Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Trial to Evaluate the Long-term Efficacy of Oral Aripiprazole in the Treatment of Pediatric Participants With Tourette's Disorder

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03661983
Recruitment Status : Terminated (Lack of feasibility for completion of the trial within a reasonable time frame.)
First Posted : September 7, 2018
Results First Posted : January 22, 2021
Last Update Posted : March 9, 2021
Sponsor:
Information provided by (Responsible Party):
Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Tourette's Disorder (TD)
Interventions Drug: Aripiprazole
Drug: Placebo
Enrollment 36
Recruitment Details Participants took part in the study at 13 investigative sites in Canada, the United States and Hungary from Oct 13, 2018 to Jun 30, 2020.
Pre-assignment Details Pediatric participants with a diagnosis of Tourette's Disorder were enrolled in this to receive oral aripiprazole in an Open-label Stabilization Phase and a Double-blind Randomized Withdrawal Phase and then followed for safety up to 30 days post-last dose.
Arm/Group Title Open Label Stabilization Phase: Aripiprazole Double Blind Phase: Aripiprazole Full Dose Double Blind Phase: Aripiprazole Half Dose Double Blind Phase: Placebo
Hide Arm/Group Description Participants began treatment with aripiprazole at a 2.0 mg/day dose, with the dose titrated to 5.0 mg/day after 2 days. Subsequent dose adjustments were based on the participant's weight to achieve optimum control of tics up to the maximum recommended doses based on the United States Labeling, up to Week 8 and then continued on the most stabilized dose up to minimum Week 14 or maximum Week 20. Participants who met stabilization criteria were randomized to Double-blind Randomization Phase. Participants who met stabilization criteria and randomized to receive full dose of aripiprazole i.e. 5 mg or 10 mg for <50 kg participants,and 10 mg or 20 mg for >50 kg participants (2 tablets a day), based on stabilized dose in open-label stabilization phase, up to 12 weeks in Double-Blind Phase. Participants who met stabilization criteria and randomized to receive half dose of aripiprazole i.e. 2 mg or 5 mg for <50 kg participants, and 5 mg or 10 mg for >50 kg participants (2 tablets a day), based on stabilized dose in open-label stabilization phase, up to 12 weeks in Double-Blind Phase. Participants who met randomization criteria and randomized to receive aripiprazole matching-placebo tablets, 2 daily, orally, up to 12 weeks in Double-Blind Phase.
Period Title: Open Label Stabilization Phase
Started 36 0 0 0
Completed 25 0 0 0
Not Completed 11 0 0 0
Reason Not Completed
Adverse Event             5             0             0             0
Lack of Efficacy             1             0             0             0
Withdrawal by Parent/Guardian             2             0             0             0
Study Terminated by Sponsor             3             0             0             0
Period Title: Double-blind Randomized Phase
Started 0 9 8 8
Completed 0 7 7 0
Not Completed 0 2 1 8
Reason Not Completed
Adverse Event             0             1             0             0
Disease Relapse             0             0             0             6
Withdrawal by Parent/Guardian             0             0             0             1
Study Terminated by Sponsor             0             1             1             1
Arm/Group Title Open Label Stabilization Phase: Aripiprazole
Hide Arm/Group Description Participants began treatment with aripiprazole at a 2.0 mg/day dose, with the dose titrated to 5.0 mg/day after 2 days. Subsequent dose adjustments were based on the participant's weight to achieve optimum control of tics up to the maximum recommended doses based on the United States Labeling, up to Week 8 and then continued on the most stabilized dose up to minimum Week 14 or maximum Week 20. Participants who met stabilization criteria were randomized to Double-blind Randomization Phase.
Overall Number of Baseline Participants 36
Hide Baseline Analysis Population Description
The Enrolled Sample included all participants who entered the open-label stabilization phase.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 36 participants
10.9  (3.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
Female
6
  16.7%
Male
30
  83.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
Hispanic or Latino
7
  19.4%
Not Hispanic or Latino
29
  80.6%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
American Indian or Alaska Native
1
   2.8%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   2.8%
White
32
  88.9%
More than one race
0
   0.0%
Unknown or Not Reported
2
   5.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 36 participants
Canada 9
Hungary 4
United States 23
1.Primary Outcome
Title Percentage of Participants With Relapse During the Double-blind Randomized Withdrawal Phase
Hide Description Relapse was defined as a loss of ≥ 50% of the improvement experienced during the open-label stabilization phase (i.e., improvement at the last assessment of Yale Global Tic Severity Scale (YGTSS) before randomization) on the Yale Global Tic Severity Scale Total Tic Score (YGTSS TTS). YGTSS provides an evaluation of the number, frequency, intensity, complexity, and interference of motor and phonic symptoms.
Time Frame From Randomization up to 12 weeks in Double-blind Randomized Withdrawal Phase
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat (ITT) Sample included all participants who were randomized and received at least 1 dose of randomized investigational medicinal product (IMP) were included in this dataset and were analyzed according to the treatment group they were randomized to.
Arm/Group Title Double Blind Phase: Aripiprazole Full Dose Double Blind Phase: Aripiprazole Half Dose Double Blind Phase: Placebo
Hide Arm/Group Description:
Participants who met stabilization criteria and randomized to receive full dose of aripiprazole i.e. 5 mg or 10 mg for <50 kg participants,and 10 mg or 20 mg for >50 kg participants (2 tablets a day), based on stabilized dose in open-label stabilization phase, up to 12 weeks in Double-Blind Phase.
Participants who met stabilization criteria and randomized to receive half dose of aripiprazole i.e. 2 mg or 5 mg for <50 kg participants, and 5 mg or 10 mg for >50 kg participants (2 tablets a day), based on stabilized dose in open-label stabilization phase, up to 12 weeks in Double-Blind Phase.
Participants who met randomization criteria and randomized to receive aripiprazole matching-placebo tablets, 2 daily, orally, up to 12 weeks in Double-Blind Phase.
Overall Number of Participants Analyzed 9 8 8
Measure Type: Number
Unit of Measure: percentage of participants
0.0 0.0 75.0
Time Frame From first dose up to 30 days after last dose of study drug (Up to approximately 36 weeks)
Adverse Event Reporting Description

