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    Summary
    EudraCT Number:2022-000565-40
    Sponsor's Protocol Code Number:GLM101-002
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-08-29
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2022-000565-40
    A.3Full title of the trial
    A Phase 2, Randomized, Open-Label, 12-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 Administered Intravenously to Adult Participants with PMM2-CDG
    Estudio en fase II, aleatorizado, abierto, de 12 semanas de
    duración, para evaluar la farmacodinámica, seguridad,
    tolerabilidad y farmacocinética de dosis múltiples de
    GLM101 administrado por vía intravenosa a participantes
    adultos con PMM2-CDG
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Randomized, Open-Label, 12-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 Administered Intravenously to Adult Participants with PMM2-CDG
    Estudio en fase II, aleatorizado, abierto, de 12 semanas de
    duración, para evaluar la farmacodinámica, seguridad,
    tolerabilidad y farmacocinética de dosis múltiples de
    GLM101 administrado por vía intravenosa a participantes
    adultos con PMM2-CDG
    A.4.1Sponsor's protocol code numberGLM101-002
    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 SponsorGlycomine, 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 supportGlycomine, Inc
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGLycomine, Inc
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address733 Industrial Road,
    B.5.3.2Town/ citySan Carlos, California
    B.5.3.3Post code94070
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1650294-0439
    B.5.6E-mailtmoors@glycomine.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 numberEMA/OD/055/18
    D.3 Description of the IMP
    D.3.1Product nameGML 101
    D.3.2Product code GLM 101
    D.3.4Pharmaceutical form Solution for 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 INNalfa-D-Mannopyranosyl phosphate dipotassium
    D.3.9.1CAS number 71888-67-0
    D.3.9.2Current sponsor codeGLM101
    D.3.9.3Other descriptive namealfa-D-Mannopyranosyl phosphate dipotassium
    D.3.9.4EV Substance CodeSUB289363
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    PMM2-CDG
    PMM2-CDG
    E.1.1.1Medical condition in easily understood language
    genetic metabolic disorder
    trastorno metabólico genético
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10027426
    E.1.2Term Metabolic disorder NOS
    E.1.2System Organ Class 100000004861
    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 changes in coagulation and antithrombosis factors in adult participants with PMM2-CDG after 12 weeks of dosing
    Evaluar los cambios en la coagulación y los factores antitrombóticos en participantes adultos con PMM2-CDG después de 12 semanas de administración
    E.2.2Secondary objectives of the trial
    - To evaluate the potential PD activity of GLM101 in adult participants with PMM2-CDG after 12 weeks of dosing.
    - To assess the 12-week safety and tolerability of multiple doses of GLM101 in cohorts of participants with PMM2- CDG.
    - To assess the PK of GLM101 in participants with PMM2-CDG after 12 weeks of dosing
    - Evaluar la posible actividad FD de GLM101 en participantes adultos después de 12 semanas de administración del tratamiento
    - Evaluar la seguridad y tolerabilidad de 12 semanas de dosis múltiples de GLM101 en cohortes de participantes con PMM2-CDG
    - Evaluar la FC de GLM101 en participantes con PMM2-CDG después de 12 semanas de administración del tratamiento
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Is a male or female, 18 to 65 years of age, inclusive, at Screening;
    2. Has been diagnosed with PMM2-CDG with genetic test confirmation;
    3. Has antithrombin III (ATIII) levels below 80%;
    4. If the participant is a female of childbearing potential, she must not be pregnant (confirmed by a negative serum pregnancy test), is using a medically accepted method of contraception (abstinence, a hormonal contraceptive in conjunction with a barrier method, double-barrier method, or use of an intrauterine device), and must agree to continue using this method for 30 days after the last infusion of GLM101;
    5. If the participant is a female of non-childbearing potential, she must be pre-pubertal, surgically sterile, or must have an ovarian dysfunction confirmed by a follicle stimulating hormone > 40 IU/L;
    6. If the participant is a sexually active male with female partners, the sexually mature, nonsterile male participant agrees to use a medically acceptable method of contraception (abstinence, the partner taking a hormonal contraceptive in conjunction with a barrier method, double-barrier method, or use by the partner of an intrauterine device) and agrees to continue using this method for 30 days after the last infusion of GLM101. Males are considered surgically sterile if they have undergone bilateral orchiectomy or vasectomy at least 3 months prior to Screening;
    7. If the participant is male, he must agree to refrain from donating sperm during the study and 30 days after the last infusion of GLM101;
    8. Is willing and able to provide informed consent/assent, directly or through his/her legally authorized representative.
    1. hombres o mujeres de entre 18 y 65 años de edad, inclusive, en el momento de la selección;
    2. haberle sido diagnosticado PMM2-CDG con confirmación mediante pruebas genéticas;
    3. tener niveles de antitrombina III (ATIII) por debajo del 80 %;
    4. si la participante es una mujer con capacidad de concebir, no debe estar embarazada (confirmado mediante una prueba de embarazo en suero negativa); deben utilizar un método de regulación de la natalidad médicamente aceptado (abstinencia sexual, un anticonceptivo hormonal junto con un método de barrera, un método de doble barrera o utilizar un dispositivo intrauterino) y debe comprometerse a seguir utilizando este método hasta 30 días después de la última infusión de GLM101;
    5. en el caso de las participantes sin capacidad de concebir, deben ser prepuberales, quirúrgicamente estériles o tener una disfunción ovárica confirmada con niveles de
    hormona foliculoestimulante >40 UI/l;
    6. en el caso de los varones sexualmente activos con pareja femenina, el participante varón sexualmente maduro y no estéril debe comprometerse a utilizar un método de regulación de la natalidad médicamente aceptable (abstinencia sexual, su pareja utiliza un anticonceptivo hormonal junto con un método de barrera, un método de doble barrera o un dispositivo intrauterino) y seguir utilizándolo hasta 30 días después de la última infusión de GLM101. Se considera que un hombre es quirúrgicamente estéril si se ha sometido a orquiectomía bilateral o vasectomía al menos 3 meses antes de la selección;
    7. los participantes varones deben abstenerse de donar esperma durante el estudio y hasta 30 días después de la última infusión de GLM101;
    8. estar dispuesto/a y tener la capacidad para otorgar consentimiento/asentimiento informado, directamente o mediante representante legal.
    E.4Principal exclusion criteria
    1. Diagnosis of congenital disorder of glycosylation (CDG) other than PMM2;
    2. Has an active infection requiring parenteral antibiotics, antivirals, or antifungals or treatment with systemic steroids within 7 days prior to Screening;
    3. Has confirmed active coronavirus disease-2019 (COVID-19) infection or tests positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or check in to clinical site;
    4. Has a history of a severe allergic reaction to any drug or excipients of GLM101 (as listed in the GLM101 Investigator’s Brochure);
    5. Has a known history of poor venous access;
    6. Has a history of liver transplant;
    7. Has a history of drug or alcohol use disorder within 12 months from Screening;
    8. Has had a major surgical procedure within 30 days prior to Screening;
    9. Has Screening or eligibility confirmation laboratory value(s) outside the laboratory reference range considered clinically significant and not related to PMM2-CDG;
    10. If female, has a positive serum pregnancy test during Screening;
    11. Has serology positive for hepatitis B surface antigen or hepatitis C antibody during Screening;
    12. Has history or presence, upon clinical evaluation, of any illness that might impact the safety of GLM101 infusion or evaluability of drug effect based on the Principal Investigator’s and Medical Monitor’s discretion;
    13. Is currently participating in another interventional clinical study or has completed another clinical study with an investigational drug or device within 30 days or 5 half-lives before GLM101 infusion, except for acetazolamide. Participants may be enrolled and continue treatment with acetazolamide only if they are on a stable dose for at least 30 days prior to dosing with GLM101, and the dose remains unchanged for the duration of the study.
    1. tener diagnosticado trastorno congénito de la glucosilación (CDG) distinto de PMM2;
    2. tener una infección activa que requiera antibióticos, antivíricos o antifúngicos por vía parenteral o tratamiento con corticoesteroides sistémicos en los 7 días previos a
    la selección;
    3. tener infección activa por el coronavirus (la COVID-19) confirmada o resultados positivos por coronavirus del síndrome respiratorio agudo grave de tipo 2 (SARS-CoV-2) en el momento de la selección o de acudir al centro clínico;
    4. tener antecedentes de reacción alérgica grave a cualquier fármaco o excipiente de GLM101 (enumerados en el Manual del investigador de GLM101);
    5. tener antecedentes conocidos de mal acceso venoso;
    6. tener antecedentes de trasplante de hígado;
    7. tener antecedentes de alcoholismo o drogadicción en los 12 meses anteriores a la selección;
    8. haberse sometido a una intervención quirúrgica mayor en los 30 días anteriores a la selección;
    9. tener valores analíticos confirmatorios de la selección o la elegibilidad fuera del intervalo de referencia que sean considerados clínicamente significativos y que no estén relacionados con la PMM2-CDG;
    10. en el caso de las mujeres, tener una prueba de embarazo en suero con resultado positivo durante la selección;
    11. dar positivo en las pruebas serológicas en antígeno de superficie del virus de la hepatitis B o en anticuerpos contra el virus de la hepatitis C durante la selección;
    12. tener antecedentes o presentar, tras una evaluación clínica, cualquier enfermedad que pueda afectar a la seguridad de la infusión de GLM101 o a la evaluabilidad del efecto del medicamento a criterio del investigador principal y del supervisor médico;
    13. estar participando actualmente en otro estudio clínico de intervención o haber completado otro estudio clínico con un medicamento o producto sanitario en investigación, excepto la acetazolamida, en los 30 días o 5 semividas anteriores a la infusión de GLM101. Los participantes pueden inscribirse y continuar el tratamiento con acetazolamida solo si están recibiendo una dosis estable durante al menos 30 días antes de la administración de GLM101, y la dosis permanece inalterada durante todo el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    • Changes in ATIII activity level
    • Changes in factor XI activity
    • Cambios en el nivel de actividad de ATIII
    • Cambios en la actividad del factor XI
    E.5.1.1Timepoint(s) of evaluation of this end point
    Timepoints are measured from Baseline of the initial 12-week Treatment Period of the study
    Desde basal al inicio de las 12 semanas de tratamiento en el estudio
    E.5.2Secondary end point(s)
    • To evaluate the potential PD activity of GLM101 in adult participants with PMM2-CDG after 12 weeks of dosing
    • To assess the 12-week safety and tolerability of multiple doses of GLM101 in cohorts of participants with PMM2-CDG
    • To assess the PK of GLM101 in participants with PMM2-CDG after 12 weeks of dosing
    • Evaluar la potencial actividad de EP del GLM101 en participantes adultos con PMM2-CDG después de 12 semanas de dosificación
    • Evaluar la seguridad y la tolerabilidad durante 12 semanas de múltiples dosis de GLM101 en cohortes de participantes con PMM2-CDG
    • Evaluar la FC de GLM101 en participantes con PMM2-CDG tras 12 semanas de dosificación
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoints are measured from Baseline of the initial 12-week Treatment Period of the study
    Desde basal al inicio de las 12 semanas de tratamiento en el estudio
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tolerabilidad
    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 Yes
    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 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
    Australia
    United States
    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
    LVLS
    Última visita del último paciente
    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 months0
    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 months0
    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: 3
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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
    As these patients, also the adults may not be able to consent themselves due to ‘possible developmental delays’
    Existe la posibilidad de que estos pacientes, aún siendo adultos, no puedan consentir a la participación en el ensayo debido a posibles retrasos en el desarrollo
    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 2
    F.4.2.2In the whole clinical trial 6
    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)
    Standard of care
    Tratamiento estándar
    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 Decision2022-11-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-28
    P. End of Trial
    P.End of Trial StatusOngoing
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