PF 06412562 in Subjects With Advanced Stage Parkinson's Disease
- Conditions
- Parkinson Disease
- Interventions
- Drug: Standard of Care Placebo
- Registration Number
- NCT03665454
- Lead Sponsor
- Milton S. Hershey Medical Center
- Brief Summary
The purpose of this study is to test the safety and tolerability of the investigational drug PF-06412562 compared to the current medical standard of care medication for Parkinson's disease, carbidopa/levodopa. This research also is being done to find out if the investigational drug PF-06412562 can help improve the motor (movement) function, alertness, and cognitive (thinking) skills of people who are considered to be in the advanced-stage of Parkinson's disease. In this study, PF06412562 is 'investigational,' which means that it is experimental and has not been approved by the US Food and Drug Administration (FDA), but can be used in clinical research studies such as this one.
- Detailed Description
The investigators are conducting a randomized, double-blind, carbidopa/levodopa-controlled crossover safety, tolerability, and efficacy study of the investigational compound, PF-06412562, in advanced Parkinson's patients. The primary purpose of this study is to test the safety and tolerability of the investigational drug PF-06412562 compared to the current medical standard of care medication for Parkinson's disease, carbidopa/levodopa, in these patients. This secondary purpose of this research is to find out if the investigational drug PF-06412562 can help improve the motor (movement) function, alertness, and cognitive (thinking) skills of people who are considered to be in the advanced-stage of Parkinson's disease. This study will inform the field as to whether PF-06412562 is safe and well-tolerated in advanced Parkinson's patients, and also may provide preliminary data on its efficacy in several domains.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 8
- Diagnosis of classic PD with history of clinically meaningful response to levodopa
- Disease duration >15 years since diagnosis
- Hoehn & Yahr stage >IV "on" or "off" levodopa
- Consent signed by subject, if possible
- If subject is cognitively impaired, consent signed by power of attorney or legally authorized subject representative
- Assent from the study subject, if possible
- Stable dose of all medications for 60 days prior to Day 1 of first week of study
- Atypical parkinsonian syndrome (e.g., never responded to levodopa, and/or atypical signs)
- Acute or unstable medical condition such as heart disease, kidney and liver failure
- History of HIV, hepatitis B and C
- Use of moderate to strong CYP 3A4 modulators (see both inhibitors and inducers in Appendix BB)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description PF-06412562 PF-06412562 Subjects will receive 25 mg of PF-06412562 twice daily on study Days 2 and 3, along with placebo carbidopa/levodopa dosed at these times. Additional carbidopa/levodopa placebo capsules also will be administered according to the subject's home regimen. Standard of Care carbidopa/levodopa Standard of Care Placebo Subjects will take placebo tablets for the PF-06412562 twice daily on study Days 2 and 3, along with active carbidopa/levodopa (25/100 mg) administered at these times. Additional active carbidopa/levodopa capsules also will be administered according to the subject's home regimen.
- Primary Outcome Measures
Name Time Method Safety and tolerability of PF-06412562 assessed by blood sample results Day 4 Red blood cell count (M/uL)
Safety and tolerability of PF-06412562 assessed by cardiology testing/cardiac autonomic function X3 on Days 3 over 15 min each time heart rate (beats/min), sinus arrhythmia (beats/min)
Safety and tolerability of PF-06412562 assessed by Columbia Suicide Rating X1 on Day 4 C-SSRS measures suicidal ideation and/or behavior. Rated from Category 1-10 with Category 1 being the least severe, and Category 10 representing the most severe, i.e. completing suicide.
Safety and tolerability of PF-06412562 assessed by vital signs X2 on Day 4 temperature (F)
Safety and tolerability of PF-06412562 assessed by UPDRS-IV X3 on Days 3 UPDRS-IV (United Parkinson's Disease Rating Scale): each questions scored on scale of 0 to 4, where 0= not present, 4= severe; subscales are summed to yield a total score, min total score:0, max total score:81; higher score corresponds to increased disease severity
- Secondary Outcome Measures
Name Time Method Pilot data on potential efficacy of PF-06412562 compared to the standard of care treatment (i.e., carbidopa/levodopa) on overall signs and quality of life in subjects with advanced PD Global Impression of Change: X3 on Days 2 and 3; Caregiver Perception of Change: X1 on Day 3 • Clinician Global Impression of Change score: measures change in disease symptoms per clinician's judgement. Each item rated 1-7, 1=marked improvement, 7= marked worsening; min Caregiver perception of change interview: interviews with participants' caregiver(s). Open-ended questions elicit open-ended answers about subject's behavior, appearance, cognition, etc. over the study
Trial Locations
- Locations (1)
Penn State Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States