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    EudraCT Number:2019-002979-34
    Sponsor's Protocol Code Number:LTFU-ABO-102
    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:2020-05-25
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2019-002979-34
    A.3Full title of the trial
    A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
    Estudio de seguimiento a largo plazo de pacientes con MPS IIIA procedentes de ensayos clínicos de terapia génica que incluyen la administración de ABO-102 (scAAV9.U1a.hSGSH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
    Estudio de seguimiento a largo plazo de pacientes con MPS IIIA procedentes de ensayos clínicos de terapia génica que incluyen la administración de ABO-102 (scAAV9.U1a.hSGSH)
    A.4.1Sponsor's protocol code numberLTFU-ABO-102
    A.5.4Other Identifiers
    Name:IND NumberNumber:16850
    Name:PEI NumberNumber:16-041
    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 SponsorAbeona Therapeutics Inc
    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 supportAbeona Therapeutics Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAbeona Therapeutics Inc
    B.5.2Functional name of contact pointHead of European Medical Affairs
    B.5.3 Address:
    B.5.3.1Street AddressAvenida de Manoteras 30, A207-208
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28050
    B.5.4Telephone number+34689166070
    B.5.5Fax number+34911726254
    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/3/16/1761
    D.3 Description of the IMP
    D.3.1Product namescAAV9.U1A.hSGSH
    D.3.2Product code ABO-102
    D.3.4Pharmaceutical form Suspension for injection
    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 INNRebisufligene etisparvovec
    D.3.9.2Current sponsor codeABO-102
    D.3.9.4EV Substance CodeSUB195015
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number10000000000000
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatment
    currently available for the disease.
    MPS IIIA es una enfermedad de depósito lisosomal, causada por un defecto genético de la enzima N-sulfoglucosamina sulfohidrolasa. Los niños parecen normales al nacer, pero la enfermedad es progresiva, con
    el deterioro de las habilidades sociales y de adaptación, la disminución neurocognitiva y la muerte prematura. La muerte se produce normalmente a finales de la segunda o principios de la tercera década. Es de destacar que no existe un tratamiento disponible actualmente para la enfermedad.
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis type IIIA is a genetic disease in children, caused by the toxicity of an accumulation of substances in the body that generate a progressive deterioration.
    La mucopolisacaridosis tipo IIIA es una enfermedad genética en niños, causada por la toxicidad de un acúmulo de sustancias en el organismo que generan un deterioro progresivo.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10056890
    E.1.2Term Mucopolysaccharidosis III
    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
    To evaluate the long-term safety/tolerability of ABO-102 in patients with MPS IIIA
    Evaluar la seguridad/tolerabilidad a largo plazo de ABO-102 en pacientes con MPS IIIA
    E.2.2Secondary objectives of the trial
    To evaluate the neurocognitive evolution of patients with MPS IIIA treated with ABO-102
    Evaluar la evolución neurocognitiva de pacientes con MPS IIIA tratados con ABO-102
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Participants that have completed a prior clinical trial involving the administration of ABO-102.
    • Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.
    • Participantes que hayan completado un ensayo clínico previo que implique la administración de ABO-102.
    • Padres/tutor(es) legal(s) del participante dispuesto(s) y capaz(ces) de completar el consentimiento informado y cumplir con los procedimientos del estudio y el calendario de visitas.
    E.4Principal exclusion criteria
    • Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-102 during the duration of this study.
    • Any other situation that precludes the participant from undergoing procedures required in this study.
    • Participación planificada o actual en otro ensayo clínico que pueda interferir en la evaluación de la seguridad y eficacia de ABO-102 durante la duración de este estudio.
    • Cualquier otra situación que impida al participante someterse a los procedimientos requeridos en este estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
    Seguridad del producto a largo plazo, tal como se define por la incidencia, tipo y gravedad de los Acontecimientos Adversos (AAs) y Acontecimientos Adversos Graves (AAGs).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Months: 30, 36, 42 (only for subjects recruited below 3 years of age) , 48, 60. After ABO-102 treatment administered in a prior clinical trial.
    Mes: 30, 36, 42 (solo en los sujetos que se han reclutado con un edad menor de 3 años), 48, 60. Después del tratamiento con ABO-102 recibido en un ensayo clínico previo.
    E.5.2Secondary end point(s)
    • Neurocognitive evolution based on Age Equivalent assessments included in the prior clinical trial including:
    o Change from baseline (in prior trial) in the Age Equivalent and Developmental Quotient (DQ) after treatment compared to Natural History Study data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children; Second Edition, based on chronological and developmental age.
    o Change from baseline (in prior trial) in the Cognitive Age Equivalent and Developmental Quotient after treatment compared to Natural History Study, calculated using the Bayley Scales of Infant and Toddler Development – Third edition or the Kaufman Assessment Battery for Children. Second Edition, based on developmental age.
    o Change from baseline (in prior trial) in the Adaptive Age Equivalent score after treatment compared to Natural History Study data, as assessed by parent report using the Vineland Adaptive Behavior Scale II Survey form .
    • Quality of life based on Pediatric Quality of Life Inventory (PedsQL™ and PedsQL Gastrointestinal Symptom scales) and Parenting Stress Index, 4th Edition (PSI-4), and sleep-pattern evaluation based on modified Children’s Sleep Habits Questionnaire (CSHQ) (only applicable for participants who completed these evaluations in the prior clinical trial).
    • Evolution on Parental Global Impression Score, Clinical Global Impression Improvement Score and Parent Symptom Score Questionnaire.
    • Long-term immunological responses defined as antibody formation (humoral) and T-cell responses (cellular) against the AAV9 capsid and against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
    • Long-term viral load, in applicable cases.
    • Evolución neurocognitiva basada las evaluaciones Equivalentes de Edad incluidas en el ensayo clínico anterior, incluyendo:
    o Cambio desde la visita de inicio (en el ensayo anterior) en el Edad Equivalente y Cociente de Desarrollo (DQ) después del tratamiento, en comparación con los datos del Estudio de Historia Natural calculados por las Escalas Mullen de Aprendizaje Temprano o Evaluación de Kaufman para niños; Segunda edición, basada en la edad cronológica y de desarrollo.
    o Cambio desde la visita de inicio (en el ensayo anterior) en el Equivalente de Edad Cognitiva y Cociente de Desarrollo después del tratamiento en comparación con el Estudio de Historia Natural, calculado usando las Escalas de Bayley para el desarrollo infantil y niños pequeños- Tercera edición, o mediante o Evaluación de Kaufman - Segunda Edición basada en la edad de desarrollo.
    o Cambio desde la visita de inicio (en el ensayo anterior) en la puntuación de Edad Equivalente Adaptativa después del tratamiento en comparación con los datos del Estudio de Historia Natural, según lo evaluado por el informe de los padres utilizando el formulario del Estudio de la Escala de Conducta Adaptativa II de Vineland.
    • Calidad de vida basada en el cuestionario de Calidad de vida (PedsQL™) y el cuestionario de stress en los padres, 4ª edición (PSI-4), y evaluación de patrones de sueño basada en el cuestionario modificado de hábitos de sueño en los niños (CSHQ) (sólo aplicable para los participantes que completaron estas evaluaciones en el ensayo clínico anterior).
    • Evolución de la puntuación en el formulario de impresión global de los padres, el formulario global de impresión global de mejoría clínica y cuestionario de puntuación de los síntomas de los padres.
    • Respuestas inmunológicas a largo plazo definidas como formación de anticuerpos (humoral) y respuesta de células T (celular) contra la cápside de AAV9 y contra el transgén de N-sulfoglucosamina sulfohidrolasa (SGSH).
    • Carga viral a largo plazo, en los casos que aplique.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Viral load is measured at each visits until two consecutive samples are negative.
    All the others secondary endpoints are evaluated at Month: 30, 36, 42, 48, 60.
    La carga viral se mide en cada visita hasta que se obtengan dos muestras negativas consecutivas.
    Todos los demás parámetros secundarios se miden en Mes: 30, 36, 42, 48, 60.
    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 No
    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
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E. trial type description
    Long term follow up
    Seguimiento a largo plazo
    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 No
    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 Yes
    E. trial design description
    Seguimiento a largo plazo
    Long term follow up
    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 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 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
    Ultima visita del ultimo participante
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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 Yes
    F.1.1Number of subjects for this age range: 34
    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. of subjects for this age range: 32
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 2
    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. of subjects incapable of giving consent
    These children have severe cognitive problems making them very difficult to understand an explanation about the study and also to sign the informed consent. Based on a expert opinion, unique ICF for parents or legal representatives is proposed.
    Estos niños tienen problemas cognitivos severos que dificultan su entendimiento de una explicación del estudio y firma del consentimiento informado. Basado en una opinión experta, se ha preparado un único ICF para los padres o representantes legales.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 34
    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)
    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 Decision2020-05-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-05-07
    P. End of Trial
    P.End of Trial StatusOngoing
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