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    Summary
    EudraCT Number:2020-003911-90
    Sponsor's Protocol Code Number:RT001-013
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-04-20
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-003911-90
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, CONTROLLED, PHASE 2 STUDY TO ASSESS EFFICACY, LONG TERM SAFETY AND TOLERABILITY OF RT001 IN SUBJECTS WITH PROGRESSIVE SUPRANUCLEAR PALSY.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A RANDOMIZED, DOUBLE-BLIND, CONTROLLED, PHASE 2 STUDY TO ASSESS EFFICACY, LONG TERM SAFETY AND TOLERABILITY OF RT001 IN SUBJECTS WITH PROGRESSIVE SUPRANUCLEAR PALSY.
    A.4.1Sponsor's protocol code numberRT001-013
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRetrotope, Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportRetrotope Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRetrotope Inc.
    B.5.2Functional name of contact pointChief Medical Officer
    B.5.3 Address:
    B.5.3.1Street Address4300 El Camino Real, Suite 201
    B.5.3.2Town/ city Los Altos
    B.5.3.3Post codeCA94022
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1408-802-9619
    B.5.6E-mailpeter.milner@retrotope.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRT001
    D.3.2Product code RT001
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot assigned
    D.3.9.1CAS number 1404475-07-5
    D.3.9.2Current sponsor codeRT001
    D.3.9.3Other descriptive name9-CIS, 12-CIS-11,11-D2-LINOLEIC ACID ETHYL ESTER
    D.3.9.4EV Substance CodeSUB194388
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number960
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, soft
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Progressive supranuclear palsy (PSP)
    E.1.1.1Medical condition in easily understood language
    Progressive supranuclear palsy (PSP)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10036813
    E.1.2Term Progressive supranuclear palsy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    This study is designed to evaluate the effect of RT001 on the 28 element PSPRS (PSPRS-28).
    E.2.2Secondary 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.
    E.2.3Trial contains a sub-study No
    E.3Principal 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.
    E.4Principal 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 days
    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 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.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint:
    - Change from baseline in the PSPRS-28 at 48 weeks for the RT001 treated group vs placebo-treated groupTo evaluate the effect of RT001 on the PSPRS-28 total score
    E.5.1.1Timepoint(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), and Week 48 (Day 336 ± 14 days).
    E.5.2Secondary end point(s)
    Secondary endpoints:
    - 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

    E.5.2.1Timepoint(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 evaluates certain items from PSPRS-28 and 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) and Week 48 (Day 336 ± 14 days).
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 50
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    An open label extension study may be offered to eligible subjects in lieu of the 28-day washout after the 48-week treatment period is completed.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-05-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-12-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-08-29
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