Trial · NCT02790034
Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
NCT02790034, Phase 2, Phase 3, Terminated, Newron Pharmaceuticals SPA; results first posted 2021-12-21
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms. Trials, record NCT02790034. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02790034
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
- NCT number
- NCT02790034
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 129 participants, actual
- Lead sponsor
- Newron Pharmaceuticals SPA
- Conditions
- Rett Syndrome
- Interventions
- Sarizotan low dose, Sarizotan high dose, Placebo
- First posted
- 2016-06-03
- Last updated
- 2021-12-21
- Results first posted
- 2021-12-21
- Limitations and caveats
- No efficacy evaluation was performed for the open label extension, as the study was terminated prematurely by the Sponsor based on lack of demonstrated efficacy in the double-blind period.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Reduction in Respiratory Abnormality in Patients With Rett Syndrome | Baseline up to week 24 | Measured as the percent change in the number of apnea episodes per hour during awake time, calculated using an ambulatory data acquisition system (BioRadioTM) as part of home monitoring procedure. BioRadioTM record specific respiratory and cardiac parameters. |
| Secondary | Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change | 24 weeks | Caregiver-rated Impression of Change (CIC): 7-point scale requiring the caregiver to rate how much the patient's illness has improved or worsened relative to the baseline state. 7-point Likert-type scale for which ratings range from 1 = very much improved to 7 = very much worse, with 4 = no change. This caregiver-rated measure considered activities, behavior, mood and functioning. This rating was performed in consultation with the study Investigator but was based largely on the caregivers' evaluation during the reporting period. The single rating of the CIC was to be based on changes in the following domains: • Activities (watching TV, interest in conversations around her, cooperation during toileting, dressing/bathing, etc.), • Communication (verbal or by eye movements, hand movements, or head movements), • Behavior (agitation, refusal to feed, scratching, social avoidance), • Participation in family/outdoor/social events) |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Reduction in Respiratory Abnormality in Patients With Rett Syndrome | Baseline up to week 24 | The ITT population included all patients who were randomized to treatment. This population was used for all efficacy analysis. | % of change in mean counts per hr | LEAST_SQUARES_MEAN |
| SECONDARY | Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change | 24 weeks | The ITT population included all patients who were randomized to treatment. This population was used for all efficacy analysis. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Reduction in Respiratory Abnormality in Patients With Rett Syndrome | Sarizotan Low Dose | 1.54 | Standard Error 10.1228 | % of change in mean counts per hr | |
| Reduction in Respiratory Abnormality in Patients With Rett Syndrome | Sarizotan High Dose | 13.211 | Standard Error 7.5075 | % of change in mean counts per hr | |
| Reduction in Respiratory Abnormality in Patients With Rett Syndrome | Placebo | 18.503 | Standard Error 8.441 | % of change in mean counts per hr | |
| Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change | Sarizotan Low Dose | 3.6 | Standard Deviation 0.67 | score on a scale | |
| Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change | Sarizotan High Dose | 3.5 | Standard Deviation 1.12 | score on a scale | |
| Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change | Placebo | 3.4 | Standard Deviation 0.79 | score on a scale |
Source ClinicalTrials.gov
Participant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sarizotan Low Dose | 33 | |
| Overall Study | STARTED | Sarizotan High Dose | 56 | |
| Overall Study | STARTED | Placebo | 40 | |
| Overall Study | COMPLETED | Sarizotan Low Dose | 27 | |
| Overall Study | COMPLETED | Sarizotan High Dose | 46 | |
| Overall Study | COMPLETED | Placebo | 36 | |
| Overall Study | NOT COMPLETED | Sarizotan Low Dose | 6 | |
| Overall Study | NOT COMPLETED | Sarizotan High Dose | 10 | |
| Overall Study | NOT COMPLETED | Placebo | 4 |
Source ClinicalTrials.gov
Adverse events96
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 | Cough | Respiratory, thoracic and mediastinal disorders | Sarizotan Low Dose | 1 | 33 | 1 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Sarizotan High Dose | 3 | 56 | 3 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Placebo | 4 | 39 | 4 |
| other | Seizure | Nervous system disorders | Sarizotan Low Dose | 8 | 33 | 8 |
| other | Seizure | Nervous system disorders | Sarizotan High Dose | 15 | 56 | 15 |
| other | Seizure | Nervous system disorders | Placebo | 5 | 39 | 5 |
| other | Somnolence | Nervous system disorders | Sarizotan Low Dose | 2 | 33 | 2 |
| other | Somnolence | Nervous system disorders | Sarizotan High Dose | 10 | 56 | 10 |
| other | Somnolence | Nervous system disorders | Placebo | 6 | 39 | 6 |
| other | Psychomotor hyperactivity | Nervous system disorders | Sarizotan Low Dose | 4 | 33 | 4 |
1 to 10 of 96Source ClinicalTrials.gov
See all 96 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sarizotan Low Dose | 0 | 33 |
| Total, all-cause mortality | Sarizotan High Dose | 0 | 56 |
| Total, all-cause mortality | Placebo | 0 | 39 |
| Total, other adverse events | Sarizotan Low Dose | 25 | 33 |
| Total, other adverse events | Sarizotan High Dose | 46 | 56 |
| Total, other adverse events | Placebo | 30 | 39 |
| Total, serious adverse events | Sarizotan Low Dose | 2 | 33 |
| Total, serious adverse events | Sarizotan High Dose | 10 | 56 |
| Total, serious adverse events | Placebo | 5 | 39 |
Source ClinicalTrials.gov
Papers29
The papers the trial lists on ClinicalTrials.gov.
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/NCT02790034
GET https://conductscience.org/api/trials/v1/NCT02790034/events?page=2