Trial · NCT07503444
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
NCT07503444, Phase 3, Recruiting, UCB BIOSCIENCES, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome. Trials, record NCT07503444. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07503444
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
- NCT number
- NCT07503444
- Status
- Recruiting
- Phases
- Phase 3
- Enrollment
- 200 participants, estimated
- Lead sponsor
- UCB BIOSCIENCES, Inc.
- Conditions
- Rett Syndrome
- Interventions
- fenfluramine hydrochloride, Placebo
- First posted
- 2026-03-31
- Last updated
- 2026-11-02
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change from Baseline to Week 14 in Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score | From Baseline (Day 1) to Week 14 | The RSBQ is a caregiver-completed, instrument assessing behavioral and emotional features in RTT. The RSBQ consists of 45 items, including 8 subscales: General mood (8 items); Breathing problems (5 items); Hand behavior (6 items); Face movements (4 items); Body rocking and expressionless face (6 items); Nighttime behaviors (3 items); Fear/anxiety (4 items); and Walking/standing (2 items). Caregivers are asked to evaluate each RTT feature based on the current status of the patients on a 3-point scale as 0 ("not true"), 1 ("somewhat or sometimes true"), or 2 ("often true"), with the total score ranging from 0 to 90. Higher scores indicate increased disease severity. Seven items that do not belong under any of the subscales are classed as "uncategorized" but contribute to the overall total score. |
| Primary | Clinical Global Impression of Change (CGIC) Score at Week 14 | At Week 14 | The CGIC is a clinician-rated single item evaluating the degree of improvement or worsening of a participant's condition from Baseline following treatment or intervention. The CGIC uses a 7-point response scale, with the following ratings: "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 from Baseline to Week 14 in Patient-Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD) score | From Baseline (Day 1) to Week 14 | Sleep disturbances will be assessed by the PROMIS-SD parent proxy 8a version. Caregivers are asked to rate 8 items over the past 7 days on a 5-point scale: "1: Never", "2: Almost never", "3: Sometimes", "4: Almost always", and "5: Always", with higher scores indicating more severe sleep disturbances. |
| Secondary | Change from Baseline to Week 14 in Observer-Reported Communication Ability (ORCA) score | From Baseline (Day 1) to Week 14 | The ORCA is an 84-item, observer-reported measure of communication ability over the past 30 days. The majority of the items included in the ORCA measure have 3 response options: "No or only once," "Sometimes," and "Yes, almost all the time", which enable derivation of an overall communication score and scores for each form of communication (expressive, receptive, and pragmatic), with higher scores indicating greater communication ability. |
| Secondary | Caregiver Global Impression of Change - Seizure (CaGIC-Seizure) score at Week 14 | At Week 14 | The CaGIC-Seizure is a caregiver-reported item assessing the change from Baseline in the participant's seizure status. The CaGIC-Seizure uses a 7-point response scale, with the following ratings: "1: Very Much improved", "2: Much improved", "3: Minimally improved", "4: No change", "5: Minimally worse", "6: Much worse", "7: Very Much Worse". |
| Secondary | Incidence of treatment-emergent adverse events (TEAEs) during the double-blind intervention period | From Baseline (Day 1) up to Week 14 | An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. |
| Secondary | Incidence of serious TEAEs during the double-blind intervention period | From Baseline (Day 1) up to Week 14 | An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed: * Results in death * Is life-threatening * Requires inpatient hospitalization or prolongation of existing hospitalization * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect * Other important medical events which based on medical or scientific judgement may jeopardize the patients or may require medical or surgical intervention to prevent any of the above. |
| Secondary | Incidence of TEAEs leading to discontinuation during the double-blind intervention period | From Baseline (Day 1) up to Week 14 | An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. TEAEs leading to discontinuation will be reported. |
| Secondary | Incidence of related TEAEs during the double-blind intervention period | From Baseline (Day 1) up to Week 14 | An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. Related TEAEs will be reported. |
| Secondary | Change from Baseline in QT interval corrected using Fridericia's formula (QTcF) interval on 12-lead ECG by visit during the double-blind intervention period | From Baseline (Day 1) up to Week 14 | Change from Baseline in QTcF interval as measured by 12-lead ECG up to Week 14 by visit will be reported |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes0
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.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT07503444
GET https://conductscience.org/api/trials/v1/NCT07503444/registered