Trial Outcomes & Findings for An Open-label Study to Define the Safety, Tolerability and Clinical Activity of Deutetrabenazine (AUstedo) in Adult Study Subjects With DYsTonia (NCT NCT04173260)

NCT ID: NCT04173260

Last Updated: 2024-09-20

Results Overview

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

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 15 participants
• Primary outcome timeframe: 3 months
• Results posted on: 2024-09-20

Participant Flow

Fifteen subjects were recruited between April 2021 and December 2023.

All subjects received IP; there was no placebo arm.

Participant milestones

Measure	Intervention Arm - Oral Deutetrabenazine This is the only arm for this trial. All subjects will receive oral Deutetrabanazine. Deutetrabenazine 6 MG: Increasing doses of Deutetrabenazine
Overall Study STARTED	15
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	Intervention Arm - Oral Deutetrabenazine This is the only arm for this trial. All subjects will receive oral Deutetrabanazine. Deutetrabenazine 6 MG: Increasing doses of Deutetrabenazine
Overall Study Early termination	4

Baseline Characteristics

An Open-label Study to Define the Safety, Tolerability and Clinical Activity of Deutetrabenazine (AUstedo) in Adult Study Subjects With DYsTonia

Baseline characteristics by cohort

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants This is the only arm for this trial. All subjects will receive oral Deutetrabanazine. Deutetrabenazine 6 MG: Increasing doses of Deutetrabenazine
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	7 Participants n=5 Participants
Age, Continuous	63 years n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: 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

Outcome measures

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants All subjects in this study received IP in an open label fashion
Proportion of Study Subjects Able to Titrate up to the Maximum Tolerated Dose	6 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants All subjects in this study received IP in an open label fashion
Number of Participants With Change in Suicidality	0 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants All subjects in this study received IP in an open label fashion
Change in Median Daytime Somnolence Score Among Subjects	0 score on a scale Interval -5.0 to 2.0

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants All subjects in this study received IP in an open label fashion
Change in Median MMSE Score Among Subjects	1 score on a scale Interval -4.0 to 3.0

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants All subjects in this study received IP in an open label fashion
Development of Parkinsonism, as Determined by Change in Median MDS-UPDRS III Score Among Subjects	2 score on a scale Interval -19.0 to 13.0

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants All subjects in this study received IP in an open label fashion
Change in Median PGI-I Score	4 score on a scale Interval 1.0 to 5.0

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Arm - Oral Deutetrabenazine n=15 Participants All subjects in this study received IP in an open label fashion
Change in Dystonia Severity, as Determined by the Change in Median GDS Score	0 score on a scale Interval -12.0 to 2.0

Adverse Events

Intervention Arm - Oral Deutetrabenazine

Serious events: 0 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Measure	Intervention Arm - Oral Deutetrabenazine n=15 participants at risk All subjects in this study received IP in an open label fashion
Psychiatric disorders Suicidal depression	0.00% 0/15 • Up to 13 weeks

Other adverse events

Measure	Intervention Arm - Oral Deutetrabenazine n=15 participants at risk All subjects in this study received IP in an open label fashion
Nervous system disorders Fatigue	40.0% 6/15 • Up to 13 weeks
Nervous system disorders Somnolence	20.0% 3/15 • Up to 13 weeks
Respiratory, thoracic and mediastinal disorders COVID-19 infection	20.0% 3/15 • Up to 13 weeks
Gastrointestinal disorders Diarrhea	13.3% 2/15 • Up to 13 weeks
Nervous system disorders Headache	6.7% 1/15 • Up to 13 weeks
Nervous system disorders Akathisia	6.7% 1/15 • Up to 13 weeks

Additional Information

Dr. Andres Deik

University of Pennsylvania

Phone: 2158297049

Email: adeik@upenn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place