CompletedPhase 2

A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)

United States42 participantsStarted 2021-12-12

Plain-language summary

This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.

Age range41 Years – 86 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Clinical diagnosis of probable progressive supranuclear palsy (PSP)
✓. Presence of PSP symptoms for less than 5 years
✓. Has a reliable caregiver/informant to accompany the patient to all study visits.
✓. Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening
✓. Patient must reside outside a skilled nursing facility or dementia care facility at the time of Screening, and admission to such a facility must not be planned. Residence in an assisted living facility is allowed

✕. Presence of other significant neurological or psychiatric disorders
✕. History of clinically significant brain abnormality
✕. Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic dysfunction, or moderate to severe resting tremor, responsive to levodopa
✕. Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean
✕. Known presence of disease-associated mutation in TARDBP, GRN, CHMPB2, or VCP genes; or any other frontotemporal lobar degeneration causative genes not associated with underlying tau pathology
✕. History of clinically significant hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease

Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)

Timeframe: 48 weeks

NCT IDNCT04993768

SponsorTransposon Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-02-24

Who can participate

Age range41 Years – 86 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Clinical diagnosis of probable progressive supranuclear palsy (PSP)
✓. Presence of PSP symptoms for less than 5 years
✓. Has a reliable caregiver/informant to accompany the patient to all study visits.
✓. Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening
✓. Patient must reside outside a skilled nursing facility or dementia care facility at the time of Screening, and admission to such a facility must not be planned. Residence in an assisted living facility is allowed

✕. Presence of other significant neurological or psychiatric disorders
✕. History of clinically significant brain abnormality
✕. Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic dysfunction, or moderate to severe resting tremor, responsive to levodopa
✕. Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean
✕. Known presence of disease-associated mutation in TARDBP, GRN, CHMPB2, or VCP genes; or any other frontotemporal lobar degeneration causative genes not associated with underlying tau pathology
✕. History of clinically significant hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease