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    Summary
    EudraCT Number:2021-006511-29
    Sponsor's Protocol Code Number:PTC857-CNS-001-ALS
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-11-11
    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 ConcernedSweden - MPA
    A.2EudraCT number2021-006511-29
    A.3Full title of the trial
    A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL STUDY TO ASSESS THE EFFICACY, SAFETY, TOLERABILITY, PK, AND BIOMARKER EFFECTS OF PTC857 IN ADULT SUBJECTS WITH AMYOTROPHIC LATERAL SCLEROSIS (CARDINALS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2 study of PTC857 in patients with Amyotrophic Lateral Sclerosis
    A.3.2Name or abbreviated title of the trial where available
    CARDINALS
    A.4.1Sponsor's protocol code numberPTC857-CNS-001-ALS
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05349721
    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 SponsorPTC Therapeutics, 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 supportPTC Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPTC Therapeutics, Inc.
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address100 Corporate Court
    B.5.3.2Town/ citySouth Plainfield
    B.5.3.3Post codeNJ 07080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1908 2227000
    B.5.6E-mailmedinfo@ptcbio.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/22/2728
    D.3 Description of the IMP
    D.3.1Product nameutreloxastat
    D.3.2Product code PTC857 60 mg/ml oral solution
    D.3.4Pharmaceutical form Oral solution
    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 INNutreloxastat
    D.3.9.1CAS number 1213269-96-5
    D.3.9.2Current sponsor codePTC857
    D.3.9.3Other descriptive namePTC857
    D.3.9.4EV Substance CodeSUB210688
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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 placeboOral solution
    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 (ALS)
    E.1.1.1Medical condition in easily understood language
    Amyotrophic Lateral Sclerosis is a type of Motor Neurone Disease. It is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control.
    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
    To evaluate the efficacy of PTC857 in reducing disease progression in subjects with ALS.
    E.2.2Secondary objectives of the trial
    Secondary Objectives:
    1. Safety and tolerability of PTC857 in subjects with ALS
    2. Respiratory function in subjects randomized to PTC857 vs placebo
    3. Motor/limb and bulbar function in subjects randomized to PTC857 vs placebo
    4. Neuropsychological function in subjects randomized to PTC857 vs placebo
    5. Survival in subjects randomized to PTC857 vs placebo
    6. Quality of life via ALSQ-40 in subjects randomized to PTC857 vs placebo
    7. PK of PTC857

    Expl. Objectives:
    1. Effects on biomarker activity in subjects randomized to PTC857 vs placebo
    2. Quality of life via EQ-5D-5L in subjects randomized to PTC857 vs placebo

    Long-Term Extension Period Objectives:
    1. Disease progression, survival, neuropsychological function, respiratory function, motor/limb and bulbar function, and effects on biomarker activity upon long-term treatment with PTC857
    2. Safety and tolerability upon long-term treatment with PTC857
    3. Quality of life upon long-term treatment with PTC857
    4. PK of PTC857
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Pharmacokinetics sub-study
    E.3Principal inclusion criteria
    1. Males or females aged between 18 and 80 years at the time of the initial Screening visit

    2. ALS with preserved function, defined as:
    a. Onset of the first symptom leading to the diagnosis of ALS ≤24 months at the time of the initial Screening Visit
    b. Revised El Escorial criteria of either:
    (i) Clinically definite ALS
    (ii) Clinically probable ALS

    3. A total ALSFRS-R score of at least 34 at the start of the Screening Period

    4. No significant respiratory compromise as evidenced by slow vital capacity ≥60% at the start of the Screening Period

    5. All chronic concomitant medications (both prescription and over the counter [OTC]) and non pharmacologic therapy regiments, excluding standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, and non pharmacologic therapy regimens should be stable and unchanged from at least 14 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study

    6. Female subjects must have a negative breast cancer imaging screening status (not considered clinically abnormal and/or requiring further evaluation/treatment) within 6 months prior to the Screening Visit or during the Screening Period.

    7. Standard-of-care therapy for the treatment of ALS (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol) should be stable and unchanged from 30 (-3) days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study
    E.4Principal exclusion criteria
    1. Females who are pregnant or nursing or plan to become pregnant during the study
    2. Subjects with clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular/ischemic disease or any other condition that, in the opinion of the investigator, would jeopardize the safety of the subject or impact the validity of the study results
    3. Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the investigator or the medical monitor, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject
    4. Current participation in any other investigational study with an investigational product or participation within 30 days prior to the start of the Screening Period or 5 half-lives of the previously taken investigational drug, whichever is longer
    5. Subject has previously received PTC857
    6. Subject is receiving a combination of edaravone and sodium phenylbutyrate/taurursodiol treatment, where applicable, within 30 (-3) days prior to the start of the Screening Period
    7. For female subjects, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) in the Intent-to-Treat 1 (ITT1) Analysis Set after 24 weeks of treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks
    E.5.2Secondary end point(s)
    Secondary Endpoints:
    1. Change from baseline in ALSFRS-R in the Intent-to-Treat 2 (ITT2) Analysis Set after 24 weeks of treatment
    2. Safety and tolerability of PTC857 as measured by the severity and number of TEAEs and TESAEs, and change in clinical laboratory tests, physical examination, vital signs, Columbia-Suicide Severity Rating Scale (C-SSRS), and 12-lead electrocardiograms (ECGs) during the Treatment Period
    3. Change from baseline in respiratory function as assessed by pulmonary function tests (PFTs) after 24 weeks of treatment
    4. Change from baseline in motor/limb and bulbar function as assessed by the Modified Norris Scale after 24 weeks of treatment
    5. Change from baseline in neuropsychological function as assessed by the ALS Cognitive Behavioral Screen (ALS CBS) after 24 weeks of treatment
    6. Survival as assessed by rate of and length of time to needing respiratory support/intubation and/or death
    7. Survival and functional change as assessed by the Combined Assessment of Function and Survival (CAFS) after 24 weeks of treatment
    8. Quality of life as assessed by Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) after 24 weeks of treatment
    9. Plasma PK and cerebrospinal fluid (CSF) exposure of PTC857

    Exploratory Endpoints:
    1. Change from baseline in blood, urine, and CSF biomarker activity after 24 weeks of treatment
    2. Quality of life as assessed by the EQ-5D-5L after 24 weeks of treatment

    Long-Term Extension Endpoints:
    1. Severity and number of TEAEs and TESAEs, change in clinical laboratory tests, physical examination, vital signs, and 12-lead ECGs during the long-term treatment
    2. Change from baseline in ALSFRS-R after I 52 weeks of treatment
    3. Change from baseline in respiratory function as assessed by PFTs after 52 weeks of treatment
    4. Change from baseline in motor/limb and bulbar function as assessed by the Modified Norris Scale after 52 weeks of treatment
    5. Change from baseline in neuropsychological function as assessed by the ALS CBS after 52 weeks of treatment
    6. Survival and functional change as assessed by CAFS after 52 weeks of treatment
    7. Time to needing respiratory support/intubation and/or death with long-term treatment
    8. Quality of life as assessed by ALSAQ-40 after 52 weeks of treatment
    9. Change from baseline in blood and urine biomarker activity after 52 weeks of treatment
    10. Change from baseline in blood and urine biomarker activity after 52 weeks of treatment
    11. PK of PTC857

    Continued Long-Term Extension Period Endpoints:
    1. Severity and number of TEAEs and TESAEs during the Continued LTE Period
    2. Change from baseline in ALSFRS-R after 160 weeks of treatment
    3. Survival after 160 weeks of treatment
    4. Time to needing respiratory support/intubation and/or death after 160 weeks of treatment
    5. Quality of life as assessed by ALSAQ-40 after 160 weeks of treatment
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary and Exploratory Endpoints: 24 weeks
    Long-Term Extension Period Endpoints: 52 weeks
    Continued Long-Term Extension Period Endpoints: 160 weeks
    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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA43
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Brazil
    Japan
    Mexico
    United States
    Belgium
    Czechia
    France
    Germany
    Ireland
    Italy
    Netherlands
    Norway
    Poland
    Spain
    Sweden
    E.8.7Trial has a data monitoring committee Yes
    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 - A subject is considered to have completed the study if she/he has completed all Part C study visits (or Part A if they are not participating in the optional LTE Period, or Part A and B if they are not participating in the optional continued LTE Period), including the follow-up telephone call.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months11
    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: 248
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    A small number of subjects may have lost, or may lose during the study, capacity to make their own decisions due to their disease (ALS). Where they cannot give consent personally, a legally authorized representative may be asked to provide consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 141
    F.4.2.2In the whole clinical trial 258
    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)
    None
    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 Decision2022-12-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-03-15
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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