Trial · NCT07503444
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 19 of 19
Download this page as JSON| 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 |
| Secondary | Incidence of treatment-emergent Doppler Echocardiogram (ECHO) results meeting the Food and Drug Administration (FDA) case definition of drug-associated valvular heart disease (VHD) during the double-blind intervention period | From Baseline (Day 1) up to Week 14 | The FDA case definition of drug-associated VHD is aortic regurgitation ≥mild and/or mitral regurgitation ≥moderate with restricted valve motion, valve thickening, and/or physical signs or symptoms attributable to valve diseases. ECHO readings related to VHD on any of the 4 valves (aortic, mitral, pulmonary, tricuspid) will be reported with grades of absent, trace, mild, moderate, or severe. |
| Secondary | Incidence of treatment-emergent Doppler ECHO results meeting the FDA case definition of PAH (defined as a PASP >35mmHg) during the double-blind intervention period | From Baseline (Day 1) up to Week 14 | ECHO readings related to pulmonary arterial hypertenstion (PAH) will be reported based on Pulmonary artery systolic pressure (PASP). |
| Secondary | Incidence of treatment-emergent adverse events (TEAEs) from baseline to end of safety follow up | From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months) | 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 from baseline to end of safety follow up | From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months) | 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 from baseline to end of safety follow up | From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months) | 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 from baseline to end of safety follow up | From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months) | 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 from baseline to end of safety follow up | From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months) | Change from Baseline in QTcF interval as measured by 12-lead ECG up to Week 98 by visit will be reported |
| Secondary | Incidence of treatment-emergent Doppler Echocardiogram (ECHO) results meeting the Food and Drug Administration (FDA) case definition of drug-associated valvular heart disease (VHD) from baseline to end of safety follow up | From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months) | The FDA case definition of drug-associated VHD is aortic regurgitation ≥mild and/or mitral regurgitation ≥moderate with restricted valve motion, valve thickening, and/or physical signs or symptoms attributable to valve diseases. ECHO readings related to VHD on any of the 4 valves (aortic, mitral, pulmonary, tricuspid) will be reported with grades of absent, trace, mild, moderate, or severe. |
| Secondary | Incidence of treatment-emergent Doppler ECHO results meeting the FDA case definition of PAH (defined as a PASP >35mmHg) from baseline to end of safety follow up | From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months) | ECHO readings related to pulmonary arterial hypertenstion (PAH) will be reported based on Pulmonary artery systolic pressure (PASP). |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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.
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