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    Summary
    EudraCT Number:2022-000389-16
    Sponsor's Protocol Code Number:1821-FSH-301
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-07-13
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2022-000389-16
    A.3Full title of the trial
    A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 study to determine the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy
    A.4.1Sponsor's protocol code number1821-FSH-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05397470
    A.5.4Other Identifiers
    Name:INDNumber:138739
    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 SponsorFulcrum Therapeutics
    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 supportFulcrum Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFulcrum Therapeutics, Inc.
    B.5.2Functional name of contact pointMarie-Helene Jouvin
    B.5.3 Address:
    B.5.3.1Street Address26 Landsdowne Street, 5th floor
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1617678 4980
    B.5.6E-mailmjouvin@fulcrumtx.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/3/20/2263
    D.3 Description of the IMP
    D.3.1Product nameLosmapimod
    D.3.2Product code FTX-1821
    D.3.4Pharmaceutical form Tablet
    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 INNLosmapimod
    D.3.9.1CAS number 585543-15-3
    D.3.9.2Current sponsor codeFTX-1821
    D.3.9.4EV Substance CodeSUB189237
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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 placeboTablet
    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
    Facioscapulohumeral Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Muscular Disease
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10064087
    E.1.2Term Facioscapulohumeral muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A:
    To evaluate the efficacy of losmapimod for the treatment of FSHD by demonstrating slowing of disease progression assessed by RWS quantification of RSA Q1-Q5 with 500 g wrist weight in the dominant arm.

    Part B:
    To assess the long-term safety and tolerability of losmapimod in patients with FSHD
    E.2.2Secondary objectives of the trial
    Part A:
    1. To evaluate the change in Neuro-QoL UE relative to placebo
    2. To evaluate PGIC relative to placebo
    3. To evaluate efficacy of losmapimod to slow accumulation of fat in muscle by MFI with WB MSK MRI relative to placebo
    4. To assess safety and tolerability of losmapimod in patients with FSHD

    Part B: No Secondary objectives
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Part A:
    1. Patient will sign and date an informed consent form (ICF).
    2. Patients will have a diagnosis of FSHD1 or FSHD2 verified by genetic testing
    − Randomization will be stratified for FSHD1 to ensure that treatment allocation is balanced across FSHD repeat number categories (ie, 1 to 3 repeats versus 4 to 9 repeats).
    − Randomization will be stratified for FSHD2 to ensure that an equal number of patients will be allocated to treatment and placebo.
    3. Patients will have a Clinical Severity Score of 2 to 4 (Ricci score; range 0 to 5) at screening. Patients who are wheelchair-dependent or dependent on walker or wheelchair for activities are not permitted to enroll in the study.
    4. Patients with screening total RSA (Q1-Q4) without weight in the dominant UE assessed by RWS ≥ 0.2 and ≤ 0.7.
    5. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
    6. No contraindications to MRI.
    7. Patients (male and female) will be between the ages of 18 and 65 years at the time of consent, inclusive.
    − A female patient is eligible to participate if she is of non-child bearing potential, defined as pre-menopausal females with a documented tubal ligation or hysterectomy; bilateral salpingectomy, or bilateral oophorectomy; if she is postmenopausal, defined as no menses for 12 months without an alternative medical cause; OR
    − if of child-bearing potential, she is using a highly effective method for avoidance of pregnancy for the duration of dosing and until 90 days after the last dose. The decision to include or exclude women of childbearing potential may be made at the discretion of the Investigator and in accordance with local practice in relation to adequate contraception.
    − Male patients must agree to use one of the contraception methods. This criterion must be followed from the time of the first dose of study medication and until 90 days after the last dose of study drug.

