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

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Brief Title

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

Official Title

A Randomized, Double-blind, Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Progressive Supranuclear Palsy

Brief Summary

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

Detailed 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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group


Condition

Progressive Supranuclear Palsy

Intervention

RT001

Study Arms / Comparison Groups

 RT001
Description:  RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

40

Start Date

June 23, 2021

Completion Date

August 30, 2022

Primary Completion Date

July 30, 2022

Eligibility Criteria

        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 progressive
             supranuclear palsy with Richardson syndrome (postural instability and falls with
             vertical 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 capable
             of 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 to
             travel to the required visits.

          8. Willing to provide the necessary blood samples

        Exclusion Criteria:

          1. 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.

          2. Previously received treatment with RT001

          3. Refusal to discontinue fish oils or other oil-based supplements for the duration of
             the 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 a
             facility is planned during the study period

          6. Evidence of any clinically significant neurological disorder other than PSP in
             particular 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 the
             dose had been stable for 60 days prior to screening

          8. The subject has a history of or currently has schizophrenia, schizoaffective disorder
             or bipolar disorder according to DSM-V or ICD-10 criteria

          9. Subject has had a significant illness or infection requiring medical intervention in
             the past 30 days

         10. 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

         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 sexually
             active and unwilling/unable to use a highly effective birth control method throughout
             the study

         15. 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

         16. History, within the last 2 years, of alcohol abuse or physical opioid dependence
      

Gender

All

Ages

40 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

Stefan Lorenzl, MD, PhD, (650) 437-0700, [email protected]

Location Countries

Germany

Location Countries

Germany

Administrative Informations


NCT ID

NCT04937530

Organization ID

RT001-013


Responsible Party

Sponsor

Study Sponsor

Retrotope, Inc.


Study Sponsor

Stefan Lorenzl, MD, PhD, Principal Investigator, Ludwig-Maximilians - University of Munich


Verification Date

June 2021