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

  • days left to enroll
  • participants needed
  • sponsor
    Retrotope, Inc.
Updated on 26 January 2022


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


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.

Condition Progressive Supranuclear Palsy
Treatment Placebo, RT001
Clinical Study IdentifierNCT04937530
SponsorRetrotope, Inc.
Last Modified on26 January 2022


Yes No Not Sure

Inclusion Criteria

Sign the informed consent form prior to entry into the study
Male or female subject with age 40 years to 80 years at the time of signed consent
Meets the MDS-PSP study group criteria for possible or probable progressive supranuclear palsy with Richardson syndrome (postural instability and falls with vertical ocular motor dysfunction)
Presence of PSP symptoms for less than 4 years
Score of <40 on the PSPRS-28
Ambulatory patients (with or without assistive device - no handheld help) and capable of performing study assessments/evaluations
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 to travel to the required visits
Willing to provide the necessary blood samples

Exclusion Criteria

Received treatment with other experimental therapies within the last 30 days prior to the first dose. The last dose of the prior experimental agent must have occurred more than 5 half-lives prior to enrollment in the current trial
Previously received treatment with RT001
Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening/Baseline till last study procedure completed)
Mini mental state examination (MMSE) score less than 20 at screening
Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period
Evidence of any clinically significant neurological disorder other than PSP in particular CBS
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 the dose had been stable for 60 days prior to screening
The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria
Subject has had a significant illness or infection requiring medical intervention in the past 30 days
Subject has evidence of any disease or condition (based on either history, physical or laboratory values) that might interfere with the conduct of the study
Is currently receiving active deep brain stimulation (DBS) that cannot be turned off
Any condition with a life expectancy of less than 2 years
Female who is breastfeeding or has a positive pregnancy test
Male participant or female participant of childbearing potential, who is sexually active and unwilling/unable to use a highly effective birth control method throughout the study
Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled
History, within the last 2 years, of alcohol abuse or physical opioid dependence
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How to participate?

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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