Trial Outcomes & Findings for Efficacy and Tolerability of IRL790 in Parkinson's Disease Dyskinesia (NCT NCT03368170)

NCT ID: NCT03368170

Last Updated: 2022-03-31

Results Overview

The change from baseline to day 28 of treatment (Visit 4) in the sum of the items comprising the Unified Dyskinesia Rating Scale (UDysRS). The Unified Dyskinesia Rating Scale (UDysRS) is administered to assess dyskinesia. The scoring range is 0-104, where higher score means more dyskinesia.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

75 participants

Primary outcome timeframe

Baseline and 4 weeks

Results posted on

2022-03-31

Participant Flow

The study was conducted as an outpatient trial and patients were identified from 20 sites across two countries. The first patient was enrolled 13 April 2018 and the last past completed 12 June 2019.

Patients completed two 24-hour diaries during run-in of which one had to be valid prior randomization.

Participant milestones

Participant milestones
Measure
Mesdopetam
Mesdopetam capsule, 2.5 mg oral administration. Dose given twice daily (5 mg, 7.5 mg or 10 mg b.i.d.) The first two weeks of treatment allowed for per patient titration of study medication to the highest tolerated predefined dose, after which patients continued on this highest per patient tolerated dose for an additional two weeks.
Placebo
Matching placebo capsule, oral administration. Dose given twice daily. The first two weeks of treatment allowed for per patient titration of study medication to the highest tolerated number of placebo capsules (1 capsule x 4), after which patients continued on this highest tolerated dose for an additional two weeks.
Overall Study
STARTED
39
36
Overall Study
COMPLETED
37
35
Overall Study
NOT COMPLETED
2
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Mesdopetam
Mesdopetam capsule, 2.5 mg oral administration. Dose given twice daily (5 mg, 7.5 mg or 10 mg b.i.d.) The first two weeks of treatment allowed for per patient titration of study medication to the highest tolerated predefined dose, after which patients continued on this highest per patient tolerated dose for an additional two weeks.
Placebo
Matching placebo capsule, oral administration. Dose given twice daily. The first two weeks of treatment allowed for per patient titration of study medication to the highest tolerated number of placebo capsules (1 capsule x 4), after which patients continued on this highest tolerated dose for an additional two weeks.
Overall Study
Adverse Event
2
1

Baseline Characteristics

Efficacy and Tolerability of IRL790 in Parkinson's Disease Dyskinesia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Mesdopetam
n=39 Participants
Mesdopetam (IRL790): 2.5 mg white hard HPMC capsule, oral administration
Placebo
n=36 Participants
Placebo: Matching placebo capsule, white hard HPMC capsule, oral administration
Total
n=75 Participants
Total of all reporting groups
Age, Continuous
65.5 years
STANDARD_DEVIATION 8.9 • n=93 Participants
67.7 years
STANDARD_DEVIATION 7.7 • n=4 Participants
66.6 years
STANDARD_DEVIATION 8.36 • n=27 Participants
Sex: Female, Male
Female
20 Participants
n=93 Participants
13 Participants
n=4 Participants
33 Participants
n=27 Participants
Sex: Female, Male
Male
19 Participants
n=93 Participants
23 Participants
n=4 Participants
42 Participants
n=27 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=93 Participants
1 Participants
n=4 Participants
1 Participants
n=27 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
39 Participants
n=93 Participants
35 Participants
n=4 Participants
74 Participants
n=27 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
Asian
1 Participants
n=93 Participants
0 Participants
n=4 Participants
1 Participants
n=27 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
White
38 Participants
n=93 Participants
35 Participants
n=4 Participants
73 Participants
n=27 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
1 Participants
n=4 Participants
1 Participants
n=27 Participants
Body Mass Index
26.6 kg/m^2
STANDARD_DEVIATION 7.2 • n=93 Participants
25.3 kg/m^2
STANDARD_DEVIATION 4.2 • n=4 Participants
26 kg/m^2
STANDARD_DEVIATION 5.92 • n=27 Participants
Height
166.7 centimeters
STANDARD_DEVIATION 10.3 • n=93 Participants
169 centimeters
STANDARD_DEVIATION 9.4 • n=4 Participants
168 centimeters
STANDARD_DEVIATION 9.87 • n=27 Participants
Years with Parkinson's disease
10.7 years
STANDARD_DEVIATION 5.9 • n=93 Participants
10.7 years
STANDARD_DEVIATION 4.3 • n=4 Participants
10.7 years
STANDARD_DEVIATION 5.15 • n=27 Participants

PRIMARY outcome

Timeframe: Baseline and 4 weeks

Population: Full analysis set (all randomized and treated patients who received one or more doses and who provided post baseline data whether or not they fully complied with the requirements of the protocol).

The change from baseline to day 28 of treatment (Visit 4) in the sum of the items comprising the Unified Dyskinesia Rating Scale (UDysRS). The Unified Dyskinesia Rating Scale (UDysRS) is administered to assess dyskinesia. The scoring range is 0-104, where higher score means more dyskinesia.

Outcome measures

Outcome measures
Measure
Mesdopetam
n=36 Participants
Mesdopetam: 2.5 mg white hard HPMC capsule, oral administration
Placebo
n=34 Participants
Placebo: Matching placebo capsule, white hard HPMC capsule, oral administration
Unified Dyskinesia Rating Scale (UDysRS)
-4.2 score on a scale
Interval -7.3 to -1.0
-7.0 score on a scale
Interval -10.3 to -3.8

SECONDARY outcome

Timeframe: Baseline and 4 weeks

Population: Full analysis set (all randomized and treated patients who received one or more doses and who provided post baseline data whether or not they fully complied with the requirements of the protocol).

Change in MDS-UPDRS sum score of questions 4.1 (Time spent with dyskinesias) and 4.2 (Functional impact of dyskinesias) in part IV from baseline to visit 4. Minimum score is 0 and maximum score is 8. A higher score means more dyskinesia.

Outcome measures

Outcome measures
Measure
Mesdopetam
n=37 Participants
Mesdopetam: 2.5 mg white hard HPMC capsule, oral administration
Placebo
n=35 Participants
Placebo: Matching placebo capsule, white hard HPMC capsule, oral administration
Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part IV, Question 4.1 and 4.2
-1.3 score on a scale
Interval -1.8 to 0.8
-0.6 score on a scale
Interval -1.1 to 0.1

SECONDARY outcome

Timeframe: Baseline and 4 weeks

Population: Full analysis set (all randomized and treated patients who received one or more doses and who provided post baseline data whether or not they fully complied with the requirements of the protocol).

Change in MDS-UPDRS sum score of parts II+III (Motor aspects of Experiences of Daily living + Motor Examination) from baseline to visit 4. Minimum value is 0 and maximum value is 124. Higher score mean a worse outcome.

Outcome measures

Outcome measures
Measure
Mesdopetam
n=37 Participants
Mesdopetam: 2.5 mg white hard HPMC capsule, oral administration
Placebo
n=35 Participants
Placebo: Matching placebo capsule, white hard HPMC capsule, oral administration
Unified Parkinson's Disease Rating Scale (MDS-UPDRS), Part II and III
-3.0 score on a scale
Interval -6.5 to 0.6
-2.2 score on a scale
Interval -5.9 to 1.4

OTHER_PRE_SPECIFIED outcome

Timeframe: Run-in and 4 weeks

Population: Full analysis set (all randomized and treated patients who received one or more doses and who provided post baseline data whether or not they fully complied with the requirements of the protocol).

Change in ON-time with troublesome dyskinesia as assessed by patient completed 24-hour diaries, from run-in to visit 4. This is a self administered diary where patients assess their motor state every half hour during 24 hours. The different motor states assessed: ON, ON with troublesome dyskinesia, OFF and asleep.

Outcome measures

Outcome measures
Measure
Mesdopetam
n=37 Participants
Mesdopetam: 2.5 mg white hard HPMC capsule, oral administration
Placebo
n=34 Participants
Placebo: Matching placebo capsule, white hard HPMC capsule, oral administration
Change in Daily Hours Spent in ON-time With Troublesome Dyskinesia as Assessed by 24-hour Patient Diaries
-3.3 daily hours
Interval -4.3 to -2.4
-1.7 daily hours
Interval -2.8 to -0.7

Adverse Events

Mesdopetam

Serious events: 0 serious events
Other events: 29 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 28 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Mesdopetam
n=39 participants at risk
Mesdopetam (IRL790): 2.5 mg white hard HPMC capsule, oral administration
Placebo
n=36 participants at risk
Placebo: Matching placebo capsule, white hard HPMC capsule, oral administration
Nervous system disorders
Dizziness
5.1%
2/39 • 5 weeks
8.3%
3/36 • 5 weeks
Nervous system disorders
Dyskinesia
10.3%
4/39 • 5 weeks
5.6%
2/36 • 5 weeks
Nervous system disorders
Freezing phenomenon
2.6%
1/39 • 5 weeks
11.1%
4/36 • 5 weeks
Nervous system disorders
Headache
12.8%
5/39 • 5 weeks
13.9%
5/36 • 5 weeks
Nervous system disorders
On and off phenomenon
5.1%
2/39 • 5 weeks
2.8%
1/36 • 5 weeks
Nervous system disorders
Parkinsonism
23.1%
9/39 • 5 weeks
8.3%
3/36 • 5 weeks
General disorders
Fatigue
5.1%
2/39 • 5 weeks
25.0%
9/36 • 5 weeks
Nervous system disorders
Gait disturbance
0.00%
0/39 • 5 weeks
5.6%
2/36 • 5 weeks
Psychiatric disorders
Insomnia
5.1%
2/39 • 5 weeks
5.6%
2/36 • 5 weeks
Psychiatric disorders
Sleep disorder
7.7%
3/39 • 5 weeks
5.6%
2/36 • 5 weeks
Musculoskeletal and connective tissue disorders
Muscle spasm
0.00%
0/39 • 5 weeks
5.6%
2/36 • 5 weeks
Musculoskeletal and connective tissue disorders
Muscoloskeletal stiffness
2.6%
1/39 • 5 weeks
8.3%
3/36 • 5 weeks
Gastrointestinal disorders
Nausea
0.00%
0/39 • 5 weeks
11.1%
4/36 • 5 weeks
Injury, poisoning and procedural complications
Fall
10.3%
4/39 • 5 weeks
8.3%
3/36 • 5 weeks
Infections and infestations
Urinary tract infection
5.1%
2/39 • 5 weeks
5.6%
2/36 • 5 weeks
Skin and subcutaneous tissue disorders
Erythema
0.00%
0/39 • 5 weeks
5.6%
2/36 • 5 weeks

Additional Information

Joakim Tedroff/Chief Medical Officer

Integrative Research Laboratories AB

Phone: + 46 707 601691

Results disclosure agreements

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