Trial Outcomes & Findings for Randomized Safety Study of CVT-301 Compared to an Observational Control Group (NCT NCT02352363)
NCT ID: NCT02352363
Last Updated: 2019-05-28
Results Overview
To characterize the pulmonary safety, as assessed by spirometry (forced expiratory volume in 1 second \[FEV1\], over a 12 month period.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
408 participants
Primary outcome timeframe
Month 12 reported
Results posted on
2019-05-28
Participant Flow
A total of 408 of the 513 screened were randomly assigned to CVT-301 (278) or observational cohort (130). Of these 408 patients, 398 received at least 1 dose of inhaled CVT-301 CVT-treatment group) or came in for OV1 (observational cohort) and were included in the Safety Population.
Participant milestones
| Measure |
CVT-301
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
|---|---|---|
|
Overall Study
STARTED
|
278
|
130
|
|
Overall Study
COMPLETED
|
271
|
127
|
|
Overall Study
NOT COMPLETED
|
7
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Randomized Safety Study of CVT-301 Compared to an Observational Control Group
Baseline characteristics by cohort
| Measure |
CVT-301
n=271 Participants
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
n=127 Participants
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
Total
n=398 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Region of Enrollment
France
|
13 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
137 Participants
n=5 Participants
|
59 Participants
n=7 Participants
|
196 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
134 Participants
n=5 Participants
|
68 Participants
n=7 Participants
|
202 Participants
n=5 Participants
|
|
Age, Continuous
|
63.6 years
STANDARD_DEVIATION 8.54 • n=5 Participants
|
64.2 years
STANDARD_DEVIATION 7.88 • n=7 Participants
|
63.8 years
STANDARD_DEVIATION 8.33 • n=5 Participants
|
|
Sex: Female, Male
Female
|
110 Participants
n=5 Participants
|
49 Participants
n=7 Participants
|
159 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
161 Participants
n=5 Participants
|
78 Participants
n=7 Participants
|
239 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
267 Participants
n=5 Participants
|
124 Participants
n=7 Participants
|
391 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
Romania
|
55 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
89 Participants
n=5 Participants
|
|
Region of Enrollment
Hungary
|
5 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
Czechia
|
22 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
21 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
28 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Region of Enrollment
Austria
|
11 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
61 Participants
n=5 Participants
|
32 Participants
n=7 Participants
|
93 Participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
15 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Region of Enrollment
Serbia
|
11 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
21 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Spirometry FEV1
|
2.842 Liters
STANDARD_DEVIATION 0.7538 • n=5 Participants
|
2.838 Liters
STANDARD_DEVIATION 0.853 • n=7 Participants
|
2.841 Liters
STANDARD_DEVIATION 0.7831 • n=5 Participants
|
|
Spirometry FVC
|
3.665 Liters
STANDARD_DEVIATION 0.9203 • n=5 Participants
|
3.696 Liters
STANDARD_DEVIATION 1.0497 • n=7 Participants
|
3.675 Liters
STANDARD_DEVIATION 0.9622 • n=5 Participants
|
|
Spirometry FEV1/FVC
|
77.6 Ratio %
STANDARD_DEVIATION 6.23 • n=5 Participants
|
76.8 Ratio %
STANDARD_DEVIATION 6.50 • n=7 Participants
|
77.3 Ratio %
STANDARD_DEVIATION 6.32 • n=5 Participants
|
PRIMARY outcome
Timeframe: Month 12 reportedTo characterize the pulmonary safety, as assessed by spirometry (forced expiratory volume in 1 second \[FEV1\], over a 12 month period.
Outcome measures
| Measure |
CVT-301
n=271 Participants
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
n=127 Participants
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
|---|---|---|
|
Pulmonary Safety Assessed by Forced Expiratory Volume in 1 Second [FEV1]
|
2.778 Liters
Standard Deviation 0.7309
|
2.767 Liters
Standard Deviation 0.8419
|
PRIMARY outcome
Timeframe: Month 12 reportedTo characterize the pulmonary safety, as assessed by spirometry (forced vital capacity).
Outcome measures
| Measure |
CVT-301
n=271 Participants
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
n=127 Participants
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
|---|---|---|
|
Pulmonary Safety Assessed by Forced Vital Capacity [FVC].
|
3.558 Liters
Standard Deviation 0.8987
|
3.642 Liters
Standard Deviation 1.0687
|
PRIMARY outcome
Timeframe: Month 12 reportedTo characterize the pulmonary safety, as assessed by spirometry (forced expiratory volume in 1 second / forced vital capacity ratio).
Outcome measures
| Measure |
CVT-301
n=271 Participants
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
n=127 Participants
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
|---|---|---|
|
Pulmonary Safety Assessed by Forced Expiratory Volume in 1 Second / Forced Vital Capacity Ratio.
|
78.1 Ratio (%)
Standard Deviation 6.04
|
76.1 Ratio (%)
Standard Deviation 6.4
|
SECONDARY outcome
Timeframe: Month 12 reportedTo describe the effects of CVT-301 on diffusion capacity of the lungs for carbon monoxide (DLco) over a 12-month period.
Outcome measures
| Measure |
CVT-301
n=271 Participants
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
n=127 Participants
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
|---|---|---|
|
Diffusion Capacity of the Lungs for Carbon Monoxide (DLco).
|
23.273 mL/min/mmHg
Standard Deviation 6.0025
|
23.473 mL/min/mmHg
Standard Deviation 7.0848
|
Adverse Events
CVT-301
Serious events: 42 serious events
Other events: 192 other events
Deaths: 1 deaths
Observational Cohort
Serious events: 13 serious events
Other events: 64 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CVT-301
n=271 participants at risk
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
n=127 participants at risk
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
|---|---|---|
|
Injury, poisoning and procedural complications
Femoral neck fracture
|
0.74%
2/271 • Number of events 2 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Nervous system disorders
Parkinson's disease
|
0.74%
2/271 • Number of events 2 • 12 Months
|
1.6%
2/127 • Number of events 2 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
1.1%
3/271 • Number of events 3 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/271 • 12 Months
|
1.6%
2/127 • Number of events 2 • 12 Months
|
|
Gastrointestinal disorders
Inguinal hernia
|
0.74%
2/271 • Number of events 2 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Metabolism and nutrition disorders
Dehydration
|
0.74%
2/271 • Number of events 2 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Blood and lymphatic system disorders
Anemia
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.74%
2/271 • Number of events 2 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc protrusion
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Injury, poisoning and procedural complications
Abdominal wound dehiscence
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Injury, poisoning and procedural complications
Hand fracture
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Injury, poisoning and procedural complications
Hip fracture
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Injury, poisoning and procedural complications
Joint dislocation
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Injury, poisoning and procedural complications
Post procedure haemorrhage
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Injury, poisoning and procedural complications
Thoracic vertebral fracture
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Nervous system disorders
Cerebrovascular accident
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Nervous system disorders
Ischaemic stroke
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Nervous system disorders
Occipital neuralgia
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Nervous system disorders
Syncope
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Infections and infestations
Urinary tract infection
|
1.5%
4/271 • Number of events 4 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Infections and infestations
Bronchitis
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Infections and infestations
Cellulitis
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Infections and infestations
Escherichia urinary tract
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Infections and infestations
Pneumonia
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Infections and infestations
Postoperative wound
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Infections and infestations
Respiratory tract infection
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Infections and infestations
Sepsis
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Lumbar spinal stenosis
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Spondylitis
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Vertebral foraminal stenosis
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Gastrointestinal disorders
Gastritis
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Gastrointestinal disorders
Pancreatitis acute
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
General disorders
Astenia
|
0.00%
0/271 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Hepatobiliary disorders
Bile duct stone
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Hepatobiliary disorders
Cholecystitis
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Benign ovarian tumor
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to central nervous system
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostatic adenoma
|
0.37%
1/271 • Number of events 1 • 12 Months
|
0.00%
0/127 • 12 Months
|
Other adverse events
| Measure |
CVT-301
n=271 participants at risk
Capsules of Levodopa Inhalational Powder (LIP) used up to 5 times per day for OFF episodes, for up to 54 weeks duration.
CVT-301
|
Observational Cohort
n=127 participants at risk
Standard of care. Patients in both the CVT-301 treatment group and the observational cohort will be managed with their standard PD treatment throughout the study.
Observational cohort
|
|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
6.6%
18/271 • Number of events 20 • 12 Months
|
5.5%
7/127 • Number of events 8 • 12 Months
|
|
Infections and infestations
Upper respiratory tract infection
|
4.8%
13/271 • Number of events 16 • 12 Months
|
2.4%
3/127 • Number of events 3 • 12 Months
|
|
Infections and infestations
Bronchitis
|
2.6%
7/271 • Number of events 7 • 12 Months
|
3.1%
4/127 • Number of events 6 • 12 Months
|
|
Infections and infestations
Influenza
|
1.5%
4/271 • Number of events 5 • 12 Months
|
3.1%
4/127 • Number of events 4 • 12 Months
|
|
Nervous system disorders
Dyskinesia
|
6.3%
17/271 • Number of events 18 • 12 Months
|
3.9%
5/127 • Number of events 5 • 12 Months
|
|
Nervous system disorders
Parkinson's disease
|
3.0%
8/271 • Number of events 8 • 12 Months
|
3.1%
4/127 • Number of events 4 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
4.4%
12/271 • Number of events 19 • 12 Months
|
3.1%
4/127 • Number of events 4 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
3.0%
8/271 • Number of events 8 • 12 Months
|
2.4%
3/127 • Number of events 3 • 12 Months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
1.1%
3/271 • Number of events 3 • 12 Months
|
3.1%
4/127 • Number of events 4 • 12 Months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.3%
36/271 • Number of events 43 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Respiratory, thoracic and mediastinal disorders
Sputum discoloured
|
3.3%
9/271 • Number of events 11 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
3.3%
9/271 • Number of events 10 • 12 Months
|
0.00%
0/127 • 12 Months
|
|
Psychiatric disorders
Insomnia
|
1.1%
3/271 • Number of events 3 • 12 Months
|
3.9%
5/127 • Number of events 5 • 12 Months
|
|
Injury, poisoning and procedural complications
Fall
|
8.1%
22/271 • Number of events 28 • 12 Months
|
5.5%
7/127 • Number of events 8 • 12 Months
|
|
Gastrointestinal disorders
Nausea
|
3.7%
10/271 • Number of events 12 • 12 Months
|
0.79%
1/127 • Number of events 1 • 12 Months
|
|
Vascular disorders
Hypertension
|
3.3%
9/271 • Number of events 9 • 12 Months
|
3.1%
4/127 • Number of events 4 • 12 Months
|
|
Vascular disorders
Orthostatic hypotension
|
2.6%
7/271 • Number of events 12 • 12 Months
|
3.1%
4/127 • Number of events 4 • 12 Months
|
|
Investigations
Blood pressure increased
|
0.37%
1/271 • Number of events 1 • 12 Months
|
3.1%
4/127 • Number of events 5 • 12 Months
|
|
Ear and labyrinth disorders
Vertigo
|
3.0%
8/271 • Number of events 10 • 12 Months
|
0.00%
0/127 • 12 Months
|
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