Parkinson's DiseasePhase 4PERFORMNCT02549573
Outpatient Physical Therapy Intervention in Subjects With Parkinson's Disease Currently Using APOKYN®
A Phase 4 study testing APOKYN and Physical Therapy in people with Parkinson's Disease and Motor Symptoms, run by MDD US Operations, LLC a subsidiary of Supernus Pharmaceuticals at 12 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 78 years
- Conditions the study is for
- Parkinson's Disease, Motor Symptoms
- What is being tested?
- APOKYN and Physical Therapy
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 12 sitesUnited States
- Study dates
- Jan 2016to Dec 2016
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 78 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
Show all 9Show fewer
- 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.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Study site
Austin, Texas, 78746
Study site
Cleveland, Ohio, 44106
Study site
Foxborough, Massachusetts, 02035
Study site
Hershey, Pennsylvania, 17033
Study site
Kirkland, Washington, 98034
Study site
Loma Linda, California, 92354
Study site
Orlando, Florida, 32803
Study site
Phoenix, Arizona, 85013
Study site
Scottsdale, Arizona, 85258
Study site
Sunnyvale, California, 94085
Study site
Tacoma, Washington, 98409
Study site
Tulsa, Oklahoma, 74136
About this study
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
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.
Read the 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.
- Conditions
- Parkinson's Disease, Motor Symptoms
- Being tested
- APOKYN, Physical Therapy
- Keywords
- Motor Symptoms, Parkinson's disease, Apokyn, Apomorphine, PD
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APOKYNDrug
Also called Apomorphine, APO
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.
Physical TherapyBehavioural
Also called PT
All subjects will participate in a standardized PT intervention
| Study group | Role | What they receive |
|---|---|---|
| Apokyn treatment before physical therapy | Active comparator | 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. Receives: Drug: APOKYN; Behavioral: Physical Therapy |
| Apokyn treatment withheld before physical therapy | Other | 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. Receives: Behavioral: Physical Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline and after week 6
Secondary outcomes (11)
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.
Measured: Baseline and after week 6
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.
Measured: Baseline and after week 6
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.
Measured: Baseline and after week 6
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.
Measured: Baseline and after week 6
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.
Measured: Baseline and after week 6
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).
Measured: Baseline and after week 6
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.
Measured: After week 6
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.
Measured: Baseline and after week 6
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.
Measured: Baseline and after week 6
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).
Measured: Baseline and after week 6
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.
Measured: Baseline and after week 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2015 (estimated)
- Study startJan 2016
- Primary completionDec 2016
- Study completionDec 2016
Why it stopped: Subject enrollment is unsatisfactory
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- MDD US Operations, LLC a subsidiary of Supernus Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark Pirner, MD, Study director, US WorldMeds
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02549573
- Sponsor's protocol ID
- USWM-AP1-4002
- Acronym
- PERFORM
- Status last verified
- Dec 2017
- First submitted
- 9 Sep 2015
- Last updated
- 1 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02549573
- Acronym / sponsor ID
- PERFORM / USWM-AP1-4002
- Conditions
- Parkinson's Disease, Motor Symptoms
- Keywords
- Motor Symptoms, Parkinson's disease, Apokyn, Apomorphine, PD
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Subject enrollment is unsatisfactory
- Enrolment
- 13 participants (actual)
- Lead sponsor
- MDD US Operations, LLC a subsidiary of Supernus PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jan 2016 (actual)
- Primary completion
- Dec 2016 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 15 Sep 2015 (estimated)
- Results
- Posted 12 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Apokyn treatment before physical therapy | Active comparator | Drug: APOKYN, Behavioral: Physical TherapyAPOKYN 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 physical therapy | Other | Behavioral: Physical TherapyAPOKYN 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. |
Who may qualify
- Ages
- 18 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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).
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| APOKYNAlso: Apomorphine, APOSubjects 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. | Drug | Apokyn treatment before physical therapy |
| Physical TherapyAlso: PTAll subjects will participate in a standardized PT intervention | Behavioural | Apokyn treatment before physical therapy, Apokyn treatment withheld before physical therapy |
Primary outcome
Change From Baseline in Activities-specific Balance Confidence (ABC) Scale
Time frame: Baseline and after week 6
Scale from 0 to 100% where an increase in percent represents an improvement in balance confidence.
11 secondary outcomes
Change From Baseline in Movement Disorder Societies Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III (Motor Examination)
Time frame: Baseline and after week 6
Change From Baseline in Modified Physical Performance Test (M-PPT)
Time frame: Baseline and after week 6
Change From Baseline in Timed-Up-and-Go (TUG) Test
Time frame: Baseline and after week 6
Change From Baseline in 6-Minute Walk Test (6MWT)
Time frame: Baseline and after week 6
Change From Baseline in Montreal Cognitive Assessment (MoCA)
Time frame: Baseline and after week 6
Change From Baseline in MDS-UPDRS Part I A
Time frame: Baseline and after week 6
Number of Participants in Each Category of Clinical Global Impression of Change (CGI-C)
Time frame: After week 6
Change From Baseline in MDS-UPDRS Part II
Time frame: Baseline and after week 6
Change From Baseline in MDS-UPDRS Part IV
Time frame: Baseline and after week 6
Change From Baseline in MDS-UPDRS Part I B
Time frame: Baseline and after week 6
Change From Baseline in Clinical Global Impression of Severity (CGI-S)
Time frame: Baseline and after week 6
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Parkinson's Disease
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Not yet recruiting
Compulsive Eating and Brain's Reward System in Different Pathologies
PARKINSON DISEASE (Disorder), Bipolar 1 Disorder
Not yet recruiting
Evaluating the Effect of the Zeen Mobility Device on Clinical Practicality, Safety, and Impact (Via Assessments of Walking, Standing Tolerance, Balance, Endurance, Effort, and Participation in Daily Tasks) in an Outpatient Rehabilitation Setting for Individuals With Mobility Impairments.
Stroke, Parkinson's Disease (PD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark Pirner, MD study director
US WorldMeds
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing APOKYN and Physical Therapy in people with Parkinson's Disease and Motor Symptoms, run by MDD US Operations, LLC a subsidiary of Supernus Pharmaceuticals at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 1 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Jan 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Activities-specific Balance Confidence (ABC) Scale (time frame: Baseline and after week 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2023.