- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04937530
RT001 in Patients With Progressive Supranuclear Palsy (PSP) PROGRESSIVE SUPRANUCLEAR PALSY
June 24, 2021 updated by: Retrotope, Inc.
A Randomized, Double-blind, Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Progressive Supranuclear Palsy
This is a randomized, placebo-controlled trial of RT001 in patients with PSP.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
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 Type
Interventional
Enrollment (Anticipated)
40
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Mark G. Midei, MD
- Phone Number: (650) 437-0700
- Email: mark@retrotope.com
Study Locations
-
-
MD
-
Munich, MD, Germany, 21111
- Recruiting
- Agaharied Teaching Hospital, University of Munich
-
Contact:
- Mark G. Midei, MD
- Email: mark@retrotope.com
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
40 years to 80 years (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: QUADRUPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: RT001
RT001 960 mg capsule.
3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.
|
RT001 8.64 g/d (3 capsules TID) for 1 month followed by 5.76 g/d (3 capsules BID) for an additional 11 months
Other Names:
|
PLACEBO_COMPARATOR: Placebo
Inactive comparator capsule 960 mg (safflower oil).
3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.
|
Placebo Comparator: Placebo Inactive comparator capsule 960 mg/capsule: 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group
Time Frame: 48 weeks
|
The PSPRS-28 is a clinician-rated instrument to assess disability and severity of PSP.
The PSPRS-28 evaluates 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline.
|
48 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Stefan Lorenzl, MD, PhD, Ludwig-Maximilians - University of Munich
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
June 23, 2021
Primary Completion (ANTICIPATED)
July 30, 2022
Study Completion (ANTICIPATED)
August 30, 2022
Study Registration Dates
First Submitted
June 16, 2021
First Submitted That Met QC Criteria
June 16, 2021
First Posted (ACTUAL)
June 24, 2021
Study Record Updates
Last Update Posted (ACTUAL)
June 25, 2021
Last Update Submitted That Met QC Criteria
June 24, 2021
Last Verified
June 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- RT001-013
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Progressive Supranuclear Palsy
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Novartis PharmaceuticalsActive, not recruitingProgressive Supranuclear Palsy (PSP)Germany, United Kingdom, Canada, United States
-
AbbVieTerminatedProgressive Supranuclear Palsy (PSP)United States, Australia, Canada, Italy, Japan
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AbbVieCompletedProgressive Supranuclear Palsy (PSP)United States
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Assistance Publique Hopitaux De MarseilleCompletedProgressive Supranuclear Palsy (PSP)France
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Oregon Health and Science UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingSupranuclear Palsy, Progressive | Palsy SupranuclearUnited States
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University of California, San FranciscoNational Institutes of Health (NIH); National Institute on Aging (NIA)Active, not recruitingProgressive Supranuclear Palsy (PSP) | Corticobasal Degeneration (CBD) | Nonfluent Variant Primary Progressive Aphasia (nfvPPA) | Corticobasal Syndrome (CBS) | Cortical-basal Ganglionic Degeneration (CBGD) | Oligosymptomatic/Variant Progressive Supranuclear Palsy (o/vPSP)United States, Canada
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University of California, San FranciscoTau Consortium; CBD SolutionsCompletedProgressive Supranuclear Palsy (PSP) | Corticobasal Degeneration (CBD) | Corticobasal Syndrome (CBS) | Primary Four Repeat Tauopathies (4RT)United States
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UCB Biopharma SRLCompletedProgressive Supranuclear PalsyBelgium, Germany, Spain, United Kingdom
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Centre Hospitalier Universitaire de Pointe-a-PitreGroupe Hospitalier Pitie-Salpetriere; University Hospital Center of MartiniqueCompletedProgressive Supranuclear Palsy
-
University of LouisvilleCompletedProgressive Supranuclear PalsyUnited States
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