E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Progressive supranuclear palsy (PSP) |
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E.1.1.1 | Medical condition in easily understood language |
Progressive supranuclear palsy (PSP) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036813 |
E.1.2 | Term | Progressive supranuclear palsy |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the effect of RT001 on the 28 element PSPRS (PSPRS-28). |
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E.2.2 | Secondary objectives of the trial |
Secondary objective: - To evaluate the effect of RT001 in the Progressive Supranuclear Palsy Quality of Life (PSP-QoL) scale Safety objective: - To evaluate the safety of RT001 in patients with PSP
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
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E.4 | Principal 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated group. |
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E.5.1.1 | 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).
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E.5.2 | Secondary end point(s) |
Secondary endpoint: - Change from baseline in the PSP-QoL scale at 48 weeks for the RT001 treated group vs placebo-treated group. Safety endpoint: - Adverse events for the RT001 treated group vs placebo-treated group at 48 weeks. Exploratory endpoints: - To evaluate the effect of RT001 on the 15 element FDA Progressive Supranuclear Palsy Rating Scale (PSPRS-15) total score - Change from baseline in the PSPRS-10-MR at 48 weeks for the RT001 treated group vs placebo-treated group - Change from baseline in the PSPRS-10 at 48 weeks for the RT001 treated group vs placebo-treated group - Change from baseline in the CBFS at 48 weeks for the RT001 treated group vs placebo-treated group - Change from baseline in the CGI-C at 48 weeks for the RT001 treated group vs placebo-treated group
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E.5.2.1 | 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. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 11 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |