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