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    EudraCT Number:2015-001955-54
    Sponsor's Protocol Code Number:BMN-051-302
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-10-05
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2015-001955-54
    A.3Full title of the trial
    An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.
    Estudio de extensión abierto sobre la seguridad, tolerabilidad y eficacia a largo plazo de drisapersen en sujetos con distrofia muscular de Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.
    Estudio clínico para dar administrar drisapersen ( medicación del estudio) a pacientes con enfermedad Duchenne ( enfermad muscular) y para evaluar la seguridad, tolerabilidad y efectos tomando la medicación a largo plazo.
    A.3.2Name or abbreviated title of the trial where available
    Drisapersen extension study
    Estudio Drisapersen de extensión
    A.4.1Sponsor's protocol code numberBMN-051-302
    A.5.4Other Identifiers
    Name:Duchenne Muscular DystrophyNumber:DMD
    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 SponsorBioMarin 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 supportBioMarin Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Pharmaceutical Inc
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address105 Digital Drive
    B.5.3.2Town/ cityNovato
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    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/3/08/599
    D.3 Description of the IMP
    D.3.1Product nameDrisapersen
    D.3.2Product code Drisapersen
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdrisapersen
    D.3.9.2Current sponsor codePRO051
    D.3.9.3Other descriptive nameDRISAPERSEN SODIUM
    D.3.9.4EV Substance CodeSUB177199
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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. 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 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
    Duchenne Muscular Dystrophy (DMD)
    Distrofia muscular de Duchenne (DMD)
    E.1.1.1Medical condition in easily understood language
    Duchenne disease, muscle weakness
    Enfermedad Duchenne, debilidad de los músculos
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    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 and tolerability of subcutaneous or intravenous drisapersen in subjects with DMD correctable by drisapersen-induced DMD exon 51 skipping who have previously participated in an eligible study.
    Evaluar la seguridad y la tolerabilidad a largo plazo de drisapersen por vía subcutánea o intravenosa en sujetos con DMD corregible saltando el exón 51 mediante drisapersen, que han participado anteriormente en un estudio elegible.
    E.2.2Secondary objectives of the trial
    -To evaluate the long-term efficacy of subcutaneous drisapersen at a dose of 6 mg/kg/week.
    -To evaluate the long-term impact on functional outcomes of continued treatment with drisapersen.
    -To evaluate the long-term safety and efficacy of an intermittent dosing option in those subjects unable to tolerate drisapersen 6 mg/kg/week.
    -To evaluate the long-term safety and efficacy of an intravenous dosing option in those subjects unable to tolerate subcutaneous administration of drisapersen.
    - Evaluar la eficacia a largo plazo de drisapersen por vía subcutánea en dosis de 6 mg/kg/semana.
    - Evaluar el impacto a largo plazo en los resultados funcionales del tratamiento continuado con drisapersen.
    - Evaluar la seguridad y la eficacia a largo plazo de una opción de administración intermitente en aquellos sujetos que no toleran la administración de drisapersen en dosis de 6 mg/kg/semana.
    - Evaluar la seguridad y la eficacia a largo plazo de una opción de administración intravenosa en aquellos sujetos que no toleran la administración subcutánea de drisapersen.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Any subject who has been previously treated with an exon 51 skipping antisense oligonucleotide (drisapersen or eteplirsen) and is not eligible for another ongoing drisapersen study. Subjects who withdrew from the previous studies due to meeting laboratory safety stopping criteria may be eligible to enroll if:
    a. The laboratory parameters that led to stopping have resolved; benefit of further treatment with drisapersen outweighs the risk to the individual subject; and following consultation with the Medical Monitor.
    2. Subjects with DMD mutation/deletion within the dystrophin gene and correctable by drisapersen-induced DMD exon 51 skipping.
    3. Male subjects age > or =5 with DMD in whom the investigator considers treatment with drisapersen is likely to lead to improvement or prevent worsening of the condition.
    4. Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on glucocorticoids for the duration of this study. Changes to or cessation of glucocorticoids will be at the discretion of the investigator conducting this study in consultation with the subject/parent and Medical Monitor.
    5. Willing and able to comply with all study requirements and procedures (with the exception of those assessments requiring a subject to be ambulant, for those subjects who have lost ambulation).
    6. Able to give informed assent and/or consent in writing by the subject and/or parent(s)/legal guardian (according to local regulations).
    1. Sujetos que han sido tratados previamente con un oligonucleótido antisentido que salte el exón 51 (drisapersen o eteplirsen) y no son elegibles para otro estudio con drisapersen en curso. Los sujetos que se retiraron de estudios anteriores por cumplimiento de los criterios analíticos de interrupción por seguridad pueden ser elegibles para inscribirse si:
    a) Los parámetros de laboratorio que llevaron a dicha interrupción se han resuelto, si el beneficio de un tratamiento adicional con drisapersen es mayor que el riesgo para el sujeto y tras consultar con el monitor médico.
    2. Sujetos que tienen una mutación DMD/deleción en el gen de la distrofina que se corrige saltando el exón 51 mediante drisapersen.
    3. Varones >5 años de edad con DMD, en los que el investigador considere que el tratamiento con drisapersen podría mejorar o prevenir el empeoramiento de la afección.
    4. Sujetos con uso continuado de glucocorticoides durante 60 días como mínimo antes de la entrada en el estudio, con la expectativa razonable de que el sujeto continuará con estos fármacos durante este estudio. Las modificaciones o interrupción de los glucocorticoides se harán a criterio del investigador que realiza este estudio en consulta con el sujeto, el padre o la madre y el monitor médico.
    5. Sujetos con disposición y capacidad para cumplir con todos los requisitos y procedimientos del estudio (salvo aquellas evaluaciones que requieran que el sujeto pueda deambular, en aquellos casos que hayan perdido esta capacidad).
    6. Sujetos y/o padres/tutores legales (conforme a las regulaciones locales) con capacidad de otorgar el asentimiento y/o consentimiento informado por escrito
    E.4Principal exclusion criteria
    1. Subjects who have previously been treated with drisapersen and who had a serious adverse experience or who met safety stopping criteria that remains unresolved, which in the opinion of the investigator could have been attributable to drisapersen. Once resolved, subject may be eligible to enter the study following investigator consultation with the Medical Monitor.
    2. Use of anticoagulants, anti-thrombotics or antiplatelet agents within 28 days of the first re-dosing of drisapersen. Chronic use of anticoagulants, anti-thrombotics or antiplatelet agents is prohibited during the study. As needed dosing (pro re nata ? PRN) may be acceptable (except for aspirin) following discussion with the Medical Monitor.
    3. Participation in any investigational clinical trial within 3 months prior to start or during this study (except for other drisapersen studies). If subjects have participated in any other study within the last 6 months this should be discussed with the Medical Monitor prior to start of this study.
    4. History of significant medical disorder which may confound the interpretation of safety data (e.g. current or history of renal or liver disease/impairment, history of inflammatory illness)
    5. Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction <45% at start of this study, the investigator should discuss inclusion of subject in this study with the Medical Monitor.
    6. A platelet count under the lower limit of normal (LLN) at start of this study. A re-test is possible at a later stage, and if within normal range, the subject may enter the study.
    1. Sujetos que han sido previamente tratados con drisapersen y que presentaron una reacción adversa grave o que cumplieron con los criterios de interrupción por seguridad que siguen sin resolverse, que a juicio del investigador podrían haber sido atribuibles a drisapersen. Una vez resueltos, el sujeto puede ser elegible para entrar en el estudio tras consulta del investigador con el monitor médico.
    2. Uso de anticoagulantes, antitrombóticos o antiagregantes en los 28 días anteriores a la primera reintroducción de drisapersen. Durante este estudio se prohíbe el uso crónico de anticoagulantes, antitrombóticos o antiagregantes. La administración cuando sea necesario (pro re nata, PRN) puede ser aceptable (salvo el ácido acetilsalicílico) tras comentar el tratamiento con el monitor médico.
    3. Participación en cualquier estudio de investigación en los 3 meses anteriores al inicio de este estudio o durante el mismo (salvo otros estudios con drisapersen). Si los sujetos han participado en otro estudio clínico en los últimos 6 meses, este aspecto debería comentarse con el monitor médico antes de iniciar este estudio.
    4. Antecedentes de un problema médico significativo que pudiera confundir la interpretación de los datos de seguridad (p. ej., enfermedad o insuficiencia hepática o renal actual o pasada o antecedentes de enfermedades inflamatorias)
    5. Miocardiopatía sintomática. Si el sujeto tiene una fracción de eyección ventricular izquierda <45 % al inicio de este estudio, el investigador deberá comentar con el monitor médico su inclusión en este estudio.
    6. Recuento de plaquetas menor que el límite inferior de la normalidad (LIN) al inicio de este estudio. Este análisis puede repetirse más adelante y el sujeto puede entrar en el estudio si la cifra se encuentra dentro del intervalo normal.
    E.5 End points
    E.5.1Primary end point(s)
    Study Endpoints/Assessments

    Safety Variables:
    - Incidence and severity of adverse events
    - Vital signs
    -ECG parameters
    - Injection Site Reactions
    - Safety haematology and biochemistry parameters including non-standard parameters such as coagulation parameters (in particular activated partial thromboplastin time [aPTT]), serum cystatin C, Complement Factor C3, haptoglobin, fibrinogen, high-sensitivity C-reactive protein (hsCRP)
    - Urinalysis (including quantitative protein and creatinine and their ratio)

    Efficacy Variables:
    Ambulant subjects
    -Muscle function using 6 minute walking distance (6MWD) test.
    -North Star Ambulatory Assessment.

    All subjects
    -Pulmonary function (forced expiratory volume in the 1st second of exhalation [FEV1], forced vital capacity [FVC]). In addition, at selected sites, Maximum Inspiratory Pressure (MIP) and Maximum Expiratory Pressure (MEP) will be assessed.
    -Time to major disease milestones (e.g. loss of ambulation, night time ventilation)
    -Performance Upper Limb (PUL)
    -Patient Reported Outcome Measures (PODCI and EQ-5D-5L)
    -MRI (at selected centers)
    Variables de seguridad:
    -Incidencia y gravedad de los acontecimientos adversos
    -Constantes vitales
    -Parámetros del ECG
    -Reacciones en el lugar de la inyección
    -Los parámetros de seguridad, hematología y bioquímica, incluidos los parámetros no habituales como los parámetros de la coagulación (en particular, el tiempo de tromboplastina parcial activada [TTPa]), cistatina C sérica, factor C3 del complemento, haptoglobina, fibrinógeno, proteína C reactiva de alta sensibilidad (PCR-as)
    -Análisis de orina (incluida la cuantificación de proteínas y creatinina, y su cociente)

    Variables de eficacia:
    Sujetos capaces de deambular
    -Función muscular mediante la prueba de distancia caminada en 6 minutos (6MWD).
    - Evaluación North Star de la deambulación (North Star Ambulatory Assessment).

    Todos los sujetos
    -Función pulmonar (volumen espiratorio forzado en el 1er segundo de la espiración [VEF1], capacidad vital forzada [CVF]). Además, en centros seleccionados, se evaluará la presión inspiratoria máxima (PIM) y la presión espiratoria máxima (PEM).
    -Tiempo hasta los principales hitos de la enfermedad (por ejemplo, pérdida de deambulación, ventilación nocturna)
    -Rendimiento del miembro superior (RMS)
    -Criterios de valoración comunicados por el paciente (CVCP y EQ-5D-5L)
    -RM (en centros seleccionados)
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of study
    Fin de estudio
    E.5.2Secondary end point(s)
    E.5.2.1Timepoint(s) of evaluation of this end point
    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 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 No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 trial3
    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 EEA45
    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
    Czech Republic
    Korea, Republic of
    Russian Federation
    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
    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 months11
    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 months11
    E.8.9.2In all countries concerned by the trial days18
    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: 220
    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: 112
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 103
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    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
    young patients starting 5 years and older.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 190
    F.4.2.2In the whole clinical trial 220
    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)
    BioMarin wishes to provide re-access to drisapersen for subjects who previously participated in drisapersen clinical studies and who are not able to participate in any ongoing drisapersen studies in their country, until such time as drisapersen may be available commercially in their country.
    Biomarin desea proporcionar re-acceso a drisapersen para los sujetos que participaron previamente en estudios clínicos drisapersen y que no pueden participar en cualquier estudio drisapersen en curso en su país, hasta el momento en drisapersen pueda estar disponible comercialmente en su país.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-11-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-09-09
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