Clinical trial NCT06355531: A Study to Assess the Efficacy, Safety, and Pharmacokinetics of FNP-223 to Slow Progression of Progressive Supranuclear Palsy (PSP)
Official title: A Randomized, Double-blind, Placebo-controlled, Phase 2 Study to Assess the Efficacy, Safety, and Pharmacokinetics of FNP-223 (Oral Formulation) to Slow the Disease Progression of Progressive Supranuclear Palsy (PSP) (PROSPER)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Progressive Supranuclear Palsy
Keywords: Progressive Supranuclear Palsy
Disease progression
FNP-223
Lead sponsor: Ferrer Internacional S.A.
Sponsor class: INDUSTRY
First posted: 2024-04-09
Enrollment: 220
Sex: ALL
Minimum age (years): 50
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: PROSPER trial is a trial to assess the efficacy of FNP-223 in slowing disease progression in participants with PSP as measured by the PSP Rating Scale (PSPRS) over 52 weeks and to assess the safety and tolerability of FNP-223 for 52 weeks in participants with PSP.


Eligibility criteria: Inclusion Criteria:

* Male or female participants aged 50 to 80 years, inclusive, at the time of informed consent.
* Diagnosis of possible or probable PSP of the Richardson's Syndrome (PSP-RS) phenotypes according to the Movement Disorders Society's Progressive Supranuclear Palsy (MDS PSP) clinical features criteria. At least 1 (either 1 or both) of the following 2 items must be met:

  1. Vertical supranuclear gaze palsy.
  2. Slowing of vertical saccades AND postural instability with falls within the first 3 years of PSP symptoms.
* Presence of PSP symptoms within ≤3 years prior to screening.
* MoCA score ≥23
* Full 28-item PSPRS score ≤40.
* Able to ambulate independently or with minimal assistance defined as the ability to take at least 10 steps (stabilization of 1 arm \[ie, use of cane\]).
* Body weight range ≥43 kg/95 lbs to ≤120 kg/265 lbs.
* Reside outside a skilled nursing facility or dementia care facility, except for participants residing in an assisted living facility.
* Has a caregiver or study partner who will accompany them to the study visits. The caregiver or study partner must be a person who has frequent contact (at least 7 hours per week at 1 time or in different days) with the participant and is able to provide information about the participant's medication and overall condition. Prior to the conduct of any study procedures, the caregiver or study partner must be willing to sign the independent ethics committee (IEC)/institutional review board (IRB) approved informed consent.

Exclusion Criteria:

Non-PSP- RS Movement Disorders or other central nervous system (CNS) Diseases

* Score of 3 on any functional domain in the PSP-CDS.
* Participants with known PSP genetic mutation (based on familiar or clinical history).
* Evidence of other neurological disorder that could explain signs of PSP (eg, Parkinson's disease, Alzheimer disease, etc.).
* Brain magnetic resonance imaging (MRI) within 1 year of screening consistent with:
* Primary degenerative diseases other than PSP.

Procedures

* For the optional substudy only: Contraindication or refusal to undergo 2 lumbar punctures for obtaining CSF.
* Contraindication or inability to tolerate MRI for screening MRI and volumetric brain MRI assessments throughout the substudy.

Primary outcomes:
measure: Change From Baseline to Week 52 in the PSPRS Outcome
timeFrame: Baseline to Week 52
measure: Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
timeFrame: Baseline to Week 52
measure: Number of Participants Experiencing Serious Adverse Events (SAEs)
timeFrame: Baseline to Week 52
Secondary outcomes:
measure: Change From Baseline to Week 52 in Clinical Global Impression of Severity Scale (CGI-S)
timeFrame: Baseline to Week 52
measure: Change From Baseline to Week 52 Participant Global Impression of Severity Scale (PGI-S)
timeFrame: Baseline to Week 52
measure: Change From Baseline to Week 52 in Caregiver Global Impression of Severity Scale (CaGI-S)
timeFrame: Baseline to Week 52
measure: Slope of Decline in PSPRS
timeFrame: Baseline to Week 52
measure: Change From Baseline to Week 52 in Individual Subitems of PSPRS
timeFrame: Baseline to Week 52
measure: Change From Baseline to Week 52 in Schwab and England Activities of Daily Living Scale
timeFrame: Baseline to Week 52
measure: Change From Baseline to Week 52 in PSP Clinical Deficits Scale (PSP-CDS)
timeFrame: Baseline to Week 52
measure: Change From Baseline to Week 52 in Montreal Cognitive Assessment (MoCA)
timeFrame: Baseline to Week 52
measure: Change From Baseline to Week 52 in PSP Quality of Life Scale (PSP-QoL)
timeFrame: Baseline to Week 52
measure: Pharmacokinetic characterization of FNP-223
timeFrame: At Week 4 and Week 16
Other outcomes:
Locations:
city: Carlsbad
country: United States
facility: The Neurology Center of Southern California - Carlsbad
state: California
city: San Francisco
country: United States
facility: UCSF Weill Institute for Neurosciences
state: California
city: Denver
country: United States
facility: Rocky Mountain Movement Disorders Center
state: Colorado
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Farmington Hills
country: United States
facility: Quest Research Institute
state: Michigan
city: New York
country: United States
facility: Robert & John M. Bendheim Parkinson and Movement Disorders Center at Mount Sinai
state: New York
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Durham
country: United States
facility: Duke Neurology
state: North Carolina
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Round Rock
country: United States
facility: Central Texas Neurology Consultants
state: Texas
city: Bordeaux
country: France
facility: Groupe Hospitalier Pellegrin
city: Marseille
country: France
facility: Hôpital de la Timone
city: Nîmes
country: France
facility: L'Hôpital Universitaire Carémeau
city: Paris
country: France
facility: Hôpital Universitaire Pitié Salpêtrière
city: Toulouse
country: France
facility: Hôpital Pierre-Paul Riquet
city: Beelitz
country: Germany
facility: Neurologisches Fachkrankenhaus für Bewegungsstörungen/Parkinson
city: Düsseldorf
country: Germany
facility: Universitätsklinikum Düsseldorf
city: Leipzig
country: Germany
facility: Universitätsklinikum Leipzig
city: Munich
country: Germany
facility: Ludwig-Maximilians-Universität München (LMU)
city: Pécs
country: Hungary
facility: Pécsi Tudományegyetem Általános Orvostudományi Kar
city: Bologna
country: Italy
facility: IRCCS Istituto Delle Scienze Neurologiche di Bologna
city: Padova
country: Italy
facility: Azienda Ospedale Università di Padova
city: Pisa
country: Italy
facility: Azienda Ospedaliero-Universitaria Pisana
city: Roma
country: Italy
facility: Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - San Raffaele Pisana
city: Salerno
country: Italy
facility: Azienda Ospedaliera Universitaria San Giovanni di Dio Ruggi d'Aragona
city: Katowice
country: Poland
facility: Neurologia Śląska Centrum Medyczne
city: Warsaw
country: Poland
facility: Mazowiecki Szpital Bródnowski w Warszawie
city: Braga
country: Portugal
facility: Hospital de Braga
city: Torres Vedras
country: Portugal
facility: Campus Neurológico Senior - Torres Vedras
city: A Coruña
country: Spain
facility: Complejo Hospitalario Universitario A Coruña
city: Badalona
country: Spain
facility: Hospital Germans Trias i Pujol
city: Barakaldo
country: Spain
facility: Hospital Universitario Cruces
city: Barcelona
country: Spain
facility: Hospital Clínic de Barcelona
city: Madrid
country: Spain
facility: Hospital Universitario Ramón y Cajal
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Pamplona
country: Spain
facility: Hospital Universitario de Navarra
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío
city: Valencia
country: Spain
facility: Hospital Universitari i Politècnic La Fe
city: Cambridge
country: United Kingdom
facility: University of Cambridge
city: London
country: United Kingdom
facility: University College London Hospitals
city: Newcastle
country: United Kingdom
facility: Newcastle Upon Tyne Hospitals
city: Southampton
country: United Kingdom
facility: University Hospital Southampton
Study officials:
References: