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    Summary
    EudraCT Number:2021-001793-36
    Sponsor's Protocol Code Number:GTX-102-001
    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:2022-10-13
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2021-001793-36
    A.3Full title of the trial
    A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)
    Ensayo clínico de fase 1/2, abierto, de dosis múltiples y de aumento gradual de la dosis para evaluar la seguridad y la tolerabilidad de GTX-102 en pacientes pediátricos con síndrome de Angelman (SA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the safety of GTX-102 in pediatric patients with Angelman Syndrome (AS)
    Ensayo clínico de la seguridad de GTX-102 en pacientes pediátricos con síndrome de Angelman (SA)
    A.4.1Sponsor's protocol code numberGTX-102-001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04259281
    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 SponsorUltragenyx Pharmaceutical 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 supportUltragenyx Pharmceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUltragenyx Pharmceutical Inc.
    B.5.2Functional name of contact pointStaffany Gaffagan
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato, CA
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.6E-mailsgaffagan@ultragenyx.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 nameGTX-102
    D.3.2Product code GTX-102
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot available
    D.3.9.2Current sponsor codeGTX-102
    D.3.9.4EV Substance CodeSUB292567
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number11
    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.IMP: 2
    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 Yes
    D.2.1.1.1Trade name ELLIOTTS B® SOLUTION (buffered intrathecal electrolyte/dextrose injection)
    D.2.1.1.2Name of the Marketing Authorisation holderLukare Medical, LLC
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameELLIOTTS B® SOLUTION
    D.3.2Product code ELLIOTTS B® SOLUTION
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    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 Yes
    D.3.11.13.1Other medicinal product typeElliotts B Solution is a sterile, nonpyrogenic, isotonic solution containing no bacteriostatic preservatives. It is a diluent for intrathecal administration of methotrexate sodium and cytarabine
    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
    Angelman Syndrome
    síndrome de Angelman (SA)
    E.1.1.1Medical condition in easily understood language
    A genetic condition that affects the nervous system and causes severe physical and learning disabilities
    Una condición genética que afecta el sistema nervioso y causa severas discapacidades físicas y de aprendizaje.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    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 safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to patients with AS
    Evaluar la seguridad y la tolerabilidad de dosis múltiples ascendentes de GTX-102 administradas mediante inyección intratecal (IT) a pacientes con SA.
    E.2.2Secondary objectives of the trial
    To evaluate the pharmacokinetics (PK) of GTX-102 in plasma and cerebrospinal fluid (CSF) of patients with AS
    Evaluar la farmacocinética (FC) de GTX-102 en plasma y líquido cefalorraquídeo (LCR) de pacientes con SA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed informed consent from parent(s) or legal guardian(s)
    2. Documented genetic confirmation of full maternal UBE3A gene deletion causing AS (eg,
    DNA methylation testing with either a chromosomal microarray or FISH) in the region of
    15q11.2-q13 including class I, II or III)
    3. Age ≥ 4 to ≤ 17 years at screening
    4. Stable seizure control (defined as clinically stable with no changes in antiepileptic
    medications over the prior 1 month before screening visit, other than weight associated
    dose adjustments)
    5. Platelet count, PT / INR, and PTT within 1.2 x the normal limits
    6. Normal renal function with serum creatinine and spot urine protein ≤ 1.2 x ULN
    7. Normal hepatic function with total bilirubin, aspartate aminotransferase, alanine
    aminotransferase, and alkaline phosphatase ≤ 1.3 x ULN. Exception: levels ≤ 2 × ULN
    are acceptable if due to AEDs or Gilbert syndrome
    8. Willing and able to comply with scheduled visits, drug administration plan, laboratory
    tests, study restrictions, and all study procedures, including LP procedure.
    9. Able to tolerate the anesthetic regimen, if required for LP procedure
    10. For any patients that are sexually mature and sexually active, willing to use birth control
    for the duration of the study
    1. Consentimiento informado firmado de los padres o tutores legales.
    2. Confirmación genética documentada de la deleción completa del gen UBE3A materno que causa SA (p. ej., análisis de metilación del ADN con una micromatriz cromosómica o FISH) en la región de 15q11.2-q13 incluidas las clases I, II o III.
    3. Edad ≥ 4 a ≤ 17 años en la selección.
    4. Control estable de las convulsiones (definido como clínicamente estable sin cambios en la medicación antiepiléptica durante el mes previo a la visita de selección, aparte de los ajustes de la dosis asociados al peso).
    5. Cifra de trombocitos, tiempo de protrombina/índice normalizado internacional y tiempo de tromboplastina parcial dentro de 1,2 veces los límites de la normalidad.
    6. Función renal normal con creatinina sérica y proteínas en una muestra puntual de orina ≤ 1,2 veces el LSN
    7. Función hepática normal con bilirrubina total, aspartato aminotransferasa, alanina aminotransferasa y fosfatasa alcalina ≤ 1,3 x LSN. Excepción: son aceptables las concentraciones ≤ 2 × LSN si se deben a antiepilépticos (AE) o síndrome de Gilbert.
    8. Disposición y capacidad para cumplir las visitas programadas, el plan de administración del fármaco, las pruebas analíticas, las restricciones del estudio y todos los procedimientos del estudio, incluido el procedimiento de punción lumbar.
    9. Capaz de tolerar la anestesia, si es necesario para el procedimiento de punción lumbar.
    10. En el caso de pacientes sexualmente maduros y sexualmente activos, deben estar dispuestos a utilizar un método anticonceptivo durante el estudio.
    E.4Principal exclusion criteria
    1. Any change in medications (excluding AEDs) or diet/supplements intended to treat
    symptoms of AS (eg, sleeping aids, supplements, ketogenic or low-glycemic index diet,
    other) over the prior 1 month before screening.
    2. Inability to ambulate independently or with an assistive device or caregiver hand-hold
    3. Any bleeding or platelet disorder
    4. Any clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary,
    gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in
    the judgment of the Investigator, will pose a safety risk, will make the patient unsuitable
    for participation in, and/or unable to complete the study procedures.
    5. Any laboratory abnormality, that, in the Investigator’s opinion, could adversely affect the
    safety of the patient, make it unlikely that the course of treatment or follow up would be
    completed, or impair the assessment of study result
    6. Known positive for hepatitis B virus, hepatitis C virus, or human immunodeficiency virus
    7. Any active infection
    8. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or
    unsuccessful lumbar puncture
    9. Drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin,
    platelet inhibitors).
    10. Use of any investigational oligonucleotide (with the exception of prior GTX-102)
    11. Any prior use of gene therapy
    12. Use of any investigational drugs in the past 6 months (with the exception of prior
    GTX-102)
    13. Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic
    reaction such as changes in pulse, blood pressure, breathing function
    14. Patient is pregnant or lactating
    15. Any medical condition that would require intubation for the anesthesia procedure
    1. Cualquier cambio en la medicación (excluidos los AE) o la dieta/suplementos destinados a tratar los síntomas del SA (p. ej., somníferos, suplementos, dieta cetogénica o de bajo índice glucémico, otros) en el mes previo a la selección.
    2. Incapacidad para caminar de forma independiente o con un dispositivo de ayuda o la mano del cuidador.
    3. Cualquier trastorno hemorrágico o plaquetario.
    4. Cualquier trastorno cardiovascular, endocrino, hepático, renal, pulmonar, digestivo, neurológico, maligno, metabólico, psiquiátrico o de otro tipo clínicamente significativo que, en opinión del investigador, suponga un riesgo para la seguridad, haga que el paciente no sea apto para participar en los procedimientos del estudio o sea incapaz de completarlos.
    5. Cualquier anomalía analítica que, en opinión del investigador, pueda afectar negativamente a la seguridad del paciente, reducir las probabilidades de completar el tratamiento o el seguimiento o afectar a la evaluación del resultado del estudio.
    6. Positividad conocida para el virus de la hepatitis B, el virus de la hepatitis C o el virus de la inmunodeficiencia humana.
    7. Cualquier infección activa.
    8. Trastorno óseo, vertebral, hemorrágico o de otro tipo que exponga al paciente al riesgo de lesión o de punción lumbar infructuosa.
    9. Fármacos que aumentan el riesgo de hemorragia (p. ej., heparina, heparina de bajo peso molecular, inhibidores plaquetarios).
    10. Uso de cualquier oligonucleótido en investigación (con la excepción de GTX-102 previo).
    11. Cualquier uso previo de terapia génica.
    12. Uso de cualquier fármaco en investigación en los 6 últimos meses (con la excepción de GTX-102 previo).
    13. Hipersensibilidad conocida a cualquier oligonucleótido, demostrada por una reacción alérgica sistémica, como alteraciones del pulso, la tensión arterial y la función respiratoria.
    14. La paciente está embarazada o en período de lactancia.
    15. Cualquier afección médica que requiera intubación para el procedimiento de anestesia.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs (SAEs),
    discontinuations due to TEAEs, and the severity of TEAEs

    Changes in physical and neurological examinations, vital signs, laboratory tests (chemistry,
    hematology, coagulation markers, inflammatory markers, urinalysis), electrocardiogram (ECG), and CSF protein levels and other potential safety biomarkers which may include, but are not limited to neurofilament-light, phosphorylated Neurofilaments-heavy (pNFheavy), brain-derived neurotrophic factor (BDNP), glial fibrillary acidic protein (GFAP),
    ubiquitin carboxy-terminal hydrolase L1 (UCH-L1), and matrix metalloproteinase 9
    (MMP-9)
    Investigar los efectos farmacodinámicos (FD) del tratamiento con GTX-102 en pacientes con SA.
    Investigar la posible eficacia del tratamiento con GTX-102 basándose en diversos criterios de valoración clínicos.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Dose Escalation Phase: Day 1, 2, 3, 16, 30, 44, 58, 72, 86, 100 and 128
    E.5.2Secondary end point(s)
    Plasma PK of GTX-102
    GTX-102 concentrations in CSF
    E.5.2.1Timepoint(s) of evaluation of this end point
    Monthly Dosing Phase
    -Plasma PK of GTX-102: Day 2, 3, 58
    -GTX-102 concentrations in CSF: Day 2, 30, 58, 86
    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) Yes
    E.7.1.1First administration to humans Yes
    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.8.3 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Canada
    Israel
    United States
    France
    Germany
    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
    Patients may continue on GTX-102 during the Maintenance phase of the study until GTX-102 is commercially available,
    intolerable toxicity occurs, the parent/legal guardian withdraws consent, or the study is terminated.
    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 months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months3
    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 Yes
    F.1.1Number of subjects for this age range: 83
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 41
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 42
    F.1.2Adults (18-64 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Written assent of minors in accordance with local regulations is not applicable in light of the cognitive deficits in children with AS.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 83
    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)
    Patients may continue on GTX-102 during the Maintenance phase of the study until GTX-102 is commercially available, intolerable toxicity occurs, the parent/legal guardian withdraws consent, or the study is terminated, intolerable toxicity occurs, the parent/legal guardian withdraws consent or the study is terminated.
    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 Decision2023-06-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-14
    P. End of Trial
    P.End of Trial StatusOngoing
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