Open-label Safety Sample included all participants that had administered at least 1 dose of IMP during the open-label stabilization phase.

Randomized Safety Sample included all participants who received at least 1 dose of randomized IMP during the double-blind randomized withdrawal phase were included and analyzed according to the treatment received.

 
Arm/Group Title Open Label Stabilization Phase: Aripiprazole Double Blind Phase: Aripiprazole Full Dose Double Blind Phase: Aripiprazole Half Dose Double Blind Phase: Placebo
Hide Arm/Group Description Participants began treatment with aripiprazole at a 2.0 mg/day dose, with the dose titrated to 5.0 mg/day after 2 days. Subsequent dose adjustments were based on the participant's weight to achieve optimum control of tics up to the maximum recommended doses based on the United States Labeling, up to Week 8 and then continued on the most stabilized dose up to minimum Week 14 or maximum Week 20. Participants who met stabilization criteria were randomized to Double-blind Randomization Phase. Participants who met stabilization criteria and randomized to receive full dose of aripiprazole i.e. 5 mg or 10 mg for <50 kg participants,and 10 mg or 20 mg for >50 kg participants (2 tablets a day), based on stabilized dose in open-label stabilization phase, up to 12 weeks in Double-blind Phase. Participants who met stabilization criteria and randomized to receive half dose of aripiprazole i.e. 2 mg or 5 mg for <50 kg participants, and 5 mg or 10 mg for >50 kg participants (2 tablets a day), based on stabilized dose in open-label stabilization phase, up to 12 weeks in Double-Blind Phase. Participants who met randomization criteria and randomized to receive aripiprazole matching-placebo tablets, 2 daily, orally, up to 12 weeks in Double-Blind Phase.
All-Cause Mortality
Open Label Stabilization Phase: Aripiprazole Double Blind Phase: Aripiprazole Full Dose Double Blind Phase: Aripiprazole Half Dose Double Blind Phase: Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/36 (0.00%)   0/9 (0.00%)   0/8 (0.00%)   0/8 (0.00%) 
Hide Serious Adverse Events
Open Label Stabilization Phase: Aripiprazole Double Blind Phase: Aripiprazole Full Dose Double Blind Phase: Aripiprazole Half Dose Double Blind Phase: Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/36 (0.00%)   1/9 (11.11%)   0/8 (0.00%)   0/8 (0.00%) 
Psychiatric disorders         
Suicidal Ideation  1  0/36 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
1
Term from vocabulary, MedDRA v23.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Open Label Stabilization Phase: Aripiprazole Double Blind Phase: Aripiprazole Full Dose Double Blind Phase: Aripiprazole Half Dose Double Blind Phase: Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   17/36 (47.22%)   3/9 (33.33%)   3/8 (37.50%)   2/8 (25.00%) 
Cardiac disorders         
Tachycardia  1  1/36 (2.78%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
General disorders         
Fatigue  1  7/36 (19.44%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Infections and infestations         
Pharyngitis streptococcal  1  0/36 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Upper respiratory tract infection  1  1/36 (2.78%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Urinary tract infection  1  0/36 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Injury, poisoning and procedural complications         
Upper limb fracture  1  0/36 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Investigations         
Weight increased  1  3/36 (8.33%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Nervous system disorders         
Somnolence  1  5/36 (13.89%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Headache  1  2/36 (5.56%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Sedation  1  4/36 (11.11%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Psychiatric disorders         
Aggression  1  0/36 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Enuresis  1  0/36 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Irritability  1  1/36 (2.78%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Restlessness  1  1/36 (2.78%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Anxiety  1  2/36 (5.56%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
1
Term from vocabulary, MedDRA v23.0
Indicates events were collected by systematic assessment
This trial was terminated early due to the withdrawal of post-marketing commitment (PMC) to FDA. The planned interim analysis was not conducted either.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Sponsor reserves the right to review results publications prior to public release and can delay such publications for a period greater than 60 days but no more than 120 days from the date that the publication is submitted to the Sponsor for review. Sponsor can require changes to the publication to protect Sponsor's intellectual property rights and/or confidential information and reserves the right to limit publication timing and scope of data published based on the number of study locations.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Global Clinical Development
Organization: Otsuka Pharmaceutical Development & Commercialization, Inc.
Phone: 1-609-524-6788
EMail: clinicaltransparency@otsuka-us.com
Layout table for additonal information
Responsible Party: Otsuka Pharmaceutical Development & Commercialization, Inc.
ClinicalTrials.gov Identifier: NCT03661983    
Other Study ID Numbers: 31-14-204
2018-002270-48 ( EudraCT Number )
First Submitted: September 5, 2018
First Posted: September 7, 2018
Results First Submitted: December 23, 2020
Results First Posted: January 22, 2021
Last Update Posted: March 9, 2021