E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Invasive fungal disease in the hematologic patient. |
Enfermedad fúngica invasiva en el paciente hematológico. |
|
E.1.1.1 | Medical condition in easily understood language |
Infectious diseases by fungi, in patients with blood diseases. |
Enfermedades infecciosas por hongos, en pacientes con enfermedades de la sangre. |
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E.1.1.2 | Therapeutic area | Body processes [G] - Microbiological Phenomena [G06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10017528 |
E.1.2 | Term | Fungal infectious disorders |
E.1.2 | System Organ Class | 10021881 - Infections and infestations |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10040054 |
E.1.2 | Term | Sepsis, bacteraemia, viraemia and fungaemia NEC |
E.1.2 | System Organ Class | 10021881 - Infections and infestations |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10029355 |
E.1.2 | Term | Neutropenias |
E.1.2 | System Organ Class | 100000004851 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10076734 |
E.1.2 | Term | Chemotherapy induced neutropenia |
E.1.2 | System Organ Class | 100000004851 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061187 |
E.1.2 | Term | Haematopoietic neoplasm |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10059041 |
E.1.2 | Term | Allogeneic peripheral haematopoietic stem cell transplant |
E.1.2 | System Organ Class | 100000004865 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10059040 |
E.1.2 | Term | Autologous peripheral haematopoietic stem cell transplant |
E.1.2 | System Organ Class | 100000004865 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Evaluating the effectiveness and efficiency of a strategy of "anticipated genotyping" of voriconazole in the treatment and prophylaxis of fungal infections by aspergillus in haematological patients. |
Evaluación de la efectividad y la eficiencia de una estrategia de "genotipado anticipado" de voriconazol en el tratamiento y profilaxis de las infecciones fúngicas por aspergillus en pacientes hematológicos. |
|
E.2.2 | Secondary objectives of the trial |
1. To evaluate the effectiveness of a strategy of "anticipated genotyping" of voriconazole to achieve adequate therapeutic levels in haematological patients with risk of fungal infection, in comparison with usual clinical practice. 2. To evaluate the efficacy and safety of "anticipated genotyping" of voriconazole in haematological patients with risk of fungal infection, in comparison with usual clinical practice. 3. Evaluate the efficiency of this strategy of "anticipated genotyping". |
1. Evaluar la efectividad de una estrategia de “genotipado anticipado” de voriconazol para conseguir niveles terapéuticos adecuados en pacientes hematológicos con riesgo de infección fúngica, en comparación con la práctica clínica habitual. 2. Evaluar la eficacia y seguridad del “genotipado anticipado” de voriconazol en pacientes hematológicos con riesgo de infección fúngica, en comparación con la práctica clínica habitual. 3. Evaluar la eficiencia de esta estrategia de “genotipado anticipado”. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patient at risk of developing invasive aspergillosis, potential receipent of treatment or prophylaxis with voriconazole: A. Pediatric population: children who are going to receive a transplant of hematopoietic precursors (HSCT) and acute myeloid leukemias, as well as relapses of it. B. Adult population: patients diagnosed with acute leukemia, and those patients with expected prolonged neutropenia, secondary to hematological process and / or after specific treatment (aplastic anemia and variants, myelodysplastic syndrome, solid organ or bone marrow transplant, etc.), and those whose responsible clinician consider individually that they could present a risk of developing a fungal infection. 2. Those who agree to participate in the study by signing informed consent (patients equal or over 18 years old) 3. Subjects under 18 yeras old whose representative / legal guardian has voluntarily signed the informed consent. 4. In the case of mature under 18 years subjects (12-17 years of age), in addition to the consent signed by the legal guardian, the consent of the subject will be obtained. |
1. Paciente con riesgo de desarrollar una aspergilosis invasiva que potencialmente puedan recibir tratamiento o profilaxis con voriconazol: A. Población pediátrica: niños que vayan a recibir un trasplante de precursores hematopoyéticos (TPH) y leucemias mieloides agudas, así como las recaídas de ésta. B. Población adulta: pacientes con diagnóstico de leucemias agudas, y aquellos pacientes con neutropenia prolongada esperada secundaria a proceso hematológico y/o tras tratamiento específico (anemia aplásica y variantes, síndrome mielodisplásico, trasplante de órgano sólido o médula, etc.) en el que el clínico responsable considere de forma individual que podrían presentar riesgo de desarrollar infección fúngica. 2. Que acepte participar en el estudio firmando el consentimiento informado (pacientes mayores de edad) 3. Sujetos menores cuyo representante/tutor legal haya firmado voluntariamente el consentimiento informado 4. En el caso de menores maduros (12-17 años de edad), además del consentimiento firmado por el tutor legal, se obtendrá el asentimiento del menor. |
|
E.4 | Principal exclusion criteria |
1. Patients who for any reason should not be included in the study according to the criteria of the research team. 2. Subjects who are not capable to understand the information sheet and unable to sign the informed consent. |
1. Pacientes que por cualquier motivo no deberían ser incluidos en el estudio según evaluación del equipo investigador. 2. Sujetos que no sean capaces de comprender la hoja de información e incapaces de firmar el consentimiento informado. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Serum voriconazole concentration within the therapeutic range start of treatment. |
Concentración sérica de voriconazol dentro del rango terapéutico |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
5th day after the treatment start. |
En el 5º día del tratamiento. |
|
E.5.2 | Secondary end point(s) |
1. Therapeutic failure and adverse events. COMBINED VARIABLE THAT INCLUDE: - Change of antifungal or the association with another antifungal agent due to poor clinical-radiological evolution. - Need for antifungal change, due to suspicion or confirmation of invasive fungal disease. 2. Adverse dose-related adverse events associated with treatment with voriconazole: visual disturbances (photopsias), skin reactions, neurotoxicity (confusion and visual hallucinations) and QTc lengthening. |
1. Fallo terapéutico y eventos adversos. VARIABLE COMBINADA QUE INCLUYE: - Cambio de antifúngicos o la asociación con otro agente antifúngico por mala evolución clínico-radiológica. - Necesidad de cambio antifúngico, debido a sospecha o confirmación de enfermedad fúngica invasiva. 2. Eventos adversos relacionados con la dosis adversa asociados con el tratamiento con voriconazol: trastornos visuales (fotopsias), reacciones cutáneas, neurotoxicidad (confusión y alucinaciones visuales) y alargamiento de QTc. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Not applicable |
Not applicable. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | Yes |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | Yes |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Regimen de Dosificación según Ficha Técnica |
Product Labels Dosing Regime |
|
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
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
|
Last visit of the last subject included. |
Última visita del último sujeto incluído. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |