THE PROGRESSIVE SUPRANUCLEAR PALSY CLINICAL TRIAL PLATFORM (PTP) - PROJECT SUMMARY / ABSTRACT PROGRESSIVE SUPRANUCLEAR PALSY (PSP) IS A SEVERE NEURODEGENERATIVE DISEASE OF AGING THAT USUALLY LEADS TO DEATH WITHIN 5-7 YEARS OF DIAGNOSIS. THERE ARE NO EFFECTIVE TREATMENTS FOR PSP, BUT THERAPEUTIC APPROACHES BEING TESTED IN CLINICAL TRIALS FOR OTHER NEURODEGENERATIVE DISEASES, SUCH AS ALZHEIMER’S (AD) AND AMYOTROPHIC LATERAL SCLEROSIS MAY BE EVEN MORE PROMISING FOR PSP. DESPITE EXCELLENT FEASIBILITY OF LARGE MULTICENTER PSP CLINICAL TRIALS, THERE ARE FEW NEW STUDIES, LIMITING OPTIONS FOR PATIENTS TO ACCESS EXPERIMENTAL THERAPIES AND SEVERELY DELAYING THE IDENTIFICATION OF EFFECTIVE TREATMENTS. NEW, EFFICIENT CLINICAL PSP TRIAL PROGRAMS AND FOCUSED EFFORTS TO IDENTIFY PSP BIOMARKERS ARE URGENTLY NEEDED. THE OVERARCHING GOAL OF THE PSP TRIAL PLATFORM (PTP) IS TO CONDUCT A RANDOMIZED, PLACEBO-CONTROLLED, PHASE 2 PLATFORM TRIAL IN MILD-MODERATE PSP THAT WILL SIMULTANEOUSLY TEST AT LEAST THREE

Summary

THE PROGRESSIVE SUPRANUCLEAR PALSY CLINICAL TRIAL PLATFORM (PTP) - PROJECT SUMMARY / ABSTRACT PROGRESSIVE SUPRANUCLEAR PALSY (PSP) IS A SEVERE NEURODEGENERATIVE DISEASE OF AGING THAT USUALLY LEADS TO DEATH WITHIN 5-7 YEARS OF DIAGNOSIS. THERE ARE NO EFFECTIVE TREATMENTS FOR PSP, BUT THERAPEUTIC APPROACHES BEING TESTED IN CLINICAL TRIALS FOR OTHER NEURODEGENERATIVE DISEASES, SUCH AS ALZHEIMER’S (AD) AND AMYOTROPHIC LATERAL SCLEROSIS MAY BE EVEN MORE PROMISING FOR PSP. DESPITE EXCELLENT FEASIBILITY OF LARGE MULTICENTER PSP CLINICAL TRIALS, THERE ARE FEW NEW STUDIES, LIMITING OPTIONS FOR PATIENTS TO ACCESS EXPERIMENTAL THERAPIES AND SEVERELY DELAYING THE IDENTIFICATION OF EFFECTIVE TREATMENTS. NEW, EFFICIENT CLINICAL PSP TRIAL PROGRAMS AND FOCUSED EFFORTS TO IDENTIFY PSP BIOMARKERS ARE URGENTLY NEEDED. THE OVERARCHING GOAL OF THE PSP TRIAL PLATFORM (PTP) IS TO CONDUCT A RANDOMIZED, PLACEBO-CONTROLLED, PHASE 2 PLATFORM TRIAL IN MILD-MODERATE PSP THAT WILL SIMULTANEOUSLY TEST AT LEAST THREE