Trial Outcomes & Findings for Efficacy and Safety Study of CVT-301 In Parkinson's Disease Patients With OFF Episodes (NCT NCT02240030)
NCT ID: NCT02240030
Last Updated: 2019-05-28
Results Overview
Primary Efficacy Analysis: Change from Predose in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III Score at 30 Minutes post-dose at Week 12 for CVT-301 High Dose versus Placebo (ITT Population). UPDRS Part III ranges from 0 minimum to 86 maximum, with lower scores indicating better movement. Units on a scale.
COMPLETED
PHASE3
351 participants
30 minutes post-dose at week 12
2019-05-28
Participant Flow
Participant milestones
| Measure |
CVT-301 Low Dose
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
CVT-301 High Dose
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
Overall Study
STARTED
|
113
|
114
|
112
|
|
Overall Study
COMPLETED
|
96
|
97
|
97
|
|
Overall Study
NOT COMPLETED
|
17
|
17
|
15
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy and Safety Study of CVT-301 In Parkinson's Disease Patients With OFF Episodes
Baseline characteristics by cohort
| Measure |
CVT-301 Low Dose
n=113 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
CVT-301 High Dose
n=114 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=112 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
Total
n=339 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
54 Participants
n=99 Participants
|
58 Participants
n=107 Participants
|
55 Participants
n=206 Participants
|
167 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
59 Participants
n=99 Participants
|
56 Participants
n=107 Participants
|
57 Participants
n=206 Participants
|
172 Participants
n=7 Participants
|
|
Age, Continuous
|
63.9 years
STANDARD_DEVIATION 9.24 • n=99 Participants
|
63.5 years
STANDARD_DEVIATION 7.97 • n=107 Participants
|
62.6 years
STANDARD_DEVIATION 8.83 • n=206 Participants
|
63.3 years
STANDARD_DEVIATION 8.69 • n=7 Participants
|
|
Sex: Female, Male
Female
|
33 Participants
n=99 Participants
|
31 Participants
n=107 Participants
|
26 Participants
n=206 Participants
|
90 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
80 Participants
n=99 Participants
|
83 Participants
n=107 Participants
|
86 Participants
n=206 Participants
|
249 Participants
n=7 Participants
|
|
Region of Enrollment
Canada
|
9 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
20 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
86 Participants
n=99 Participants
|
81 Participants
n=107 Participants
|
81 Participants
n=206 Participants
|
248 Participants
n=7 Participants
|
|
Region of Enrollment
Poland
|
18 Participants
n=99 Participants
|
29 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
66 Participants
n=7 Participants
|
|
Region of Enrollment
Spain
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: 30 minutes post-dose at week 12Population: 12 participants were randomized but withdrew before receiving any study drug (4 placebo, 2 CVT-301 low-dose, 6 CVT-301 high-dose).
Primary Efficacy Analysis: Change from Predose in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III Score at 30 Minutes post-dose at Week 12 for CVT-301 High Dose versus Placebo (ITT Population). UPDRS Part III ranges from 0 minimum to 86 maximum, with lower scores indicating better movement. Units on a scale.
Outcome measures
| Measure |
CVT-301 High Dose
n=114 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=112 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
Placebo
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
Unified Parkinson's Disease Rating Scale (UPDRS) Part III
|
-9.83 units on a scale
Standard Error 1.506
|
-5.91 units on a scale
Standard Error 1.500
|
—
|
SECONDARY outcome
Timeframe: at week 12Population: 12 participants were randomized but withdrew before receiving any study drug (4 placebo, 2 CVT-301 low-dose, 6 CVT-301 high-dose).
Examiner-assessed observation - Subject Achieving Resolution of an OFF to and ON state within 60 Minutes at TV4 - Observed
Outcome measures
| Measure |
CVT-301 High Dose
n=113 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=114 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
Placebo
n=112 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
Proportion of Patients Achieving Resolution of an OFF to an ON State Within 60 Minutes.
|
55 Participants
|
56 Participants
|
35 Participants
|
SECONDARY outcome
Timeframe: at week 12Population: 12 participants were randomized but withdrew before receiving any study drug (4 placebo, 2 CVT-301 low-dose, 6 CVT-301 high-dose).
Change from Predose in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III Score at 20 Minutes post-dose at Week 12 for CVT-301 High Dose versus Placebo (ITT Population) and CVT 301 Low Dose versus Placebo. UPDRS Part III ranges from 0 minimum to 86 maximum, with lower scores indicating better movement. Units on a scale.
Outcome measures
| Measure |
CVT-301 High Dose
n=113 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=114 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
Placebo
n=112 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
UPDRS Part III Motor Score at 20 Minutes
|
-8.47 units on a scale
Interval -11.11 to -5.82
|
-9.04 units on a scale
Interval -11.7 to -6.37
|
-6.49 units on a scale
Interval -9.15 to -3.83
|
SECONDARY outcome
Timeframe: week 12Population: 12 participants were randomized but withdrew before receiving any study drug (4 placebo, 2 CVT-301 low-dose, 6 CVT-301 high-dose).
Patient Global impression of change at treatment visit 4 (week 12) by improvement category. Seven point Likert scale ranging from 1= much worse to 7= much better.
Outcome measures
| Measure |
CVT-301 High Dose
n=113 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=114 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
Placebo
n=112 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
Proportion of Subjects Who Improved PGIC With CVT-301 vs. Placebo at Week 12
|
61 Participants
|
70 Participants
|
45 Participants
|
SECONDARY outcome
Timeframe: week 12Population: 12 participants were randomized but withdrew before receiving any study drug (4 placebo, 2 CVT-301 low-dose, 6 CVT-301 high-dose).
