Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2018-003618-41
    Sponsor's Protocol Code Number:MOM-M281-005
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2019-05-13
    Trial results View 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 number2018-003618-41
    A.3Full title of the trial
    An Open-label Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients with Generalized Myasthenia Gravis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    To further evaluate the efficacy, safety and tolerability of M281 injection for the treatment of patients with Myasthenia gravis, a neuromuscular disease
    A.4.1Sponsor's protocol code numberMOM-M281-005
    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 SponsorMomenta Pharmaceuticals, 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 supportMomenta Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMomenta Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address301 Binney Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    B.5.3.4CountryUnited States
    B.5.4Telephone numberNA
    B.5.5Fax numberNA
    B.5.6E-mailclinicaltrialinfo@momentapharma.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 nameM281
    D.3.2Product code M281
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN/A
    D.3.9.2Current sponsor codeM281
    D.3.9.3Other descriptive nameM281
    D.3.9.4EV Substance CodeSUB182674
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Generalized Myasthenia gravis
    E.1.1.1Medical condition in easily understood language
    Myasthenia gravis (a neuromuscular 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 21.1
    E.1.2Level PT
    E.1.2Classification code 10028417
    E.1.2Term Myasthenia gravis
    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
    To evaluate the long-term safety and tolerability of M281 in patients with generalized myasthenia gravis (gMG)
    E.2.2Secondary objectives of the trial
    To evaluate the long-term efficacy of M281, long term immunogenicity of M281 and long-term pharmacodynamic (PD) activity of M281
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Has completed the M281 POC study (ie, participated in the POC study without discontinuing study drug for reasons other than the need for rescue therapy as specified in the POC protocol, and completed the protocol-specified 8 weeks of follow-up after the last dose of study drug) and had no major eligibility deviations or other major protocol deviations in the POC study. If the patient received rescue therapy during the POC, the Medical Monitor should be consulted before the patient is enrolled in the OLE study.
    2. Has sufficient venous access to allow drug administration by infusion and blood sampling as per this OLE protocol.
    3. Is up to date on all age-appropriate vaccinations as per routine local medical guidelines.
    4. Women of childbearing potential, defined as women physiologically capable of becoming pregnant, must have a negative urine pregnancy test predose on OLE study Day 1. Menopausal women who did not have elevated follicle-stimulating hormone (FSH) at the time of enrollment in the POC study must also have a negative urine pregnancy test predose on OLE study Day 1.
    5. Women of childbearing potential (including menopausal women who did not have elevated FSH at the time of enrollment in the POC study) must agree to remain totally abstinent (ie, refrain from sexual intercourse during the study) or to consistently use a reliable and highly effective method of contraception (eg, condom plus diaphragm, condom plus spermicide, diaphragm plus spermicide, or intrauterine device or oral/injectable/implanted hormonal contraceptive used in combination with an additional barrier method) during the OLE study and for 30 days after the last M281 infusion. Note: Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception. Female condom and male condom should not be used together.
    6. Male patients must agree to remain totally abstinent (ie, refrain from sexual intercourse during the study) or to consistently use a reliable and highly effective method of contraception (eg, condom plus diaphragm, condom plus spermicide, diaphragm plus spermicide, or intrauterine device or oral/injectable/implanted hormonal contraceptive used in combination with an additional barrier method) to avoid pregnancy of the patient’s partner(s) during the OLE study and for 100 days following the last M281 infusion, unless the patient provides documentation of a vasectomy at least 6 months prior to OLE study enrollment. Male patients must also abstain from sperm donation during the OLE study and for 100 days following the last M281 infusion. Note: Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception. Female condom and male condom should not be used together.
    7. A patient using herbal, naturopathic, and traditional Chinese remedies and ayurvedic and nutritional supplements is eligible if the use of these medications is acceptable to the Investigator. These remedies should be at a stable dose and regimen using the same preparation since enrollment in the POC study.
    8. Is able to understand and voluntarily provide written informed consent to participate in the long-term OLE study and comply with all study procedures for duration of the OLE study.
    E.4Principal exclusion criteria
    1. Has Myasthenia Gravis Foundation of America (MGFA) Class IVb or V disease.
    2. Met any of the study drug stopping criteria in the POC study or discontinued study drug in the POC study for any reason (eg, adverse event [AE]) other than the need for rescue therapy as specified in the POC protocol. (If the patient received rescue therapy during the POC, the Medical Monitor should be consulted before the patient is enrolled in the OLE study.)
    3. Has current suicidal ideation evidenced by a "yes" response to questions 4 or 5 in the Suicidal Ideation section of the Columbia-Suicide Severity Rating Scale (C-SSRS) administered predose on OLE study Day 1.
    4. Currently has a serious or clinically significant infection (eg, pneumonia, biliary tract infection, diverticulitis, Clostridium difficile infection) requiring parenteral anti-infectives and/or hospitalization. Note: An uncomplicated, presumably viral, upper respiratory tract infection (eg, ‘the common cold’) is not an exclusion.
    5. Has current alcohol/substance abuse/dependence, or in the Investigator’s opinion, shows evidence of ongoing alcohol/substance abuse/dependence.
    6. Has donated or had significant loss of whole blood (480 mL or more) within 30 days, or plasma within 14 days prior to OLE study Day 1.
    7. Has a hypersensitivity to M281 or any constituent of the study drug solution.
    8. Has had a prior severe drug reaction that included shock or severe hypersensitivity.
    9. Has liver impairment with Child-Pugh Class B or C.
    10. Has any other medical condition(s) likely to require treatment with oral or parenteral glucocorticosteroids (eg, severe asthma), or has required oral or parenteral glucocorticosteroids in the past 3 months before OLE study Day 1 for conditions other than MG (inhalational, intra-articular, topical or ocular glucocorticosteroids are not exclusionary), or taking any immunosuppressive agents not being used to treat MG.
    11. Is receiving a systemic biologic antibody for any concurrent disease.
    12. Has a QT interval corrected for heart rate (QTc) at OLE study Day 1 of >450 msec for males or >470 msec for females; or QTc >480 msec in patients with Bundle Branch Block, by the Fridericia formula.
    13. Had any of the following abnormal laboratory values based on the safety laboratory tests done at the Day 85 POC Visit: aspartate aminotransferase (AST), alanine aminotransferase (ALT), or alkaline phosphatase ≥2× upper limit of normal (ULN); or bilirubin ≥1.5×ULN (isolated bilirubin greater than 1.5×ULN is acceptable if bilirubin is fractionated and direct bilirubin is <35%, or if there is a prior diagnosis of Gilbert disease without any condition that causes elevation of bilirubin).
    14. Had elevated CK ≥2×ULN based on the safety laboratory tests done at the Day 85 POC Visit.
    Note: If the CK value is ≥2×ULN at the Day 85 POC visit, the value from the Day 113 POC Visit can be used to assess eligibility. If the value from the Day 113 POC Visit is also ≥2×ULN, but <5×ULN, and in the opinion of the investigator the patient has no CK related disease and CK level is believed to be the result of non-pathologic factors, the CK may be repeated once within 28 days and if stable/improved the patient can be enrolled.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and Tolerability of M281 will be evaluated in terms of the Incidence and severity of adverse events (AEs; including serious AEs [SAEs], and AEs of special interest [AESIs]), vital signs, physical examinations, clinical laboratory testing (including chemistry, hematology, coagulation, and urinalysis), electrocardiograms (ECGs), and the Columbia-Suicide Severity Rating Scale (C-SSRS)
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs are monitored throughout the study
    Vital signs will be measured immediately prior to the start of each infusion and 30 minutes after the first infusion and second infusions only.
    Full physical examinations to be performed at study enrollment and end of treatment/early termination, and a focused physical examination at all other site visits
    clinical laboratory testing (including chemistry, hematology, coagulation, and urinalysis) will be done on day 1, week 2,4,8,12 and every 12 weeks thereafter
    ECG will be taken immediately prior to the beginning of the infusion and right after the end of the infusion
    C-SSRS will be administered at the enrollment and then on week 2,4,8,12 and every 12 weeks thereafter
    E.5.2Secondary end point(s)
    1. Change from baseline in the total Myasthenia Gravis – Activities of Daily Living (MG-ADL) score over time
    2. Number of patients with a 2-, 3-, 4-, 5-, 6-, 7-, or ≥8-point improvement in total MG-ADL score over time
    3. Change from baseline in total Quantitative Myasthenia Gravis (QMG) score over time
    4. Change from baseline in total Revised Myasthenia Gravis Quality of Life – 15 Scale (MG-QoL15r) score over time
    5. Change from baseline in Clinical Global Impression of Severity (CGI-S) over time and Clinical Global Impression of Improvement (CGI-I) ratings
    6. Change from baseline in Myasthenia Gravis Foundation of America (MGFA) classification over time
    7. Incidence of anti-drug antibody (ADA) and neutralizing ADA (nADA) seroconversion over time
    8. Total serum immunoglobulin (Ig)G concentrations over time and change from baseline
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. week 4 and 8, week 12 and every 12 weeks thereafter, EoT visit and on follow- up visit
    2. week 4 and 8, week 12 and every 12 weeks thereafter, EoT visit and on follow- up visit
    3. week 4 and 8, week 12 and every 12 weeks thereafter, EoT visit and follow -up visit
    4. week 4 and 8, week 12 and every 12 weeks thereafter, EoT visit and on follow up visit
    5. baseline, week 4 and 8, week 12 and every 12 weeks thereafter, EoT visit and on follow up visit
    6. baseline, week 8, and every 24 weeks thereafter and EoT visit
    7. week 4 and 8, week 12 and every 12 weeks thereafter, EoT visit and on follow up visit
    8. week 2, 4, 8, 12 and every 12 weeks thereafter, EoT visit and on follow up 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 No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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
    tolerability, immunogenicity and biomarkers
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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 States
    Belgium
    Germany
    Italy
    Poland
    Spain
    United Kingdom
    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
    On 09-Dec-2020 the Sponsor made the decision not to recommence the MOM-M281-005 study and to move forward with study closure.
    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 months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    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: 16
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 46
    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)
    patient will continue their treatment per physician's discretion.
    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 Decision2019-07-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-25
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-12-09
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Thu Apr 25 11:13:41 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA