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

Progressive Supranuclear Palsy Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06355531
• Other study ID #: FNP223-CT-2301
• Status: Recruiting
• Phase: Phase 2
• Start date: June 2024
• Est. completion date: November 2026

Study information

• Verified date: May 2024
• Source: Ferrer Internacional S.A.
• Contact: Ferrer MedInfo
• Phone: +34 609 850 565
• Email: medinfo[@]ferrer.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 220
• Est. completion date: November 2026
• Est. primary completion date: November 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years to 80 Years

Eligibility

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.

• 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.

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.

Related Conditions & MeSH terms

• Disease Progression
• Paralysis
• Progressive Supranuclear Palsy
• Supranuclear Palsy, Progressive

Intervention

Drug:
• FNP-223
Oral tablets
• Placebo
Oral tablets

Locations

Country	Name	City	State
United States	The Neurology Center of Southern California - Carlsbad	Carlsbad	California
United States	Rocky Mountain Movement Disorders Center	Denver	Colorado
United States	Quest Research Institute	Farmington Hills	Michigan
United States	Central Texas Neurology Consultants	Round Rock	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Ferrer Internacional S.A.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline to Week 52 in the PSPRS Outcome		Baseline to Week 52
Primary	Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)	Clinically significant changes in vital signs, clinical laboratory evaluations, physical examinations, and electrocardiogram (ECG) are included in TEAEs.	Baseline to Week 52
Primary	Number of Participants Experiencing Serious Adverse Events (SAEs)		Baseline to Week 52
Secondary	Change From Baseline to Week 52 in Clinical Global Impression of Severity Scale (CGI-S)		Baseline to Week 52
Secondary	Change From Baseline to Week 52 Participant Global Impression of Severity Scale (PGI-S)		Baseline to Week 52
Secondary	Change From Baseline to Week 52 in Caregiver Global Impression of Severity Scale (CaGI-S)		Baseline to Week 52
Secondary	Slope of Decline in PSPRS		Baseline to Week 52
Secondary	Change From Baseline to Week 52 in Individual Subitems of PSPRS		Baseline to Week 52
Secondary	Change From Baseline to Week 52 in Schwab and England Activities of Daily Living Scale		Baseline to Week 52
Secondary	Change From Baseline to Week 52 in PSP Clinical Deficits Scale (PSP-CDS)		Baseline to Week 52
Secondary	Change From Baseline to Week 52 in Montreal Cognitive Assessment (MoCA)		Baseline to Week 52
Secondary	Change From Baseline to Week 52 in PSP Quality of Life Scale (PSP-QoL)		Baseline to Week 52
Secondary	Pharmacokinetic characterization of FNP-223	Mean plasma concentration of FNP-223.	At Week 4 and Week 16