Trial · NCT04173260
An Open-label Study to Define the Safety, Tolerability and Clinical Activity of Deutetrabenazine (AUstedo) in Adult Study Subjects With DYsTonia
NCT04173260, Phase 1, Phase 2, Completed, University of Pennsylvania; results first posted 2024-06-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-label Study to Define the Safety, Tolerability and Clinical Activity of Deutetrabenazine (AUstedo) in Adult Study Subjects With DYsTonia. Trials, record NCT04173260. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04173260
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-label Study to Define the Safety, Tolerability and Clinical Activity of Deutetrabenazine (AUstedo) in Adult Study Subjects With DYsTonia
- NCT number
- NCT04173260
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 15 participants, actual
- Lead sponsor
- University of Pennsylvania
- Conditions
- Dystonia, Primary
- Interventions
- Deutetrabenazine 6 MG
- First posted
- 2019-11-21
- Last updated
- 2024-09-20
- Results first posted
- 2024-06-12
- Limitations and caveats
* Open label design~* Patients were allowed to continue receiving BoNT injections during the trial~* No genetic testing pursued~* Patients with different phenomenologies were allowed to participate and data analyzed in aggregate
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Study Subjects Able to Titrate up to the Maximum Tolerated Dose | 3 months | Proportion of study subjects able to titrate up to 48 mg/d (or up to 36 mg/d if receiving a strong CYP2D6 inhibitor) and able to complete the study at this dosage |
| Secondary | Number of Participants With Change in Suicidality | Baseline and 3 months | Documentation of change in the Columbia Suicide Severity Rating Scale. Items on this scale are both binary (Yes/No) and numeric (0-5). "No" answers and lower numeric values indicate a better outcome. |
| Secondary | Change in Median Daytime Somnolence Score Among Subjects | Baseline and 3 months | Documentation of change in the Stanford Sleepiness Scale. Scale was assessed at the initial and last study visit; difference was calculated between both scores, and reported herein as a median among all participants' scores and full range. Items on this scale are numeric (1-7). Lower numeric values indicate a better outcome. |
| Secondary | Change in Median MMSE Score Among Subjects | Baseline and 3 months | Documentation of change in the Mini Mental Scale. Scale was assessed at the initial and last study visit; difference was calculated between both scores, and reported herein as a median among all participants' scores and full range. The higher the total score on this scale, the better the outcome. Values range from 0 to a maximum of 30. |
| Secondary | Development of Parkinsonism, as Determined by Change in Median MDS-UPDRS III Score Among Subjects | Baseline and 3 months | Documentation of change in the MDS-Unified Parkinson's Disease Rating Scale, Part III. Scale was assessed at the initial and last study visit; difference was calculated between both scores, and reported herein as a median among all participants' scores and full range. The lower the total score on this scale, the better the outcome. Values range from 0 to a maximum of 128. |
| Secondary | Change in Median PGI-I Score | Baseline and 3 months | Documentation of change in the Patient Global Impression of Improvement Scale (PGI-I). This is a Likert scale, with values from 1-7. A value of 1 indicates the best outcome. A value of 4 indicates no perceived change. |
| Secondary | Change in Dystonia Severity, as Determined by the Change in Median GDS Score | Baseline and 3 months | Documentation of change in the Global Dystonia Rating Scale. Scale was assessed blindly from videos captured at the initial and last study visit; difference was calculated between both scores, and reported herein as a median among all participants' scores and full range. The lower the total score on this scale, the better the outcome. Values range from 0 to a maximum of 140. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion of Study Subjects Able to Titrate up to the Maximum Tolerated Dose | 3 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Change in Suicidality | Baseline and 3 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Change in Median Daytime Somnolence Score Among Subjects | Baseline and 3 months | score on a scale | MEDIAN | |
| SECONDARY | Change in Median MMSE Score Among Subjects | Baseline and 3 months | score on a scale | MEDIAN | |
| SECONDARY | Development of Parkinsonism, as Determined by Change in Median MDS-UPDRS III Score Among Subjects | Baseline and 3 months | score on a scale | MEDIAN | |
| SECONDARY | Change in Median PGI-I Score | Baseline and 3 months | score on a scale | MEDIAN | |
| SECONDARY | Change in Dystonia Severity, as Determined by the Change in Median GDS Score | Baseline and 3 months | score on a scale | MEDIAN |
Source ClinicalTrials.gov
Results by group7
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion of Study Subjects Able to Titrate up to the Maximum Tolerated Dose | Intervention Arm - Oral Deutetrabenazine | 6 | Participants | ||
| Number of Participants With Change in Suicidality | Intervention Arm - Oral Deutetrabenazine | 0 | Participants | ||
| Change in Median Daytime Somnolence Score Among Subjects | Intervention Arm - Oral Deutetrabenazine | 0 | Full Range -5 to 2 | score on a scale | |
| Change in Median MMSE Score Among Subjects | Intervention Arm - Oral Deutetrabenazine | 1 | Full Range -4 to 3 | score on a scale | |
| Development of Parkinsonism, as Determined by Change in Median MDS-UPDRS III Score Among Subjects | Intervention Arm - Oral Deutetrabenazine | 2 | Full Range -19 to 13 | score on a scale | |
| Change in Median PGI-I Score | Intervention Arm - Oral Deutetrabenazine | 4 | Full Range 1 to 5 | score on a scale | |
| Change in Dystonia Severity, as Determined by the Change in Median GDS Score | Intervention Arm - Oral Deutetrabenazine | 0 | Full Range -12 to 2 | 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 | Intervention Arm - Oral Deutetrabenazine | 15 | |
| Overall Study | COMPLETED | Intervention Arm - Oral Deutetrabenazine | 11 | |
| Overall Study | NOT COMPLETED | Intervention Arm - Oral Deutetrabenazine | 4 |
Source ClinicalTrials.gov
Adverse events7
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 | Fatigue | Nervous system disorders | Intervention Arm - Oral Deutetrabenazine | 6 | 15 | |
| other | Somnolence | Nervous system disorders | Intervention Arm - Oral Deutetrabenazine | 3 | 15 | |
| other | COVID-19 infection | Respiratory, thoracic and mediastinal disorders | Intervention Arm - Oral Deutetrabenazine | 3 | 15 | |
| other | Diarrhea | Gastrointestinal disorders | Intervention Arm - Oral Deutetrabenazine | 2 | 15 | |
| other | Headache | Nervous system disorders | Intervention Arm - Oral Deutetrabenazine | 1 | 15 | |
| other | Akathisia | Nervous system disorders | Intervention Arm - Oral Deutetrabenazine | 1 | 15 | |
| serious | Suicidal depression | Psychiatric disorders | Intervention Arm - Oral Deutetrabenazine | 0 | 15 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Intervention Arm - Oral Deutetrabenazine | 0 | 15 |
| Total, other adverse events | Intervention Arm - Oral Deutetrabenazine | 15 | 15 |
| Total, serious adverse events | Intervention Arm - Oral Deutetrabenazine | 0 | 15 |
Source ClinicalTrials.gov
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/NCT04173260
GET https://conductscience.org/api/trials/v1/NCT04173260/registered