E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Facial tumors (angiofibromas) associated with tuberous sclerosis complex. |
Tumores faciales (angiofibromas) asociados al Complejo Esclerosis Tuberosa. |
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E.1.1.1 | Medical condition in easily understood language |
Facial tumors (angiofibromas) associated with tuberous sclerosis complex. |
Tumores faciales (angiofibromas) asociados al Complejo Esclerosis Tuberosa. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main 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) |
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E.2.2 | Secondary 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) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
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E.4 | Principal 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. ?Lactancia ?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 |
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E.5 End points |
E.5.1 | Primary 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. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1ªweek month1 month2 month 4 month 7 |
1ªs 1ºmes 3ºmes 4ºmes 7ºmes |
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E.5.2 | Secondary 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) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1ªweek month1 month2 month 4 month 7 |
1ªs 1ºmes 3ºmes 4ºmes 7ºmes |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial 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
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the last visit of the last subject undergoing the trial |
ultima visita del último paciente incluido en el ensayo |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |