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

NCT04937530 | Unknown Phase 2 FDA Drug

• 1 other identifier: RT001-013
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Progressive Supranuclear Palsy

Outcome Measures

Primary Outcomes (1)

• 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.

  48 weeks

Study Arms (2)

RT001

EXPERIMENTAL

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

Drug: RT001

Placebo

PLACEBO COMPARATOR

Inactive comparator capsule 960 mg (safflower oil). 3 capsules TID for 4 weeks, followed by 3 capsules BID for the remaining 44 weeks.

Drug: Placebo

Interventions

RT001 DRUG

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

Also known as: di-deuterated linoleic acid ester

RT001

Placebo DRUG

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.

Also known as: safflower oil

Placebo

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Biojiva LLC: lead

Study Sites (1)

Agaharied Teaching Hospital, University of Munich

Munich, MD, 21111, Germany

RECRUITING

MeSH Terms

Conditions

Supranuclear Palsy, Progressive

Interventions

RT001; Safflower Oil

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Ophthalmoplegia; Ocular Motility Disorders; Cranial Nerve Diseases; Tauopathies; Neurodegenerative Diseases; Paralysis; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Dietary Fats, Unsaturated; Dietary Fats; Fats; Lipids; Fats, Unsaturated; Plant Oils; Oils; Plant Preparations; Biological Products; Complex Mixtures; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Study Officials

• Stefan Lorenzl, MD, PhD

  Ludwig-Maximilians - University of Munich

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mark G. Midei, MD

CONTACT

(650) 437-0700; mark@retrotope.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 16, 2021
• First Posted: June 24, 2021
• Study Start: June 23, 2021
• Primary Completion: July 30, 2022
• Study Completion: August 30, 2022
• Last Updated: June 25, 2021

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)