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    Summary
    EudraCT Number:2020-003962-38
    Sponsor's Protocol Code Number:RT001-014
    National Competent Authority:Estonia - SAM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-11-03
    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 ConcernedEstonia - SAM
    A.2EudraCT number2020-003962-38
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 2 STUDY TO ASSESS EFFICACY, LONG TERM SAFETY AND TOLERABILITY OF RT001 IN SUBJECTS WITH AMYOTROPHIC LATERAL SCLEROSIS
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 2 STUDY TO ASSESS EFFICACY, LONG TERM SAFETY AND TOLERABILITY OF RT001 IN SUBJECTS WITH AMYOTROPHIC LATERAL SCLEROSIS
    A.4.1Sponsor's protocol code numberRT001-014
    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/ cityLos Altos
    B.5.3.3Post codeCA 94022
    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
    Amyotrophic Lateral Sclerosis
    E.1.1.1Medical condition in easily understood language
    Amyotrophic Lateral Sclerosis
    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 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary objective is to evaluate the effect of RT001 on the ALSFRS-R in subjects with ALS.
    E.2.2Secondary objectives of the trial
    Secondary Objectives:
    • To evaluate the effect of RT001 on ALS clinical outcomes
    • To evaluate the effect of RT001 on various functional assessments, pulmonary functional assessments and motor assessments
    • To assess the long-term safety and tolerability of RT001
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subject with age 20 years to 75 years at the time of signed consent for double-blind part of the study
    2. Patients who are defined as "definite ALS," "probable ALS" or "probable-laboratory-supported ALS," met diagnostic criteria revised EL Escorial for Airlie House.
    3. ALS-FRS > 20
    4. Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life.
    5. Patients of less than 3 years after the onset of ALS.
    6. Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend)
    7. If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation
    E.4Principal exclusion criteria
    1. Received treatment with other experimental therapies within the last 30 days prior to the first dose (for double-blind part of the study only)
    2. Previously received treatment with RT001 (for double-blind part of the study only)
    3. Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)
    4. SVC < 70 at screening
    5. Subject has a feeding tube or the need for a feeding tube is anticipated for the duration of the trial.
    6. Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period
    7. Evidence of any clinically significant neurological disorder other than ALS
    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. The subject has a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect
    10. Subject has had a significant illness or infection requiring medical intervention in the past 30 days (for double-blind part of the study only)
    11. Female who is breastfeeding or has a positive pregnancy test
    12. Male participant or female participant of childbearing potential, who is sexually active and unwilling/unable to use a medically acceptable and effective birth control method throughout the study
    13. 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
    14. History, within the last 2 years, of alcohol abuse or physical opioid dependence
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoints:
    Change from baseline in the ALSFRS-R total score at 24 weeks of the double-blind treatment period
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks (Day 168) of the double-blind treatment period
    E.5.2Secondary end point(s)
    Secondary endpoints are:
    - Time to Death or a Specified State of Disease Progression (composite of death, loss of independent ambulation, decline of ALSFRS-R from baseline of more than 6 points, loss of upper arm function, need for tracheostomy, use of respirator, or use of tube feeding)
    - Change from Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40)
    - Change from baseline in SVC

    Exploratory endpoints
    Double-blind part of the study:
    - Change from Baseline in the Clinical Global Impression of Change (CGI-C) scale
    - Change from Baseline in the Clinical Global Impression of Severity (CGI-S)
    - Change from Baseline in evaluation of upper- and lower-limb muscle strength using hand-held dynamometry (HHD)
    Open-label part of the study:
    Change from baseline in ALSFRS-R at 24 weeks of the extension treatmen period

    Safety endpoints
    - Safety will be evaluated by AEs, physical examination results, vital signs, ECG findings, and clinical laboratory test results. All descriptive statistics will be presented by treatment group.
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 weeks (Day 168) 24 weeks (Day 168) of the double-blind treatment period and at 24 weeks of the extension treatment period
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    The study will be concluded when the final subject completes the telephone follow-up visit 30 days after the final dose of RT001.
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 14
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 44
    F.4.2.2In the whole clinical trial 44
    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 will be offered to eligible subjects at the completion of the Randomized period. The blind will be maintained for all subjects during the Open-Label extension until the data-lock when the last subject enrolled completes the randomized period.
    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 Decision2021-02-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-06-28
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