RT001 in Patients With Progressive Supranuclear Palsy (PSP) PROGRESSIVE SUPRANUCLEAR PALSY
Primary Purpose
Progressive Supranuclear Palsy
Status
Unknown status
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
RT001
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Progressive Supranuclear Palsy
Eligibility Criteria
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
Sites / Locations
- Agaharied Teaching Hospital, University of MunichRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
RT001
Placebo
Arm Description
RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.
Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.
Outcomes
Primary Outcome Measures
Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group
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.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04937530
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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2021
Overall Recruitment Status
Unknown status
Study Start Date
June 23, 2021 (Actual)
Primary Completion Date
July 30, 2022 (Anticipated)
Study Completion Date
August 30, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Retrotope, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Progressive Supranuclear Palsy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
RT001
Arm Type
Experimental
Arm Description
RT001 960 mg capsule. 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.
Intervention Type
Drug
Intervention Name(s)
RT001
Other Intervention Name(s)
di-deuterated linoleic acid ester
Intervention Description
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
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
safflower oil
Intervention Description
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.
Primary Outcome Measure Information:
Title
Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group
Description
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.
Time Frame
48 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Mark G. Midei, MD
Phone
(650) 437-0700
Email
mark@retrotope.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stefan Lorenzl, MD, PhD
Organizational Affiliation
Ludwig-Maximilians - University of Munich
Official's Role
Principal Investigator
Facility Information:
Facility Name
Agaharied Teaching Hospital, University of Munich
City
Munich
State/Province
MD
ZIP/Postal Code
21111
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mark G. Midei, MD
Email
mark@retrotope.com
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
RT001 in Patients With Progressive Supranuclear Palsy (PSP) PROGRESSIVE SUPRANUCLEAR PALSY
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