A Study to Assess the Efficacy, Safety, and Pharmacokinetics of FNP-223 to Slow Progression of Progressive Supranuclear Palsy (PSP)

Phase 2

Recruiting

• Conditions: Progressive Supranuclear Palsy
• Interventions: Drug: FNP-223; Drug: Placebo

• Registration Number: NCT06355531
• Lead Sponsor: Ferrer Internacional S.A.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-03-26
• Study First Submitted QC: 2024-04-03
• Study First Posted: 2024-04-09
• Study Start: 2024-07-23
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-04
• Last Update Posted: 2024-12-05
• Primary Completion: 2026-11
• Study Completion: 2026-11

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 220

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 ≤3 years.

• 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.

• 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.

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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 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 volumetric brain MRI assessments throughout the study.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
FNP-223	FNP-223	Participants will receive FNP-223 orally (PO), 3 times daily (TID).
Placebo	Placebo	Participants will receive matching placebo, PO, TID.

Primary Outcome Measures

Name	Time	Method
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)	Baseline to Week 52	Clinically significant changes in vital signs, clinical laboratory evaluations, physical examinations, and electrocardiogram (ECG) are included in TEAEs.
Number of Participants Experiencing Serious Adverse Events (SAEs)	Baseline to Week 52
Change From Baseline to Week 52 in the PSPRS Outcome	Baseline to Week 52

Secondary Outcome Measures

Name	Time	Method
Change From Baseline to Week 52 in Clinical Global Impression of Severity Scale (CGI-S)	Baseline to Week 52
Change From Baseline to Week 52 Participant Global Impression of Severity Scale (PGI-S)	Baseline to Week 52
Change From Baseline to Week 52 in Caregiver Global Impression of Severity Scale (CaGI-S)	Baseline to Week 52
Slope of Decline in PSPRS	Baseline to Week 52
Change From Baseline to Week 52 in PSP Quality of Life Scale (PSP-QoL)	Baseline to Week 52
Pharmacokinetic characterization of FNP-223	At Week 4 and Week 16	Mean plasma concentration of FNP-223.
Change From Baseline to Week 52 in Individual Subitems of PSPRS	Baseline to Week 52
Change From Baseline to Week 52 in Schwab and England Activities of Daily Living Scale	Baseline to Week 52
Change From Baseline to Week 52 in PSP Clinical Deficits Scale (PSP-CDS)	Baseline to Week 52
Change From Baseline to Week 52 in Montreal Cognitive Assessment (MoCA)	Baseline to Week 52

Trial Locations

Locations (29)

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

Central Texas Neurology Consultants; 🇺🇸 Round Rock, Texas, United States

Groupe Hospitalier Pellegrin; 🇫🇷 Bordeaux, France

IRCCS Istituto Delle Scienze Neurologiche di Bologna; 🇮🇹 Bologna, Italy

Neurologia Śląska Centrum Medyczne; 🇵🇱 Katowice, Poland

Mazowiecki Szpital Bródnowski w Warszawie; 🇵🇱 Warsaw, Poland

Hospital de Braga; 🇵🇹 Braga, Portugal

Campus Neurológico Senior - Torres Vedras; 🇵🇹 Torres Vedras, Portugal

Complejo Hospitalario Universitario A Coruña; 🇪🇸 A Coruña, Spain

Hospital Germans Trias i Pujol; 🇪🇸 Badalona, Spain

Hospital Universitario Cruces; 🇪🇸 Barakaldo, Spain

Hospital Clínic de Barcelona; 🇪🇸 Barcelona, Spain

Hospital Universitario Ramón y Cajal; 🇪🇸 Madrid, Spain

Hospital Universitario 12 de Octubre; 🇪🇸 Madrid, Spain

Hospital Universitario de Navarra; 🇪🇸 Pamplona, Spain

L'Hôpital Universitaire Carémeau; 🇫🇷 Nîmes, France

Hospital Universitari i Politècnic La Fe; 🇪🇸 Valencia, Spain

Neurologisches Fachkrankenhaus für Bewegungsstörungen/Parkinson; 🇩🇪 Beelitz, Germany

Pécsi Tudományegyetem Általános Orvostudományi Kar; 🇭🇺 Pécs, Hungary

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - San Raffaele Pisana; 🇮🇹 Roma, Italy

Azienda Ospedaliera Universitaria San Giovanni di Dio Ruggi d'Aragona; 🇮🇹 Salerno, Italy

Hospital Universitario Virgen del Rocío; 🇪🇸 Sevilla, Spain

The Neurology Center of Southern California - Carlsbad; 🇺🇸 Carlsbad, California, United States

UCSF Weill Institute for Neurosciences; 🇺🇸 San Francisco, California, United States

Rocky Mountain Movement Disorders Center; 🇺🇸 Denver, Colorado, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Quest Research Institute; 🇺🇸 Farmington Hills, Michigan, United States

Robert & John M. Bendheim Parkinson and Movement Disorders Center at Mount Sinai; 🇺🇸 New York, New York, United States

Duke Neurology; 🇺🇸 Durham, North Carolina, United States