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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7258   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).

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    EudraCT Number:2011-006308-12
    Sponsor's Protocol Code Number:TSP-RAP-1
    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:2012-03-29
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2011-006308-12
    A.3Full title of the trial
    phase II clinical trial, to study the efficacy and safety of topical rapamycin in reducing facial angiofibromas.
    Ensayo piloto, fase II, para el estudio de la eficacia y seguridad de la rapamicina tópica en la reducción de los angiofibromas faciales.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial in patients with tuberous sclerosis for the study of the effects of topical rapamycin in reducing facial tumors associated with the disease ..
    Ensayo clínico en pacientes con esclerosis tuberosa para el estudio de los efectos de la rapamicina tópica en la reducción de los tumores faciales asociados a la enfermedad.
    A.4.1Sponsor's protocol code numberTSP-RAP-1
    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 SponsorFundación Investigación Hospital Ramón y Cajal
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportConvocatoria Independiente Ministerio de Sanidad
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFundación Investigación Hospital Ramón y Cajal
    B.5.2Functional name of contact pointFundación Hospital Ramón y Cajal
    B.5.3 Address:
    B.5.3.1Street Addresscarretera Colmenar Viejo KM 9,100
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28034
    B.5.4Telephone number00349133688258825
    B.5.5Fax number00349133688258825
    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 Yes
    D. name Rapamune 1 mg/ml solución oral
    D. of the Marketing Authorisation holderPfizer, S.L.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPCutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRAPAMUNE
    D.3.9.2Current sponsor codeSIROLIMUS
    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. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCutaneous solution
    D.8.4Route of administration of the placeboCutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Facial tumors (angiofibromas) associated with tuberous sclerosis complex.
    Tumores faciales (angiofibromas) asociados al Complejo Esclerosis Tuberosa.
    E.1.1.1Medical condition in easily understood language
    Facial tumors (angiofibromas) associated with tuberous sclerosis complex.
    Tumores faciales (angiofibromas) asociados al Complejo Esclerosis Tuberosa.
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    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 clinical effectiveness of a topical treatment with rapamycin solution 1mg/ml for the treatment of facial angiofibromas associated to TSC.
    Evaluar la eficacia clínica del tratamiento tópico con rapamicina en solución 1mg/ml en tumores faciales (angiofibromas faciales; AF) en pacientes con esclerosis tuberosa (ET)
    E.2.2Secondary objectives of the trial
    To evaluate the safety of the treatment treatment with rapamycin solution 1mg/ml for the treatment of facial angiofibromas associated to TSC.
    To evaluate treatment efficiency through the characterization of tumor recurrence timing.
    Evaluar la seguridad del tratamiento tópico con rapamicina en pacientes con CET.
    Evaluar la duración en el tiempo de los efectos del tratamiento tras suspenderse el mismo (seguimiento de los efectos)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Patients, who, after receiving information about the design, the purpose of the study, the potential risks it can occur and that at any time may refuse your help, give written consent to participate in the study and for the provision material for cellular and molecular studies.
    -Subject is male or female and over 13 years of age.
    -Subject has a diagnosis of TSC and has visible fibromatosus lesions (angiofibromas or angifibromata)
    -women of childbearing age should get a negative pregnancy test the screening visit; further agree to use adequate contraception (among them are considered: double barrier as condom + diaphragm, surgical sterilization) within 14 days following administration of study drug.
    ?Pacientes, que tras haber recibir información sobre el diseño, los fines del estudio, los posibles riesgos que de él pueden derivarse y de que en cualquier momento pueden denegar su colaboración, otorguen por escrito su consentimiento para participar en el estudio y para la cesión de material para estudios celulares y moleculares.
    ?Tener esclerosis tuberosa con manifestación cutánea en forma de angiofibromas faciales.
    ?Ser mayor de 13 años.
    ?Prueba de embarazo en orina negativa realizada previa a la visita de screening.Las mujeres en edad fertil y los varones con pareja en edad fértil deben comprometerse a utilizar un método anticonceptivo de gran eficacia (como método de doble barrera, anticonceptivos orales o implantes hormonales contraceptivos) y a continuar utilizándolos hasta 6 meses después de la última dosis de tratamiento.
    E.4Principal exclusion criteria
    1.Pregnant, intention to become pregnant during treatment phase of the trial, or breastfeeding.
    2. Patient is currently participating or has participated within the last two months in any clinical trial.
    3.Patient is actually receiving or has received in the last three mounths any m-TOR inhibitor treatment or any form of immunosuppression drug.
    4. Hypersensitivity to rapamycin
    5.Any serious medical or psychiatric illness currently ongoing, or experienced within the past three months, that in the opinion of the investigator would compromise the study
    ?Embarazo o planificación de quedarse embarazada durante el transcurso del estudio.
    ?Estar participando en otros ensayos clínicos o haberlo hecho en los dos meses anteriores
    ?Estar recibiendo un inhibidor del complejo mTOR o cualquier tratamiento inmunosupresor.
    ?Hipersensibilidad conocida a rapamicina
    ?Cualquier patología importante que a juicio del investigador imposibilite la participación del paciente en el ensayo
    E.5 End points
    E.5.1Primary end point(s)
    The proportion of participants with clinical evidence of "success" (defined as reduction of at least 50% in size and number of tumors and a decrease in the puntuaction of the FASI index of at least 40% at the end of the treatmen period (three months). Success includes parcial and total responses.
    Proporción de pacientes con evidencia clínica de ?éxito terapéutico?, definido como la reducción de al menos el 50% del tamaño y número de los tumores y la mejoría de al menos el 40% en la puntuación obtenida en la escala FASI a los tres meses de tratamiento. El éxito incluye por tanto respuestas parciales y totales.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1ªweek month1 month2 month 4 month 7
    1ªs 1ºmes 3ºmes 4ºmes 7ºmes
    E.5.2Secondary end point(s)
    1.Proportion of patients achieving a total response at the end of the treatment period (3 months)
    2.Proportion of patients achieving a total response at the first month of the study.
    3.Proportion of patients achieving a complete or a parcial response after one week of treatment.
    4.Proportion of patients achieving a total response at the first week of the study.
    5.Proportion of patients achieving a complete or a parcial response after one month of treatment.
    6.Time of relapse

    Safety secondary endpoint
    1.Proportion of patients who suffer any AE
    2.Proportion of local reactions
    3.Proportion of patiens whose serum rapamycin level is detectable
    4.Proportion of patients whose laboratory tests became abnormal when at screening were not.
    5.Proportion of patients whose blood preasure or whose respiratory/ heart rate became abnormal
    -De eficacia: Proporción de pacientes que alcanzan respuesta completa al tratamiento a los tres meses (final del periodo de tratamiento). Por respuesta completa se entiende la desaparición de los tumores y del eritema.
    1.Proporción de pacientes que alcanzan respuesta completa al tratamiento al mes de tratamiento.
    2.Proporción de pacientes que alcanzan una respuesta, parcial o completa, a la semana de tratamiento.
    3.Proporción de pacientes que alcanzan respuesta completa al tratamiento a la semana de tratamiento.
    4.Proporción de pacientes que alcanzan una respuesta, parcial o completa, al mes de tratamiento.
    5.Tiempo transcurrido hasta recaída.

    De seguridad:

    1.Proporción de pacientes que desarrollan cualquier tipo de efecto adverso.
    2.Proporción de pacientes con síntomas locales.
    3.Proporción de casos en los que el fármaco se detecta en sangre
    4.Proporción de casos en los que se detectan alteraciones analíticas.
    5.Proporción de casos en los que se detectan alteraciones en las constantes vitales (Tensión arterial, frecuencia cardiaca y frecuencia respiratoria)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1ªweek month1 month2 month 4 month 7
    1ªs 1ºmes 3ºmes 4ºmes 7ºmes
    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 No
    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 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    the last visit of the last subject undergoing the trial
    ultima visita del último paciente incluido en el ensayo
    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
    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: 25
    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) Yes
    F. of subjects for this age range: 25
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    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
    sujetos menores de 18 años
    subjects under 18 years
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    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)
    The expected treatment of that condition
    El tratamiento esperado para la patología
    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 Decision2012-07-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-12-23
    P. End of Trial
    P.End of Trial StatusOngoing
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