A RANDOMIZED, DOUBLE-BLIND, CONTROLLED, PHASE 2 STUDY TO ASSESS EFFICACY, LONG TERM SAFETY AND TOLERABILITY OF RT001 IN SUBJECTS WITH PROGRESSIVE SUPRANUCLEAR PALSY.

• Register: EUCTR
• Last refreshed on: 21 December 2021
• Main ID: EUCTR2020-003911-90-DE
• Date of registration: 30/11/2020
• Prospective Registration: Yes
• Primary sponsor: Retrotope, Inc
• Public 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.
• Scientific 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.
• Date of first enrolment: 31/03/2021
• Target sample size: 50
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-003911-90
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Germany, Hungary, Poland, Slovakia

Contacts

• Name: Chief Medical Officer
• Affiliation: Retrotope Inc.

• Name: Chief Medical Officer
• Affiliation: Retrotope Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
To be enrolled into the study subjects must meet the following requirements:
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 PSP with Richardson Syndrome (postural instability and falls with vertical ocular motor dysfunction)
4. Presence of PSP symptoms for less than 4 years
5. Scoring 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 repeated blood samples.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15

Exclusion criteria:
Subjects meeting one or more of the following may not enter the study:
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. 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 day.
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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Progressive supranuclear palsy (PSP)
MedDRA version: 21.1 Level: PT Classification code 10036813 Term: Progressive supranuclear palsy System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: RT001
Product Code: RT001
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: not assigned
CAS Number: 1404475-07-5
Current Sponsor code: RT001
Other descriptive name: 9-CIS, 12-CIS-11,11-D2-LINOLEIC ACID ETHYL ESTER
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 960-
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: To evaluate the effect of RT001 on the 15 element FDA Progressive Supranuclear Palsy Rating Scale (PSPRS-15) total score.
To evaluate the effect of RT001 in the Progressive Supranuclear Palsy Quality of Life (PSP-QoL) scale .
To evaluate the safety of RT001 in patients with PSP.

Timepoint(s) of evaluation of this end point: The PSPRS-28 will be performed at Screening, Baseline, Week 12 (Day 84 ± 14 days), Week 24 Day 168 ± 14 days), Week 36 (Day 252 ± 14 days), Week 48 (Day 336 ± 14 days) and Week 52 (Day 364 ± 7 Days).

Main Objective: To evaluate the effect of RT001 on the 28 element PSPRS (PSPRS-28).

Primary end point(s): Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: The PSP-QoL will be performed at Baseline, Week 24 (Day 168 ± 14 days), and Week 48 (Day 336 ± 14 days).
The PSPRS-15 is a subset of the PSPRS-28 that evaluate certain items from PSPRS-28.

Secondary end point(s): Change from baseline in the PSPRS-15 at 48 weeks for the RT001 treated group vs placebo-treated group.
Change from baseline in the PSP-QoL scale at 48 weeks for the RT001 treated group vs placebo-treated group.
Adverse events for the RT001 treated group vs placebo-treated group at 48 weeks.

Secondary ID(s)

RT001-013

Source(s) of Monetary Support

Retrotope Inc.

Ethics review

Status: Approved
Approval date: 01/03/2021
Contact: