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    Summary
    EudraCT Number:2017-004903-33
    Sponsor's Protocol Code Number:IBM4809
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-04-05
    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 ConcernedUK - MHRA
    A.2EudraCT number2017-004903-33
    A.3Full title of the trial
    Phase 2/3 Study of Arimoclomol in Inclusion Body Myositis (IBM)
    A Randomized, Double-blind, Placebo-Controlled Trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Arimoclomol in patients with Inclusion Body Myositis (IBM)
    A.4.1Sponsor's protocol code numberIBM4809
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02753530
    A.5.4Other Identifiers
    Name:IND NumberNumber:076773
    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 SponsorOrphazyme A/S
    B.1.3.4CountryDenmark
    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 supportOrphazyme A/S
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOrphazyme A/S
    B.5.2Functional name of contact pointClincial Department
    B.5.3 Address:
    B.5.3.1Street AddressOle Maaløes Vej 3
    B.5.3.2Town/ cityCopenhagen
    B.5.3.3Post codeN DK-2200
    B.5.3.4CountryDenmark
    B.5.4Telephone number+454061 3043
    B.5.6E-mailcsu@orphazyme.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/3/16/1659
    D.3 Description of the IMP
    D.3.1Product nameArimoclomol
    D.3.2Product code BRX-345
    D.3.4Pharmaceutical form Capsule, hard
    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 INNARIMOCLOMOL
    D.3.9.1CAS number 368860-21-3
    D.3.9.2Current sponsor codeBRX-345
    D.3.9.3Other descriptive nameArimoclomol citrate
    D.3.9.4EV Substance CodeSUB187159
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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, hard
    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
    Sporadic Inclusion Body Myositis (sIBM)
    E.1.1.1Medical condition in easily understood language
    progressive weakening of the muscles
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10075052
    E.1.2Term Sporadic inclusion body myositis
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of arimoclomol at a daily dosage of 1200 mg (400 mg t.i.d) compared to placebo in the treatment of sporadic IBM at 20 months.

    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM.1 (as per Appendix 1 of the protocol)
    2. Demonstrate being able to arise from a chair without support from another person or device
    3. Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, cane, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
    4. Age at onset of weakness > 45 years
    5. Body weight of > 40 kgs
    6. Able to give informed consent
    E.4Principal exclusion criteria
    1. History of any of the following excludes subject participation in the study: chronic infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer less than five years prior, or other chronic serious medical illnesses.
    2. Presence of any of the following on routine blood screening: WBC < 3000; platelets < 100,000; hematocrit < 30%; BUN > 30 mg; creatinine > 1.5 x upper limit of normal; symptomatic liver disease with serum albumin < 30mg/dl
    3. History of most recent creatine kinase >15x the upper limit of normal without any other explanation besides IBM.
    4. History of non-compliance with other therapies.
    5. Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
    6. Coexistence of any other disease that would likely to affect outcome measures.
    7. Drug or alcohol abuse within past three months. Patient has recent history (within 6 months before Screening) of chronic alcohol or drug abuse which may compromise the patient’s safety or ability to participate in study activities. Cannabis for IBM symptoms will be allowed (where legal).
    8. Participation in a recent drug study in the last 30 days prior to the screening visit or use of biologic agents less than 6 months prior to the screening visit.
    9. Women who are lactating or pregnant, or sexually active female subjects of child-bearing potential* intending to become pregnant or unwilling to use a highly effective method of contraception** during the trial through 1 month after the last dose of trial medication. Sexually active males with female partners of child-bearing potential* unwilling to use a condom with or without spermicide in addition to the birth control used by their partners during the trial until 3 months after the last dose of trial medication unless surgically sterile (vasectomy).
    Non child-bearing potential is defined as post-menopausal (minimum of 12 months with no menses and follicle-stimulating hormone in the post-menopausal range) or sterilisation (hysterectomy, oophorectomy, or bilateral tubal ligation).
    ** Highly effective methods of contraception include combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion; and vasectomised partner.
    According to the recommendations from the Clinical Trial Facilitation Group (CTFG, 2014), sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the trial until 1 month after the last dose of trial medication (for female subjects of child-bearing potential) and for 3 months after the last dose of trial medication (for male subjects with female partners of child-bearing potential). The reliability of sexual abstinence needs to be evaluated by the investigator in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject.
    10. Participants taking >7.5 mg prednisolone or equivalent or participants on intravenous immunoglobulin (IVIg) or other immunosuppressants within the last 3 months. Topical, nasal, and ocular corticosteroids are allowed unless they are being widely applied or the severity of the underlying condition makes them unsuitable in the Investigator’s opinion. Local steroid injections are allowed.
    11. Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment (results ≥3 times the upper limit of normal [ULN] for alanine aminotransferase combined with bilirubin ≥2 times the ULN; symptomatic liver disease with serum albumin < 3 g/dL; or creatinine ≥1.5 times the ULN). Laboratory tests may be repeated once at screening. Reasons to repeat laboratory tests may include that the medication causing laboratory abnormality was suspended, any other suspected cause may no longer exist, or to rule out laboratory error.
    E.5 End points
    E.5.1Primary end point(s)
    Primary efficacy endpoint is the change from baseline to Month 20 in the IBM Functional Rating Scale (IBMFRS) total score.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy endpoint will be checked at Visit at Month 20

    E.5.2Secondary end point(s)
    Secondary efficacy endpoints will include changes from baseline over months 12 to 20 in the following measures of strength and function:
    • IBMFRS total score (12 months)
    • 6 minute walk test with 2 minute distance captured
    • Modified Timed Up and Go (mTUG)
    • Muscle Strength Testing
    o Manual Muscle Testing (MMT)
    o Isometric Contraction Testing of bilateral quadriceps strength using the MicroFET
    • Health Assessment Questionnaire (HAQ-DI)
    • Grip strength using the Jamar device
    • SF-36
    • Falls and near falls
    • Patient Global Impression of Severity (PGIS) and Patient Global Impression of Change (PGIC)
    • Clinician Global Impression of Severity (CGIS) and Clinician Global Impression of Change (CGIC)
    • Patient Global Impression of Severity (PGIS) and Patient Global Impression of Change (PGIC)

    Safety endpoints:
    • Adverse events (AEs);
    • Hematology;
    • Clinical chemistry;
    • Vital signs;
    • Columbia Suicide Severity Rating Scale (C SSRS)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoints will be checked as follows:
    • IBMFRS total score: at every visit after the screening visit
    • 6 minute walk test with 2 minute distance captured: at every clinical visit
    • Modified Timed Up and Go (mTUG): at every clinical visit
    • Muscle Strength Testing: at every clinical visit
    • Health Assessment Questionnaire (HAQ-DI): at every clinical visit
    • Grip strength using the Jamar device: at every clinical visit
    • SF-36: at every clinical visit
    • Falls and near falls: at every clinical visit
    • PGIS and CGIS at all site visits,
    • PGIC and CGIC at all post-baseline site visits (no PGI/CGI at Month1/Visit 3 or Month 2/Visit 4).

    Safety endpoints will be checked at every visit
    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 Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    biomarker CN1A Ab testing and biobanking for future analyses.
    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) 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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
    United Kingdom
    United States
    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
    Last visit of the last subject
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days8
    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: 75
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 75
    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 state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 150
    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)
    The patients will continue the standard of care.
    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 Decision2018-05-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-18
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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