Trial Outcomes & Findings for Open-label Extension Study of Pramipexole in the Treatment of Children and Adolescents With Tourette Syndrome (NCT NCT00681863)
NCT ID: NCT00681863
Last Updated: 2014-05-23
Results Overview
Number of patients with Adverse Events leading to discontinuation of trial drug
TERMINATED
PHASE3
45 participants
24 Weeks
2014-05-23
Participant Flow
Participant milestones
| Measure |
Pramipexole
4 weeks individual dose titration starting with 0.0625 mg BID (twice daily), down-titration to 0.0625 QD (once daily) if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID (three times daily) and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
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|---|---|
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Overall Study
STARTED
|
45
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
23
|
Reasons for withdrawal
| Measure |
Pramipexole
4 weeks individual dose titration starting with 0.0625 mg BID (twice daily), down-titration to 0.0625 QD (once daily) if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID (three times daily) and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
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|---|---|
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Overall Study
Adverse Event
|
1
|
|
Overall Study
Lost to Follow-up
|
2
|
|
Overall Study
Lack of Efficacy
|
2
|
|
Overall Study
Other
|
18
|
Baseline Characteristics
Open-label Extension Study of Pramipexole in the Treatment of Children and Adolescents With Tourette Syndrome
Baseline characteristics by cohort
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
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|---|---|
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Age, Continuous
|
11.8 Years
STANDARD_DEVIATION 2.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
36 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
38 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
5 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
40 participants
n=5 Participants
|
|
Duration of Tourettes Syndrome
More than 5 years
|
10 Participants
n=5 Participants
|
|
Duration of Tourettes Syndrome
Less than 1 year
|
15 Participants
n=5 Participants
|
|
Duration of Tourettes Syndrome
1 to 5 years
|
20 Participants
n=5 Participants
|
|
Height
|
152.6 centimeters
STANDARD_DEVIATION 19.4 • n=5 Participants
|
|
Weight
|
53.01 kilograms
STANDARD_DEVIATION 21.58 • n=5 Participants
|
|
Body mass index
|
22.064 kilograms/square meter
STANDARD_DEVIATION 5.930 • n=5 Participants
|
|
Body temperature
|
36.752 Degrees centigrade
STANDARD_DEVIATION 0.718 • n=5 Participants
|
|
Respiration
|
17.4 breaths/minute
STANDARD_DEVIATION 2.0 • n=5 Participants
|
|
Obsessive Compulsive Disorder
Positive
|
4 Participants
n=5 Participants
|
|
Obsessive Compulsive Disorder
Intermediate
|
4 Participants
n=5 Participants
|
|
Obsessive Compulsive Disorder
Negative
|
37 Participants
n=5 Participants
|
|
Attention Deficit Hyperactive Disorder
Positive
|
17 participants
n=5 Participants
|
|
Attention Deficit Hyperactive Disorder
Intermediate
|
6 participants
n=5 Participants
|
|
Attention Deficit Hyperactive Disorder
Negative
|
22 participants
n=5 Participants
|
|
Treatment received in previous trial (NCT00558467)
Received placebo
|
14 Participants
n=5 Participants
|
|
Treatment received in previous trial (NCT00558467)
Received pramipexole
|
31 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 WeeksPopulation: Treated set
Number of patients with Adverse Events leading to discontinuation of trial drug
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
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|---|---|
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Patients With Adverse Events Leading to Discontinuation of Trial Drug
|
1 participants
|
SECONDARY outcome
Timeframe: baseline and week 24Population: The Full Analysis Set (FAS) with last observation carried forward (LOCF).
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-9.8 Score on a scale
Standard Deviation 8.9
|
SECONDARY outcome
Timeframe: baseline and Week 24 (end of treatment visit)Population: The Full Analysis Set (FAS) with Last Observation Carried Forward (LOCF).
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-22.0 Score on a scale
Standard Deviation 21.0
|
SECONDARY outcome
Timeframe: baseline and Week 1Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
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Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-7.2 Score on a scale
Standard Deviation 8.5
|
SECONDARY outcome
Timeframe: baseline and Week 2Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-8.3 Score on a scale
Standard Deviation 7.7
|
SECONDARY outcome
Timeframe: baseline and Week 3Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=44 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
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Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-8.6 Score on a scale
Standard Deviation 8.6
|
SECONDARY outcome
Timeframe: baseline and week 4Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=44 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-9.3 Score on a scale
Standard Deviation 9.7
|
SECONDARY outcome
Timeframe: baseline and Week 8Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=41 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-10.7 Score on a scale
Standard Deviation 9.4
|
SECONDARY outcome
Timeframe: baseline and Week 12Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=35 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-12.3 Score on a scale
Standard Deviation 9.0
|
SECONDARY outcome
Timeframe: baseline and Week 16Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=32 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-12.4 Score on a scale
Standard Deviation 9.3
|
SECONDARY outcome
Timeframe: baseline and Week 20Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=26 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-11.7 Score on a scale
Standard Deviation 11.3
|
SECONDARY outcome
Timeframe: baseline and Week 24Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Tic Score is the sum of ten individual ratings of the impairment due to tics. Each scale ranges from 0 (None/Absent) to 5 (Severe) and total score ranges from 0 to 50.
Outcome measures
| Measure |
Pramipexole
n=22 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Tic Score (TTS) of the Yale Global Tic Severity Scale
|
-9.3 Score on a scale
Standard Deviation 10.4
|
SECONDARY outcome
Timeframe: baseline and Week 1Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-14.5 Score on a scale
Standard Deviation 18.2
|
SECONDARY outcome
Timeframe: baseline and Week 2Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-17.4 Score on a scale
Standard Deviation 17.6
|
SECONDARY outcome
Timeframe: baseline and Week 3Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=44 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-18.4 Score on a scale
Standard Deviation 19.8
|
SECONDARY outcome
Timeframe: baseline and Week 4Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=44 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-20.9 Score on a scale
Standard Deviation 21.5
|
SECONDARY outcome
Timeframe: baseline and Week 8Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=41 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-22.4 Score on a scale
Standard Deviation 21.9
|
SECONDARY outcome
Timeframe: baseline and Week 12Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=35 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-27.7 Score on a scale
Standard Deviation 20.9
|
SECONDARY outcome
Timeframe: baseline and Week 16Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=32 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-28.3 Score on a scale
Standard Deviation 20.2
|
SECONDARY outcome
Timeframe: baseline and Week 20Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=26 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-26.7 Score on a scale
Standard Deviation 24.9
|
SECONDARY outcome
Timeframe: baseline and Week 24Population: Observed Cases Full Analysis Set (OC FAS). All participants in FAS having observed data at the particular timepoint.
Total Score is a rating of the overall impairment due to motor and phonic tics. The scale ranges from 0 (None) to 50 (Severe).
Outcome measures
| Measure |
Pramipexole
n=22 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Mean Change From Baseline in Total Score of the Yale Global Tic Severity Scale
|
-22.0 Score on a scale
Standard Deviation 23.6
|
SECONDARY outcome
Timeframe: week 24Population: The Full Analysis Set (FAS) with last observation carried forward (LOCF).
Overall improvement during the last week compared to baseline ranging from 1 (very much improved), 2 (much improved), to 7 (very much worse). Responder has 'very much' or 'much' improvement. Non responder has less improvement than 'much' improvement.
Outcome measures
| Measure |
Pramipexole
n=42 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Clinical Global Impressions - Severity of Illness
|
-1.1 score on a scale
Standard Deviation 1.1
|
SECONDARY outcome
Timeframe: week 24Population: The Full Analysis Set (FAS) with last observation carried forward (LOCF).
Assessment of the overall severity of illness on a scale ranging from 1 (not at all ill) to 7 (among the most extremely ill patients). Overall improvement during the last week compared to baseline ranging from 1 (very much improved), 2 (much improved), to 7 (very much worse). Responder has 'very much' or 'much' improvement. Non responder has less improvement than 'much' improvement.
Outcome measures
| Measure |
Pramipexole
n=42 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Clinical Global Impressions - Severity of Illness, Categorized
Improved (change score <= -2)
|
11 participants
|
|
Clinical Global Impressions - Severity of Illness, Categorized
Unchanged (change score of -1, 0, or +1)
|
31 participants
|
|
Clinical Global Impressions - Severity of Illness, Categorized
Worsened (change score >= +2)
|
0 participants
|
SECONDARY outcome
Timeframe: week 1Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 2Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 3Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 4Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 8Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 12Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 16Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 20Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 24Population: The Full Analysis Set (FAS) with last observation carried forward (LOCF).
Assessment of the overall improvement during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much improved) to 7 (very much worse).
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Clinical Global Impressions - Improvement
Responder (Much improved or Very much improved)
|
22 participants
|
|
Clinical Global Impressions - Improvement
Not Responder
|
23 participants
|
SECONDARY outcome
Timeframe: week 1Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 2Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 3Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 4Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 8Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 12Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 16Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 20Population: This outcome measure was not analyzed due to the premature ending of the trial.
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: week 24Population: The Full Analysis Set (FAS) with last observation carried forward (LOCF).
Assessment of the change of the patient's overall condition during the last week compared to the patient's condition at baseline on a scale ranging from 1 (very much better) to 7 (very much worse). A responder is defined as having a response of very much (1) or much better (2).
Outcome measures
| Measure |
Pramipexole
n=45 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Patient Global Impression - Improvement
Responder (Much better or Very much better)
|
17 participants
|
|
Patient Global Impression - Improvement
Not Responder
|
28 participants
|
SECONDARY outcome
Timeframe: Baseline and 24 weeksPopulation: Observed Cases Treated set (OC TS). All participants in Treated Set having observed data at the particular timepoint.
Frequency of patients with possible clinically significant abnormalities for laboratory parameters (blood hematology and electrolyte assessments, serum chemistry, including follicle-stimulating hormone (FSH), luteinizing hormone (LH) and estradiol for pubertal female patients, prolactin in all patients, testosterone in pubertal male patients, urine analysis)
Outcome measures
| Measure |
Pramipexole
n=43 Participants
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Frequency of Patients With Possible Clinically Significant Abnormalities for Laboratory Parameters
Haemoglobin - decrease
|
2 participants
|
|
Frequency of Patients With Possible Clinically Significant Abnormalities for Laboratory Parameters
Eosinophils - increase
|
3 participants
|
|
Frequency of Patients With Possible Clinically Significant Abnormalities for Laboratory Parameters
Phosphate - increase
|
2 participants
|
|
Frequency of Patients With Possible Clinically Significant Abnormalities for Laboratory Parameters
Alkaline phosphatase - increase
|
1 participants
|
Adverse Events
Pramipexole
Serious adverse events
| Measure |
Pramipexole
n=45 participants at risk
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Nervous system disorders
Syncope
|
2.2%
1/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
Other adverse events
| Measure |
Pramipexole
n=45 participants at risk
4 weeks individual dose titration starting with 0.0625 mg BID, down-titration to 0.0625 QD if not tolerated, and next steps 0.125 mg BID, 0.125 mg TID and 0.25 mg BID, according to efficacy assessment; established optimal dose for the remainder of the 24-week treatment period.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Gastrointestinal disorders
Nausea
|
17.8%
8/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Gastrointestinal disorders
Vomiting
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
General disorders
Fatigue
|
13.3%
6/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
General disorders
Pyrexia
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Immune system disorders
Hypersensitivity
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Infections and infestations
Nasopharyngitis
|
11.1%
5/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Infections and infestations
Upper respiratory tract infection
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
6.7%
3/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Metabolism and nutrition disorders
Increased appetite
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.7%
3/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Nervous system disorders
Dizziness
|
6.7%
3/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Nervous system disorders
Headache
|
20.0%
9/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Nervous system disorders
Somnolence
|
6.7%
3/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Psychiatric disorders
Tic
|
8.9%
4/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.7%
3/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
|
Vascular disorders
Orthostatic hypotension
|
6.7%
3/45 • up to 24 weeks
At each visit, the Investigator asked the patient and parent(s) (legal guardians) to elucidate any adverse events that may have occurred since the last visit in order to document if any adverse events had occurred, and carefully recorded the adverse event information reported.
|
Additional Information
Boehringer Ingelheim Call Center
Boehringer Ingelheim Pharmaceuticals
Results disclosure agreements
- Principal investigator is a sponsor employee Any publication of the result of this trial must be consistent with the Boehringer Ingelheim publication policy. The rights of the investigator and of the sponsor with regard to publication of the results of this trial are described in the investigator contract
- Publication restrictions are in place
Restriction type: OTHER