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Outpatient Physical Therapy Intervention in Subjects With Parkinson's Disease Currently Using APOKYN® (PERFORM)

Primary Purpose

Parkinson's Disease, Motor Symptoms

Status
Terminated
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
APOKYN
Physical Therapy
Sponsored by
MDD US Operations, LLC a subsidiary of Supernus Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Parkinson's Disease focused on measuring Motor Symptoms, Parkinson's disease, Apokyn, Apomorphine, PD

Eligibility Criteria

18 Years - 78 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Able to understand and sign an Investigational Review Board (IRB)-approved written informed consent and privacy language (e.g., Health Insurance Portability and Accountability Act (HIPAA) authorization) as per national regulations before any study-related procedures are performed.
  • Must have a diagnosis of idiopathic PD.
  • Adult male or female 18 to 78 years of age, inclusive.
  • Currently using APOKYN, per the Package Insert, for at least 4 weeks before Baseline Assessment Visit(s).
  • Currently using carbidopa/levodopa therapy at a steady maintenance dose, representing a stable treatment regimen in the opinion of the Investigator, for at least 4 weeks before Baseline Assessment Visit(s).
  • Able to walk 50 feet independently (cane permissible) at the Baseline Assessment Visit.
  • Meet all of the following parameters to demonstrate an optimal response to APOKYN at the APOKYN Response Verification Visit: must be "on" in the opinion of the subject and investigator within 20 minutes after the APOKYN injection; and have a motor state improvement of >25% per the Movement Disorder Society (MDS)-UPDRS Motor Score (Part III), within 20 minutes after the APOKYN injection, as compared to pre-injection.
  • Willing and able to comply with scheduled visits, treatment plan, other study-related procedures, and have available caregiver to provide transportation to clinic visits as needed.

Exclusion Criteria:

  • Received PT targeting Parkinson's disease within 6 months before Baseline Assessment Visit(s).
  • Received any investigational (i.e., unapproved) drug product within the last 30 days (or 5 half-lives, whichever is longer) before the Baseline Assessment Visit(s).
  • Currently taking, or likely to need to take at any time during the course of the study, any 5HT3 antagonist (i.e., ondansetron, granisetron, dolasetron, palonosetron, alosetron).
  • Currently on or likely to transition to the DUOPA (carbidopa and levodopa enteral suspension) during the course of the study.
  • Has orthostatic hypotension (defined as >30 mmHg decrease in systolic blood pressure or >15 mmHg decrease in diastolic blood pressure within 2 minutes of standing from a supine position) at the Baseline Assessment Visit(s), or within 20 minutes following APOKYN injection at the APOKYN Verification Visit.
  • Has a score of 24 or higher on the Modified Physical Performance Test (M-PPT).
  • Has a score of 80% or higher on the Activities-specific Balance Confidence Scale (ABC).
  • Has any significant current medical disorder, physical, or mental condition that would, in the Investigator's opinion, represent a hazard to the subject or prevent the subject from completing the study.
  • Subjects with dementia, in the Investigator's opinion, or subjects with a Montreal Cognitive Assessment (MoCA) <18.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Other

Arm Label

Apokyn treatment before physical therapy

Apokyn treatment withheld before physical therapy

Arm Description

APOKYN treatment before the PT Intervention Visit. Subjects in the "APO+" group will administer their usual dose of APOKYN at the PT clinic, as instructed by the Physical Therapist. These subjects will not take carbidopa/levodopa for at least 3 hours before or during the PT Intervention Visit.

APOKYN treatment withheld before the PT Intervention Visit. Subjects in the "APO-" group will not take carbidopa/levodopa and APOKYN for at least 3 hours before or during the PT Intervention Visit. No "rescue therapy" will be allowed during the PT. Intervention Visit. If the PT Intervention Visit needs to be stopped early, the Physical Therapist will record this along with a reason for the discontinuation.

Outcomes

Primary Outcome Measures

Change From Baseline in Activities-specific Balance Confidence (ABC) Scale
Scale from 0 to 100% where an increase in percent represents an improvement in balance confidence.

Secondary Outcome Measures

Change From Baseline in Movement Disorder Societies Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III (Motor Examination)
MDS-UPDRS Part III is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 72 (worst score possible). A negative change from baseline represents an improvement in motor function.
Change From Baseline in Modified Physical Performance Test (M-PPT)
Investigator scale assessing 9 domains of physical functioning where total scores range from 0 to 28 with higher scores indicating better physical performance. A positive change from baseline represents an improvement in physical performance.
Change From Baseline in Timed-Up-and-Go (TUG) Test
Test captures the amount of time for a subject to get up from an arm chair, walk 3 meters, turn around, walk back to chair, and sit. A negative change from baseline represents an improvement of physical function.
Change From Baseline in 6-Minute Walk Test (6MWT)
Objective performance based measure of functional exercise capacity that measures the distance a subject can cover on a 30 meter flat walking area over 6 minutes. A positive change from baseline represents an improvement in the distance walked (ft) in 6 minutes.
Change From Baseline in Montreal Cognitive Assessment (MoCA)
The MoCA is a validated, 30-point test (sum of the 6 domains), with scores ranging from 0 (worst) to 30 (best). The test is designed to assess several cognitive domains, including visuospatial abilities (5 points), animal naming (3 points), attention (6 points), language (3 points), abstraction (2 points), delayed recall (5 points), and orientation to time and place (6 points). Total MoCA scores higher than 26 indicate normal cognitive function. A positive change from baseline represents an improvement in cognitive function.
Change From Baseline in MDS-UPDRS Part I A
MDS-UPDRS Part I A is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 24 (worst score possible). A negative change from baseline represents an improvement in non-motor experiences of daily living (investigator rated).
Number of Participants in Each Category of Clinical Global Impression of Change (CGI-C)
Investigator's impression of change in the subject's symptoms from baseline assessment visit to end of study assessment, on a 7-point scale ranging from very much improved to very much worse.
Change From Baseline in MDS-UPDRS Part II
MDS-UPDRS Part II is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 52 (worst score possible). A negative change from baseline represents an improvement in motor experience of daily living.
Change From Baseline in MDS-UPDRS Part IV
MDS-UPDRS Part IV is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 24 (worst score possible). A negative change from baseline represents fewer motor complications.
Change From Baseline in MDS-UPDRS Part I B
MDS-UPDRS Part I B is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 28 (worst score possible). A negative change from baseline represents an improvement in non-motor experiences of daily living (subject rated).
Change From Baseline in Clinical Global Impression of Severity (CGI-S)
Investigator's impression of the severity of the subject's illness on a 7-point scale ranging from normal (1) to among the most extremely ill subjects (7). A negative change from baseline represents a decrease in the severity of the subject's illness.

Full Information

First Posted
September 9, 2015
Last Updated
August 8, 2023
Sponsor
MDD US Operations, LLC a subsidiary of Supernus Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02549573
Brief Title
Outpatient Physical Therapy Intervention in Subjects With Parkinson's Disease Currently Using APOKYN®
Acronym
PERFORM
Official Title
Outpatient Physical Therapy Intervention in Subjects With Parkinson's DiseaseCurrently Using APOKYN®: A Phase IV Study of Outcome Assessments of Physical Therapy in Subjects in an "On" Versus "End-of-Dose-Off" Motor State
Study Type
Interventional

