Clinical trial NCT02352363: Randomized Safety Study of CVT-301 Compared to an Observational Control Group
Official title: A Phase 3, Randomized Study Investigating the Safety of CVT-301 (Levodopa Inhalation Powder) in Parkinson's Disease (PD) Patients With Motor Response Fluctuations (OFF Phenomena) Compared to an Observational Cohort Control
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Idiopathic Parkinson's Disease
Keywords: Parkinson's Disease
Motor fluctuations
levodopa
inhaled drugs
OFF episodes
Lead sponsor: Acorda Therapeutics
Sponsor class: INDUSTRY
First posted: 2015-02-02
Enrollment: 408
Sex: ALL
Minimum age (years): 30
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is a 12-month, open-label, randomized, multicenter study which will evaluate the safety and efficacy of CVT-301 for the treatment of up to 5 OFF episodes per day in Parkinson's Disease (PD) patients experiencing motor fluctuations (OFF episodes) and will include a concurrent observational cohort of PD patients managed using the usual standards of care.

Detailed description: A randomized, double-blind, placebo-controlled, Phase 2b study in patients with PD (CVT-301-003) demonstrated clinically important, and statistically significant CVT-301-associated improvements in motor function at doses of 35 and 50 mg LD FPD, compared with placebo, as measured by the Unified Parkinson's Disease Rating Scale (UPDRS) motor assessment (Part 3); furthermore, CVT-301 was generally safe and well tolerated. A Phase 3, randomized, placebo-controlled study designed to assess the efficacy and safety of 35 mg and 50 mg FPD as an adjunct to a CD/LD regimen in the treatment of OFF symptoms over 12 weeks (CVT-301-004) was conducted in parallel with this study.

Eligibility criteria: Inclusion Criteria:

* Has signed and dated an Internal Review Board/Independent Ethics Committee (IRB/IEC)-approved informed consent form before any protocol-specific screening procedures are performed.
* Women of child-bearing potential must use protocol-defined contraceptive measures and must have a negative serum human chorionic gonadotropin (hCG) test at screening. These patients must be willing to remain on their current form of contraception for the duration of the study.
* Patients who have idiopathic PD (i.e., not induced by drugs or other diseases) as defined by fulfilling Steps 1 and 2 of the United Kingdom (UK) Brain Bank criteria, diagnosed after the age of 30 years.
* Patients who are classified as Stage 1 to 3 (in the ON state) on the modified Hoehn and Yahr scale for staging of PD severity.
* Patients who have experienced motor fluctuations for a minimum of 2 hours of average daily OFF time per waking day (excluding early morning OFF time) by self-report and confirmed by the PD Diary (on 3 consecutive days) during the screening period.

Exclusion Criteria:

* Patients who have dyskinesia of a severity that would significantly interfere with their ability to participate or perform study procedures.
* Pregnant or lactating females or females wishing to become pregnant.
* Patients who have any known contraindication to the use of levodopa (LD), including a history of malignant melanoma or a history of narrow-angle glaucoma.
* Patients who have had previous surgery for PD (including but not limited to deep brain stimulation \[DBS\] or cell transplantation).
* Patients with a history of psychotic symptoms requiring treatment, or suicide ideation or attempt within the prior 12 months.

Primary outcomes:
measure: Pulmonary Safety Assessed by Forced Expiratory Volume in 1 Second [FEV1]
timeFrame: Month 12 reported
measure: Pulmonary Safety Assessed by Forced Vital Capacity [FVC].
timeFrame: Month 12 reported
measure: Pulmonary Safety Assessed by Forced Expiratory Volume in 1 Second / Forced Vital Capacity Ratio.
timeFrame: Month 12 reported
Secondary outcomes:
measure: Diffusion Capacity of the Lungs for Carbon Monoxide (DLco).
timeFrame: Month 12 reported
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Acorda Site #7145
state: Arizona
city: Fountain Valley
country: United States
facility: Acorda Site # 7142
state: California
city: Panorama City
country: United States
facility: Acorda Site #7139
state: California
city: Sunnyvale
country: United States
facility: Acorda Site #7141
state: California
city: Ormond Beach
country: United States
facility: Acorda Site #7137
state: Florida
city: Saint Petersburg
country: United States
facility: Acorda Site #7135
state: Florida
city: Sunrise
country: United States
facility: Acorda Site #7130
state: Florida
city: Atlanta
country: United States
facility: Acorda Site #7133
state: Georgia
city: Atlanta
country: United States
facility: Acorda Site #7134
state: Georgia
city: Decatur
country: United States
facility: Acorda Site #7131
state: Georgia
city: Honolulu
country: United States
facility: Acorda Site #7138
state: Hawaii
city: Des Moines
country: United States
facility: Acorda Site #7140
state: Iowa
city: Baton Rouge
country: United States
facility: Acorda Site #7150
state: Louisiana
city: Toledo
country: United States
facility: Acorda Site #7148
state: Ohio
city: Innsbruck
country: Austria
facility: Acorda Site #7004
city: Linz
country: Austria
facility: Acorda Site #7002
city: Vienna
country: Austria
facility: Acorda Site #7003
city: Brussels
country: Belgium
facility: Acorda Site #7011
city: Edegem
country: Belgium
facility: Acorda Site #7012
city: Ghent
country: Belgium
facility: Acorda Site #7013
city: Chocen
country: Czechia
facility: Acorda Site #7024
city: Ostrava
country: Czechia
facility: Acorda Site #7025
city: Pardubice
country: Czechia
facility: Acorda Site #7021
city: Prague 10
country: Czechia
facility: Acorda Site #7022
city: Rychnov nad Kneznou
country: Czechia
facility: Acorda Site #7023
city: Amiens
country: France
facility: Acorda Site #7036
city: Bordeaux
country: France
facility: Acorda Site #7037
city: Marseille Cedex 05
country: France
facility: Acorda Site #7034
city: MONTPELLIER Cedex 5
country: France
facility: Acorda Site #7031
city: Nimes Cedex
country: France
facility: Acorda Site #7033
city: Strasbourg
country: France
facility: Acorda Site #7032
city: Toulouse Cedex 9
country: France
facility: Acorda Site #7035
city: Achim
country: Germany
facility: Acorda Site #7041
city: Beelitz-Heilstätten
country: Germany
facility: Acorda Site #7043
city: Berlin
country: Germany
facility: Acorda Site #7049
city: Bochum
country: Germany
facility: Acorda Site #7050
city: Bremerhaven
country: Germany
facility: Acorda Site #7048
city: Cologne
country: Germany
facility: Acorda Site #7047
city: Marburg
country: Germany
facility: Acorda Site #7046
city: Munich
country: Germany
facility: Acorda Site #7042
city: Ulm
country: Germany
facility: Acorda Site #7044
city: Budapest
country: Hungary
facility: Acorda Site #7053
city: Budapest
country: Hungary
facility: Acorda Site #7051
city: Jerusalem
country: Israel
facility: Acorda Site #7062
city: Petah Tikva
country: Israel
facility: Acorda Site #7064
city: Ramat Gan
country: Israel
facility: Acorda Site #7061
city: Tel-Aviv
country: Israel
facility: Acorda Site #7063
city: Den Haag
country: Netherlands
facility: Acorda Site #7045
city: Gdansk
country: Poland
facility: Acorda Site #7085
city: Katowice
country: Poland
facility: Acorda Site #7084
city: Kracow
country: Poland
facility: Acorda Site #7083
city: Kraków
country: Poland
facility: Acorda Site #7086
city: Kraków
country: Poland
facility: Acorda Site #7087
city: Lodz
country: Poland
facility: Acorda Site #7082
city: Warsaw
country: Poland
facility: Acorda Site #7081
city: Warsaw
country: Poland
facility: Acorda Site #7088
city: Brasov
country: Romania
facility: Acorda Site #7094
city: Brasov
country: Romania
facility: Acorda Site #7092
city: Bucharest
country: Romania
facility: Acorda Site #7093
city: Constanta
country: Romania
facility: Acorda Site #7091
city: Targu Mures
country: Romania
facility: Acorda Site #7095
city: Belgrade
country: Serbia
facility: Acorda Site #7101
city: Belgrade
country: Serbia
facility: Acorda Site #7102
city: Kragujevac
country: Serbia
facility: Acorda Site #7103
city: Sant Cugat
country: Spain
facility: Acorda Site #7112
state: Barcelona
city: Barcelona
country: Spain
facility: Acorda Site #7116
city: Barcelona
country: Spain
facility: Acorda Site #7111
city: Barcelona
country: Spain
facility: Acorda Site #7120
city: Barcelona
country: Spain
facility: Acorda Site #7119
city: Burgos
country: Spain
facility: Acorda Site #7113
city: Madrid
country: Spain
facility: Acorda Site #7118
city: Madrid
country: Spain
facility: Acorda Site #7114
city: San Sebastian
country: Spain
facility: Acorda Site #7115
city: Cambridge
country: United Kingdom
facility: Acorda Site #7123
city: Glasgow
country: United Kingdom
facility: Acorda Site #7121
city: London
country: United Kingdom
facility: Acorda Site #7122
Study officials:
affiliation: Acorda Therapeutics
name: Charles Oh, MD
role: STUDY_DIRECTOR
References:
citation: Kaminsky DA, Grosset DG, Kegler-Ebo DM, Cangiamilla S, Klingler M, Zhao P, Oh C. Natural history of lung function over one year in patients with Parkinson's disease. Respir Med. 2021 Jun;182:106396. doi: 10.1016/j.rmed.2021.106396. Epub 2021 Apr 16.