    Part B:
    1. Patient completed 48 weeks of treatment during Part A.
    2. Patient will sign and date an ICF.
    3. Patient is willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and
    other study procedures.
    4. Patients agree to the following methods of contraception:
    - Female patients of childbearing potential agree to continue using a highly effective method for avoidance of pregnancy for the duration of
    dosing and until 90 days after the last dose. For details regarding contraception requirements. The decision to include or exclude women
    of childbearing potential may be made at the discretion of the Investigator and in accordance with local practice in relation to adequate contraception.
    - Male patients must agree to use one of the contraception methods listed in Section 5.5.1 of the protocol. This criterion must be followed
    from the time of the first dose of study medication and until 90 days after the last dose of study drug.
    E.4Principal exclusion criteria
    Part A:
    1. Hx of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study
    drug to the patient.
    2. Previously diagnosed cancer that has not been in complete remission for at least 5 years. Localized carcinomas of the skin and carcinoma in
    situ of the cervix that have been resected or ablated for cure are not exclusionary.
    3. For patients who are on drug(s) or supplements that may affect muscle function or that are included in the list of drugs presented in Appendix 2 of the Protocol: pt must be on a stable dose of that drug(s) or supplement for at least 3 months. If the drug(s) or supplements that
    may affect muscle function as determined by the Investigator are also CYP3A4 (orally administered), MATE, or OAT3 substrates, they are not permitted as concomitant therapy, regardless of whether patients are on a stable dose.
    4. Orally administered CYP3A4 substrates and MATE and OAT3 substrates are not permitted as concomitant during the administration of
    5. History of febrile illness within 5 days before the first study drug dose
    6. Known active opportunistic or life-threatening infections including HIV and hepatitis B or C
    7. Known active or inactive tuberculosis infection.
    8. Current acute liver disease or chronic liver disease as defined by any of the following: current ALT ≥2 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN (unless participant has Gilbert's syndrome characterized by the combination of total bilirubin < 3 × ULN, direct bilirubin within the normal range and normal ALT and AST, or the presence of mutations in the UDP-glucuronosyltransferase 1 gene,
    indicative of Gilbert's syndrome); or Positive for hepatitis B or surface antigen; or Positive for hepatitis C antibody unless additional testing for
    hepatitis C viral RNA is negative, ALT is < 2 × ULN and total bilirubin is ≤ 1.5 × ULN, indicating inactive/resolved hepatitis C infection
    9. Known severe renal impairment (defined as a glomerular filtration rate of < 30 mL/min/1.73 m2).
    10. Standard 12-lead ECG demonstrating QTcF >450 msec for male patients and QTcF >470 msec for female patients at screening. If QTcF
    exceeds 450 msec for males or 470 msec for females, the ECG will be repeated 2 more times, and the average of the 3 QTcF values will be used to determine the patient's eligibility.
    11. History of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ECGs
    12. Male patients with a female partner who is planning to become pregnant during the study or within 90 days after the last study drug dose
    13. Concomitant use of cytotoxic chemotherapy for cancer or known ongoing or anticipated use of chronic severe immunosuppressive agents.
    14. Positive pregnancy test or known to be pregnant or lactating or planning to become pregnant during study drug administration and until 90 days after last dose
    15. Any current mental condition (psychiatric disorder, senility or dementia)
    16. Patient has any condition possibly affecting drug absorption
    17. History of alcohol, analgesic/opioid, and/or illicit drug abuse, as defined by the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (American Psychiatric Association, 2013), in the last 6 months before screening or a positive test for drugs of abuse at screening. Use of CBD/THC is permitted
    18. Use of another IP within 30 days or 5 half-lives
    19. Current or anticipated participation in a natural hx study.
    20. Known hypersensitivity or intolerance to losmapimod or any of its excipients
    21. Previous participation in a Fulcrum-sponsored FSHD losmapimod study
    22. Anticipated inability to comply with any study procedures, study visits according to the visit schedule through 48 weeks.
    23. Abnormal laboratory results indicative of any significant medical disease
    24. Pt, or close relative of the patient to staff directly involved with the conduct of the study
    25.Pt is vulnerable (i.e., deprived of freedom), including inmates of psychiatric wards and prison or state institutions, patients with commitments to an institution, or a patient who is detained or committed to an institution by a law court or by legal authorities.

    Part B:
    1. Any clinical condition that, in the opinion of the Investigator, might confound the results of the study or pose an additional risk in administering study drug to the patient.
    2. Orally administered CYP3A4 substrates and MATE and OAT3 substrates are not permitted as concomitant therapy during the administration of losmapimod (defined as baseline visit through end of study treatment). Refer to Appendix 3 for lists of CYP3A4, MATE, and OAT3 substrates.
    3. Male patients with a female partner who is planning to become pregnant during the study or within 90 days after the last study drug dose.
    4. Anticipated inability to comply with any study procedures, including participation in study visits.
    E.5 End points
    E.5.1Primary end point(s)
    Part A:
    Change from baseline compared to placebo in total RSA Q1-Q5 with 500 g wrist weight in dominant arm at Week 48

    Part B:
    Safety and tolerability of long-term treatment with losmapimod, based on the assessment of AEs, clinical laboratory tests, ECGs, vital signs, and physical examinations
    E.5.1.1Timepoint(s) of evaluation of this end point
    RSA Q1-Q5 with 500 g wrist weight in dominant arm at Week 48
    E.5.2Secondary end point(s)
    Part A:
    1. Change from baseline compared to placebo in Neuro-QoL UE at Week 48
    2. Change from baseline compared to placebo in PGIC at Week 48
    3. Change from baseline compared to placebo in WB longitudinal composite MFI of B muscles at Week 48
    4. Safety and tolerability, based on the assessment of AEs, clinical laboratory tests, ECGs, vital signs and physical examinations.

    Part B: No secondary endpoints
    E.5.2.1Timepoint(s) of evaluation of this end point
    At Week 48 and throughout the study
    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 Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 EEA16
    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
    Canada
    United Kingdom
    United States
    Denmark
    France
    Germany
    Italy
    Netherlands
    Spain
    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
    The end of the study is defined as the date on which the last patient completes the last visit (includes the safety follow-up visit).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days6
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months9
    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: 260
    F.1.3Elderly (>=65 years) No
    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 state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 144
    F.4.2.2In the whole clinical trial 260
    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)
    All patients who discontinue from treatment early or who choose not to rollover into Part B will return for a complete safety follow-up visit 7±3 days after the last dose of study drug and will complete a safety follow-up phone screen 30±5 days after the last dose of study drug. Patients may continue to receive study drug in Part B until after commercial drug is available post regulatory approval or until the study is discontinued by the sponsor.
    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 Decision2022-08-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-08-26
    P. End of Trial
    P.End of Trial StatusOngoing
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