2. Study Status

Record Verification Date
December 2017
Overall Recruitment Status
Terminated
Why Stopped
Subject enrollment is unsatisfactory
Study Start Date
January 2016 (Actual)
Primary Completion Date
December 2016 (Actual)
Study Completion Date
December 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MDD US Operations, LLC a subsidiary of Supernus Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
To determine if PT intervention will be improved while in the "on" motor state vs. the "end-of-dose-off" motor state during the PT Intervention Visit in subjects with PD.
Detailed Description
Multicenter, outpatient trial to evaluate PT outcomes in subjects diagnosed with PD and currently using APOKYN. Those subjects who satisfy all eligibility criteria will be randomized to 2 treatment groups: "APO+" (APOKYN treatment before the PT Intervention Visit) and; "APO-" (APOKYN treatment withheld before the PT Intervention Visit). The study will have: Baseline Assessment Visit(s) - 1 day at Investigator's clinic and PT clinic APOKYN Response Verification Visit - 1 day PT Intervention Visits - for 6 weeks End-of-study Assessment Visit(s) - 1 day at Investigator's clinic and PT clinic All subjects will participate in a standardized PT intervention. In both treatment groups, ALL subjects will arrive for the PT Intervention Visit in an "end-of-dose-off" motor state. The "end-of-dose-off" motor state will be achieved by the subject withholding his/her carbidopa/levodopa dose and APOKYN dose for at least 3 hours before the PT Intervention Visit. It is hypothesized that the outcome of PT will be improved when subjects are in the "on" motor state during PT Intervention visits compared with being in the "end-of-dose-off" motor state during PT Intervention visits.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease, Motor Symptoms
Keywords
Motor Symptoms, Parkinson's disease, Apokyn, Apomorphine, PD

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
13 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Apokyn treatment before physical therapy
Arm Type
Active Comparator
Arm Description
APOKYN treatment before the PT Intervention Visit. Subjects in the "APO+" group will administer their usual dose of APOKYN at the PT clinic, as instructed by the Physical Therapist. These subjects will not take carbidopa/levodopa for at least 3 hours before or during the PT Intervention Visit.
Arm Title
Apokyn treatment withheld before physical therapy
Arm Type
Other
Arm Description
APOKYN treatment withheld before the PT Intervention Visit. Subjects in the "APO-" group will not take carbidopa/levodopa and APOKYN for at least 3 hours before or during the PT Intervention Visit. No "rescue therapy" will be allowed during the PT. Intervention Visit. If the PT Intervention Visit needs to be stopped early, the Physical Therapist will record this along with a reason for the discontinuation.
Intervention Type
Drug
Intervention Name(s)
APOKYN
Other Intervention Name(s)
Apomorphine, APO
Intervention Description
Subjects in the APOKYN+ group will administer his/her usual dose of APOKYN at the PT clinic, as instructed by the Physical Therapist. These subjects will not take carbidopa/levodopa for at least 3 hours before or during the PT Intervention Visit.
Intervention Type
Behavioral
Intervention Name(s)
Physical Therapy
Other Intervention Name(s)
PT
Intervention Description
All subjects will participate in a standardized PT intervention
Primary Outcome Measure Information:
Title
Change From Baseline in Activities-specific Balance Confidence (ABC) Scale
Description
Scale from 0 to 100% where an increase in percent represents an improvement in balance confidence.
Time Frame
Baseline and after week 6
Secondary Outcome Measure Information:
Title
Change From Baseline in Movement Disorder Societies Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III (Motor Examination)
Description
MDS-UPDRS Part III is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 72 (worst score possible). A negative change from baseline represents an improvement in motor function.
Time Frame
Baseline and after week 6
Title
Change From Baseline in Modified Physical Performance Test (M-PPT)
Description
Investigator scale assessing 9 domains of physical functioning where total scores range from 0 to 28 with higher scores indicating better physical performance. A positive change from baseline represents an improvement in physical performance.
Time Frame
Baseline and after week 6
Title
Change From Baseline in Timed-Up-and-Go (TUG) Test
Description
Test captures the amount of time for a subject to get up from an arm chair, walk 3 meters, turn around, walk back to chair, and sit. A negative change from baseline represents an improvement of physical function.
Time Frame
Baseline and after week 6
Title
Change From Baseline in 6-Minute Walk Test (6MWT)
Description
Objective performance based measure of functional exercise capacity that measures the distance a subject can cover on a 30 meter flat walking area over 6 minutes. A positive change from baseline represents an improvement in the distance walked (ft) in 6 minutes.
Time Frame
Baseline and after week 6
Title
Change From Baseline in Montreal Cognitive Assessment (MoCA)
Description
The MoCA is a validated, 30-point test (sum of the 6 domains), with scores ranging from 0 (worst) to 30 (best). The test is designed to assess several cognitive domains, including visuospatial abilities (5 points), animal naming (3 points), attention (6 points), language (3 points), abstraction (2 points), delayed recall (5 points), and orientation to time and place (6 points). Total MoCA scores higher than 26 indicate normal cognitive function. A positive change from baseline represents an improvement in cognitive function.
Time Frame
Baseline and after week 6
Title
Change From Baseline in MDS-UPDRS Part I A
Description
MDS-UPDRS Part I A is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 24 (worst score possible). A negative change from baseline represents an improvement in non-motor experiences of daily living (investigator rated).
Time Frame
Baseline and after week 6
Title
Number of Participants in Each Category of Clinical Global Impression of Change (CGI-C)
Description
Investigator's impression of change in the subject's symptoms from baseline assessment visit to end of study assessment, on a 7-point scale ranging from very much improved to very much worse.
Time Frame
After week 6
Title
Change From Baseline in MDS-UPDRS Part II
Description
MDS-UPDRS Part II is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 52 (worst score possible). A negative change from baseline represents an improvement in motor experience of daily living.
Time Frame
Baseline and after week 6
Title
Change From Baseline in MDS-UPDRS Part IV
Description
MDS-UPDRS Part IV is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 24 (worst score possible). A negative change from baseline represents fewer motor complications.
Time Frame
Baseline and after week 6
Title
Change From Baseline in MDS-UPDRS Part I B
Description
MDS-UPDRS Part I B is a sub-scale of MDS-UPDRS. Sub-scale scores range from 0 (best score possible) to 28 (worst score possible). A negative change from baseline represents an improvement in non-motor experiences of daily living (subject rated).
Time Frame
Baseline and after week 6
Title
Change From Baseline in Clinical Global Impression of Severity (CGI-S)
Description
Investigator's impression of the severity of the subject's illness on a 7-point scale ranging from normal (1) to among the most extremely ill subjects (7). A negative change from baseline represents a decrease in the severity of the subject's illness.
Time Frame
Baseline and after week 6
Other Pre-specified Outcome Measures:
Title
Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39)
Description
PDQ-39 is a scale measuring subject reported aspects of functioning and well-being. Each of the 39 items is rated using a 5-points Likert scale, with 0 for never having difficulties/problems and 4 for all always having difficulties/problems. The total score is the sum of the 39 items, with a minimum score of 0 and a maximum score of 156. A negative change from baseline represents an improvement in functioning and well-being.
Time Frame
Baseline and after week 6
Title
Change From Baseline in Parkinson's Fatigue Scale (PFS-16)
Description
PFS-16 is a subject-reported scale evaluating the physical effects of fatigue and its impact on daily function, with a minimum score of 16 and a maximum score of 80. Ratings are based on feelings and experiences over the prior 2 weeks, with scoring ranging from 1 (strongly disagree) to 5 (strongly agree), where lower scores are associated with less fatigue and higher scores are associated with greater fatigue. A negative change from baseline represents an improvement in the physical effects of fatigue and the impact on daily function. .
Time Frame
Baseline and after week 6
Title
Hospital Anxiety and Depression Scale - Change From Baseline in Anxiety
Description
The Hospital Anxiety and Depression Scale is a 14-item Investigator-rated scale designed to determine levels of anxiety and depression in people with physical health problems. Seven items relate to anxiety and 7 items related to depression, where the scale ranges from 0 (best score possible) to 21 (worst score possible). A negative change from baseline represents a decrease in anxiety.
Time Frame
Baseline and after week 6
Title
Number of Participants in Each Category of Patient Global Impression of Change (PGI-C)
Description
Self reported (caregiver, if applicable) impression of change in the subject's symptoms from baseline assessment visit to end of study assessment, on a 7-point scale ranging from much improved to very much worse.
Time Frame
After week 6
Title
Change From Baseline in Number of Falls as Reported by the Subject in the Past 1 Week
Description
Subject reported number of falls. A negative change from baseline represents a decrease in the frequency of falls.
Time Frame
Baseline and after week 6
Title
Change From Baseline in Patient Global Impression of Severity (PGI-S)
Description
Self reported (caregiver, if applicable) impression of the severity of the subject's illness on a 7-point scale ranging from normal (1) to among the most extremely ill subjects (7). A negative change from baseline represents a decrease in the severity of the subject's illness.
Time Frame
Baseline and after week 6
Title
Hospital Anxiety and Depression Scale - Change From Baseline in Depression
Description
The Hospital Anxiety and Depression Scale is a 14-item Investigator-rated scale designed to determine levels of anxiety and depression in people with physical health problems. Seven items relate to anxiety and 7 items related to depression, where the scale ranges from 0 (best score possible) to 21 (worst score possible). A negative change from baseline represents a decrease in depression.
Time Frame
Baseline and after week 6

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
78 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Able to understand and sign an Investigational Review Board (IRB)-approved written informed consent and privacy language (e.g., Health Insurance Portability and Accountability Act (HIPAA) authorization) as per national regulations before any study-related procedures are performed. Must have a diagnosis of idiopathic PD. Adult male or female 18 to 78 years of age, inclusive. Currently using APOKYN, per the Package Insert, for at least 4 weeks before Baseline Assessment Visit(s). Currently using carbidopa/levodopa therapy at a steady maintenance dose, representing a stable treatment regimen in the opinion of the Investigator, for at least 4 weeks before Baseline Assessment Visit(s). Able to walk 50 feet independently (cane permissible) at the Baseline Assessment Visit. Meet all of the following parameters to demonstrate an optimal response to APOKYN at the APOKYN Response Verification Visit: must be "on" in the opinion of the subject and investigator within 20 minutes after the APOKYN injection; and have a motor state improvement of >25% per the Movement Disorder Society (MDS)-UPDRS Motor Score (Part III), within 20 minutes after the APOKYN injection, as compared to pre-injection. Willing and able to comply with scheduled visits, treatment plan, other study-related procedures, and have available caregiver to provide transportation to clinic visits as needed. Exclusion Criteria: Received PT targeting Parkinson's disease within 6 months before Baseline Assessment Visit(s). Received any investigational (i.e., unapproved) drug product within the last 30 days (or 5 half-lives, whichever is longer) before the Baseline Assessment Visit(s). Currently taking, or likely to need to take at any time during the course of the study, any 5HT3 antagonist (i.e., ondansetron, granisetron, dolasetron, palonosetron, alosetron). Currently on or likely to transition to the DUOPA (carbidopa and levodopa enteral suspension) during the course of the study. Has orthostatic hypotension (defined as >30 mmHg decrease in systolic blood pressure or >15 mmHg decrease in diastolic blood pressure within 2 minutes of standing from a supine position) at the Baseline Assessment Visit(s), or within 20 minutes following APOKYN injection at the APOKYN Verification Visit. Has a score of 24 or higher on the Modified Physical Performance Test (M-PPT). Has a score of 80% or higher on the Activities-specific Balance Confidence Scale (ABC). Has any significant current medical disorder, physical, or mental condition that would, in the Investigator's opinion, represent a hazard to the subject or prevent the subject from completing the study. Subjects with dementia, in the Investigator's opinion, or subjects with a Montreal Cognitive Assessment (MoCA) <18.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mark Pirner, MD
Organizational Affiliation
US WorldMeds
Official's Role
Study Director
Facility Information:
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85258
Country
United States
City
Loma Linda
State/Province
California
ZIP/Postal Code
92354
Country
United States
City
Sunnyvale
State/Province
California
ZIP/Postal Code
94085
Country
United States
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
City
Foxboro
State/Province
Massachusetts
ZIP/Postal Code
02035
Country
United States
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74136
Country
United States
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
City
Austin
State/Province
Texas
ZIP/Postal Code
78746
Country
United States
City
Kirkland
State/Province
Washington
ZIP/Postal Code
98034
Country
United States
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98409
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Outpatient Physical Therapy Intervention in Subjects With Parkinson's Disease Currently Using APOKYN®

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