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

• ClinicalTrials.gov Identifier: NCT04993768
• Recruitment Status: Active, not recruiting
• First Posted: August 6, 2021
• Last Update Posted: July 13, 2023
• Sponsor: Transposon Therapeutics, Inc.
• Information provided by (Responsible Party): Transposon Therapeutics, Inc.

Tracking Information

• First Submitted Date: July 19, 2021
• First Posted Date: August 6, 2021
• Last Update Posted Date: July 13, 2023
• Actual Study Start Date: October 15, 2021
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 29, 2021)

Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP) [ Time Frame: 48 weeks ]

Incidence and severity of spontaneously reported treatment-emergent adverse events (TEAEs) associated with TPN-101 v. placebo administered for up to 48 weeks in patients with PSP

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: August 10, 2021)

• Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF) [ Time Frame: 48 weeks ]
• Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL) [ Time Frame: 48 weeks ]
• Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: 48 weeks ]
  The PSPRS is comprised of 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline. Scores range from 0 to 100, each item is graded 0-2 (six items) or 0-4 (22 items), with lower scores indicating better clinical and functional status.

Original Secondary Outcome Measures (submitted: July 29, 2021)

• Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF) [ Time Frame: 48 weeks ]
• Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL) [ Time Frame: 48 weeks ]
• Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: 48 weeks ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
• Official Title: A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
• Brief Summary: This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.
• Detailed Description: This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 4-arm study with an open-label treatment phase in patients with PSP. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open label Treatment Period, and a Follow-up Visit 4 weeks post treatment.
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment
• Condition: Progressive Supranuclear Palsy

Intervention

• Drug: TPN-101, 100 mg/day
  100 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
• Drug: TPN-101, 200 mg/day
  200 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
• Drug: TPN-101, 400 mg/day
  400 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
• Drug: Placebo
  Placebo once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).

Study Arms

• Experimental: TPN-101, Dose A
  Intervention: Drug: TPN-101, 100 mg/day
• Experimental: TPN-101, Dose B
  Intervention: Drug: TPN-101, 200 mg/day
• Experimental: TPN-101, Dose C
  Intervention: Drug: TPN-101, 400 mg/day
• Placebo Comparator: Placebo
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: July 29, 2021): 40
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 31, 2023
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Clinical diagnosis of probable progressive supranuclear palsy (PSP)
2. Presence of PSP symptoms for less than 5 years
3. Has a reliable caregiver/informant to accompany the patient to all study visits.
4. Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening
5. 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

Exclusion Criteria:

Patients must not meet any of the following criteria:

1. Presence of other significant neurological or psychiatric disorders
2. History of clinically significant brain abnormality
3. Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic dysfunction, or moderate to severe resting tremor, responsive to levodopa
4. Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean
5. 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
6. History of clinically significant hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 41 Years to 86 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04993768
• Other Study ID Numbers: TPN-101-PSP-201
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Transposon Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Transposon Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Transposon Therapeutics, Inc.
• Verification Date: July 2023