Change from Predose in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III Score at 10 Minutes post-dose at Week 12 for CVT-301 High Dose versus Placebo (ITT Population). UPDRS Part III ranges from 0 minimum to 86 maximum, with lower scores indicating better movement. Units on a scale.
Outcome measures
| Measure |
CVT-301 High Dose
n=113 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=114 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
Placebo
n=112 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
UPDRS Part III at 10 Min.
|
-5.16 units on a scale
Interval -7.31 to -3.0
|
-6.45 units on a scale
Interval -8.62 to -4.27
|
-4.18 units on a scale
Interval -6.35 to -2.01
|
SECONDARY outcome
Timeframe: post week 12Population: 12 participants were randomized but withdrew before receiving any study drug (4 placebo, 2 CVT-301 low-dose, 6 CVT-301 high-dose).
Change in total daily OFF times for 3 consecutive days prior to week 12 visit compared to 3 consecutive days prior to baseline visit. Participants recorded On or Off state in 30 minute intervals during waking hours.
Outcome measures
| Measure |
CVT-301 High Dose
n=113 Participants
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=114 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
Placebo
n=112 Participants
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
PD Patient Diary
|
-0.58 hours
Interval -1.13 to -0.03
|
-0.47 hours
Interval -1.02 to 0.09
|
-0.48 hours
Interval -1.03 to 0.08
|
Adverse Events
CVT-301 Low Dose
CVT-301 High Dose
Placebo
Serious adverse events
| Measure |
CVT-301 Low Dose
n=113 participants at risk
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
CVT-301 High Dose
n=114 participants at risk
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=112 participants at risk
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
Cardiac disorders
Pericarditis
|
0.88%
1/113 • 12 Week Study
|
0.00%
0/114 • 12 Week Study
|
0.00%
0/112 • 12 Week Study
|
|
Cardiac disorders
Atrial fibrillation
|
0.88%
1/113 • 12 Week Study
|
0.88%
1/114 • 12 Week Study
|
0.89%
1/112 • 12 Week Study
|
|
Cardiac disorders
Bradycardia
|
0.88%
1/113 • 12 Week Study
|
0.00%
0/114 • 12 Week Study
|
0.00%
0/112 • 12 Week Study
|
|
General disorders
Chest pain
|
1.8%
2/113 • 12 Week Study
|
0.00%
0/114 • 12 Week Study
|
0.00%
0/112 • 12 Week Study
|
|
Nervous system disorders
Syncope
|
0.88%
1/113 • 12 Week Study
|
0.00%
0/114 • 12 Week Study
|
0.00%
0/112 • 12 Week Study
|
|
Cardiac disorders
Coronary artery disease
|
0.00%
0/113 • 12 Week Study
|
0.00%
0/114 • 12 Week Study
|
0.89%
1/112 • 12 Week Study
|
|
Gastrointestinal disorders
Pancreatitis acute
|
0.00%
0/113 • 12 Week Study
|
0.00%
0/114 • 12 Week Study
|
0.89%
1/112 • 12 Week Study
|
|
Musculoskeletal and connective tissue disorders
Lumbar spinal stinosis
|
0.00%
0/113 • 12 Week Study
|
0.88%
1/114 • 12 Week Study
|
0.89%
1/112 • 12 Week Study
|
Other adverse events
| Measure |
CVT-301 Low Dose
n=113 participants at risk
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
CVT-301 High Dose
n=114 participants at risk
Capsules of levodopa inhalational powder used up to 5 times/day for OFF episodes for 3 months duration
CVT-301
|
Placebo
n=112 participants at risk
Capsules of inhalation-grade lactose used up to 5 times/day for OFF episodes for 3 months duration.
CVT-301
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.0%
17/113 • 12 Week Study
|
14.9%
17/114 • 12 Week Study
|
1.8%
2/112 • 12 Week Study
|
|
Respiratory, thoracic and mediastinal disorders
Throat Irritation
|
7.1%
8/113 • 12 Week Study
|
0.88%
1/114 • 12 Week Study
|
0.00%
0/112 • 12 Week Study
|
|
Respiratory, thoracic and mediastinal disorders
Sputum discoloured
|
0.00%
0/113 • 12 Week Study
|
5.3%
6/114 • 12 Week Study
|
0.00%
0/112 • 12 Week Study
|
|
Infections and infestations
Upper respiratory tract
|
1.8%
2/113 • 12 Week Study
|
6.1%
7/114 • 12 Week Study
|
2.7%
3/112 • 12 Week Study
|
|
Nervous system disorders
Dyskinesia
|
4.4%
5/113 • 12 Week Study
|
3.5%
4/114 • 12 Week Study
|
0.00%
0/112 • 12 Week Study
|
|
Nervous system disorders
Dizziness
|
1.8%
2/113 • 12 Week Study
|
0.88%
1/114 • 12 Week Study
|
4.5%
5/112 • 12 Week Study
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/113 • 12 Week Study
|
5.3%
6/114 • 12 Week Study
|
2.7%
3/112 • 12 Week Study
|
|
Injury, poisoning and procedural complications
Fall
|
4.4%
5/113 • 12 Week Study
|
2.6%
3/114 • 12 Week Study
|
1.8%
2/112 • 12 Week Study
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding the study results for a period up to 30 days from the date the communication is submitted to the sponsor. The sponsor shall have the right to defer proposed publication an additional 60 days from the end of the review period. The sponsor cannot require changes to the communication and cannot unilaterally extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER