RT001 in Patients With Progressive Supranuclear Palsy (PSP) PROGRESSIVE SUPRANUCLEAR PALSY

Last updated: June 24, 2021
Sponsor: Retrotope, Inc.
Overall Status: Active - Recruiting

Phase

2

Condition

Progressive Supranuclear Palsy

Cataplexy

Treatment

N/A

Clinical Study ID

NCT04937530
RT001-013
  • Ages 40-80
  • All Genders

Study Summary

This is a randomized, placebo-controlled trial of RT001 in patients with PSP.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Sign the informed consent form prior to entry into the study
  2. Male or female subject with age 40 years to 80 years at the time of signed consent
  3. Meets the MDS-PSP study group criteria for possible or probable progressivesupranuclear palsy with Richardson syndrome (postural instability and falls withvertical ocular motor dysfunction)
  4. Presence of PSP symptoms for less than 4 years
  5. Score of <40 on the PSPRS-28
  6. Ambulatory patients (with or without assistive device - no handheld help) and capableof performing study assessments/evaluations
  7. Subject has an identified, reliable, study partner (e.g., caregiver, family member,social worker, or friend) who can assist in assuring that the subject is able totravel to the required visits.
  8. Willing to provide the necessary blood samples

Exclusion

Exclusion Criteria:

  1. Received treatment with other experimental therapies within the last 30 days prior tothe first dose. The last dose of the prior experimental agent must have occurred morethan 5 half-lives prior to enrollment in the current trial.
  2. Previously received treatment with RT001
  3. Refusal to discontinue fish oils or other oil-based supplements for the duration ofthe study (Screening/Baseline till last study procedure completed)
  4. Mini mental state examination (MMSE) score less than 20 at screening
  5. Subject resides at a skilled nursing or dementia care facility, or admission to such afacility is planned during the study period
  6. Evidence of any clinically significant neurological disorder other than PSP inparticular CBS
  7. Evidence of a clear and robust benefit from levodopa at the time of screening.Participants are permitted to take levodopa and other Parkinson's medications if thedose had been stable for 60 days prior to screening
  8. The subject has a history of or currently has schizophrenia, schizoaffective disorderor bipolar disorder according to DSM-V or ICD-10 criteria
  9. Subject has had a significant illness or infection requiring medical intervention inthe past 30 days
  10. Subject has evidence of any disease or condition (based on either history, physical orlaboratory values) that might interfere with the conduct of the study
  11. Is currently receiving active deep brain stimulation (DBS) that cannot be turned off
  12. Any condition with a life expectancy of less than 2 years
  13. Female who is breastfeeding or has a positive pregnancy test
  14. Male participant or female participant of childbearing potential, who is sexuallyactive and unwilling/unable to use a highly effective birth control method throughoutthe study
  15. Unwilling or unable to comply with the requirements of this protocol, including thepresence of any condition (physical, mental, or social) that is likely to affect thesubject's ability to return for visits as scheduled
  16. History, within the last 2 years, of alcohol abuse or physical opioid dependence

Study Design

Total Participants: 40
Study Start date:
June 23, 2021
Estimated Completion Date:
August 30, 2022

Study Description

This is a randomized, placebo-controlled trial of RT001 in patients with PSP to assess the efficacy, safety and tolerability of RT001. Subjects will be randomized to RT001 or placebo and will receive study drug for 48 weeks.

Connect with a study center

  • Agaharied Teaching Hospital, University of Munich

    Munich, MD 21111
    Germany

    Active - Recruiting

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