This is a Phase 2b study investigating the efficacy and safety of pirepemat as adjunct therapy on falls frequency in patients with Parkinson disease. Pirepemat is taken for 84 days.
At the screening visit consenting patients will be screened for eligibility according to study specific inclusion/exclusion criteria within 6 weeks before start of Investigational Medicinal Product (IMP) administration. Patients will be asked to complete a fall diary for at least 4 consecutive weeks during the screening period and to be eligible for randomization, the patient should have experienced at least 2 falls during the 4 weeks preceding the baseline visit. At the baseline visit, patients will be randomized to receive one of two doses of Pirepemat (dose 1 or dose 2) or placebo t.i.d. (1:1:1). Dosing will start with half the dose for the first week of treatment and then continue with full dose until Week 11. Dosing will be de-escalated according to pre-specified schedule during the last week of study treatment, ending with the last dose on Day 84. The treatment allocation will be double-blind, i.e. it will not be disclosed to the patients, the site staff or the Sponsor. During the treatment period, patients will capture falls at home using a fall diary and changes in cognitive, postural, motor and mental functions will be assessed using the Montreal Cognitive Assessment (MoCA), Movement Disorder Society - Unified Parkinson´s Disease Rating Scale (MDS-UPDRS), Neuropsychiatric Inventory (NPI) (Apathy/Indifference part), Single Leg Stance Test, Tandem walking test, and Clinician's Global Impression of Severity (CGI-S) and Improvement (CGI-I). Blood samples for pharmacokinetic (PK) analysis will be collected at visit 5 (week 6) and visit 8 (week 11). Following the last IMP dose, a safety follow-up period (including laboratory assessments at 3 instances) of approximately 1 month will take place.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
104
Change in falls frequency with Pirepemat compared to placebo as assessed with fall diary from baseline period (4 weeks prior to randomization) to the end of treatment.
Falls being recorded using a paper fall diary (Patient Reported Outcome, PRO)
Time frame: Baseline to end of treatment (week 12)
Change in the total score of MDS-UPDRS part 2 (M-EDL) from baseline to end of full dose treatment (with pirepemat compared to placebo).
The total scoring range is 0-52, where a higher score indicates more severe impact on Motor Aspects of Experiences of Daily Living (M-EDL).
Time frame: Baseline to end of full dose treatment (week 11)
Change in total score (Frequency*Severity) of NPI Item G (Apathy/Indifference) from baseline to end of full dose treatment (with pirepemat compared to placebo).
The total scoring range is 1-12, where a higher score indicates a higher degree of apathy/indifference.
Time frame: Baseline to end of full dose treatment (week 11)
Change in Caregiver distress of NPI Item G (Apathy/Indifference) from baseline to end of full dose treatment (with pirepemat compared to placebo).
The total scoring range is 0-5, where a higher score indicates a more severe caregiver distress.
Time frame: Baseline to end of full dose treatment (week 11)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Hôpital Neurologique Pierre Wertheimer
Bron, France
Hopital de la Timone
Marseille, France
Hôpital Laennec - Centre d'investigation clinique de Neurologie
Nantes, France
CHU Charles Nicolle
Rouen, France
CHU Toulouse - Hôpital Purpan
Toulouse, France
Charite Universitatsmedizin Berlin - Klinik fuer neurologie mit experimenteller neurologie
Berlin, Germany
Praxis Dr.med. Christian Oehlwein Facharzt für Neurologie und Psychiatrie
Gera, Germany
Universitätsmedizin Göttingen - Klinik für Neurologie
Göttingen, Germany
Klinische Forschung Hamburg GmbH
Hamburg, Germany
Universitaetsklinikum Leipzig - Klinik und Poliklinik fuer Neurologie
Leipzig, Germany
...and 27 more locations