorders | Fenfluramine Hydrochloride | 1 | 7 | |
| other | Low appetite | General disorders | Fenfluramine Hydrochloride | 6 | 7 | |
| other | Upper respiratory infection | Infections and infestations | Fenfluramine Hydrochloride | 3 | 7 | |
| other | Common cold | Infections and infestations | Fenfluramine Hydrochloride | 3 | 7 | |
| other | Teething | General disorders | Fenfluramine Hydrochloride | 1 | 7 | |
| other | Urinary tract infection | Infections and infestations | Fenfluramine Hydrochloride | 1 | 7 | |
| other | Vomitting | Gastrointestinal disorders | Fenfluramine Hydrochloride | 1 | 7 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Fenfluramine Hydrochloride | 0 | 7 |
| Total, other adverse events | Fenfluramine Hydrochloride | 7 | 7 |
| Total, serious adverse events | Fenfluramine Hydrochloride | 1 | 7 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 33979451 | 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/NCT03861871
GET https://conductscience.org/api/trials/v1/NCT03861871/events?page=2