E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Children affected or at risk of oral mucositis from chemoterapy and/or radiotherapy |
Bambini affetti o a rischio di mucosito orale da chemio e/o radioterapia |
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E.1.1.1 | Medical condition in easily understood language |
Childern who have or may develop signs and symptoms of inflammation of the mucosa of the mouth due to chemotherapy and/or radiotherapy |
Bambini che presentano o potrebbero sviluppare segni e sintomi di una infiammazione della mucosa della bocca dovuta alla chemio e/o radioterapia |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10029104 |
E.1.2 | Term | Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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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 |
Assess the feasibility of administering SAMITAL¿ in different ages in pediatric patients with oncological diseases at risk of developing or with oral mucositis induced by CT and / or RT |
Valutare la fattibilit¿ della somministrazione di SAMITAL¿ nelle diverse fasce d¿et¿ pediatrica in pazienti affetti da malattie oncologiche a rischio di sviluppare o affetti da mucosite orale indotta da CT e/o RT |
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E.2.2 | Secondary objectives of the trial |
Evaluate the efficacy of SAMITAL¿ in addition to standard treatment in the prevention and treatment of mucositis CT / RT-induced. Determine the pharmacokinetics of SAMITAL¿ to determine whether systemic absorption of the main active ingredients. Assess the palatability of SAMITAL¿ in different age groups |
Valutare l¿efficacia di SAMITAL¿ in aggiunta al trattamento standard nella prevenzione e nel trattamento della mucosite CT/RT indotta. Determinare la farmacocinetica di SAMITAL¿ per stabilire l¿eventuale assorbimento sistemico dei principali principi attivi. Valutare la palatabilit¿ di SAMITAL¿ nelle diverse fasce d¿et¿. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Patient is 2 to 17 years and 364 days • Karnofsky / Lansky Performance Status = 70 • Predicted life expectancy of = 6 months • Ability to take the study medication as prescribed • Willingness to undergo follow-up checks • Use of adequate contraception if patient is female of reproductive potential • Written Informed Consent.
GROUP A - therapy: • Patients suffering from mucositis CT / RT-induced, = grade 2 (WHO) whatever treatment plan CT / RT they will receive.
GROUP B - prophylaxis: Patients at risk of developing mucositis for the following reasons: a) diagnosis of acute lymphocytic leukemia, non-Hodgkin lymphoma, Ewing's sarcoma, Soft tissue sarcomas or other cancers for which is scheduled Metothrexate treatment with high doses, anthracyclines and / or Alkylating. b) Patients will receive high-dose therapy with autologous stem cell transplantation. c) Patients will receive radiotherapy to head and neck. |
· Età compresa tra 2 e 17 anni e 364 giorni · Karnofsky/Lansky Performance Status = 70 · Spettanza di vita = 6 mesi · Capacità di assumere il farmaco in studio secondo le modalità previste · Disponibilità a sottoporsi a controlli di follow-up · Utilizzo di adeguati metodi contraccettivi se in età fertile · Consenso informato scritto.
GRUPPO A – terapia: · Pazienti affetti da mucosite CT/RT indotta, di grado = 2 (WHO) qualsiasi sia il piano di cura CT/RT in corso.
GRUPPO B – profilassi: Pazienti a rischio di sviluppare mucosite per i seguenti motivi: a) Diagnosi di Leucemia Linfatica Acuta, Linfoma non Hodgkin, Sarcoma di Ewing, Sarcomi dei tessuti molli o altri tumori per i quali sia in programma trattamento con Metothrexate ad alte dosi, Antracicline e/o Alchilanti. b) Candidati a terapia ad alte dosi con trapianto di cellule staminali autologhe. c) Candidati a radioterapia nel distretto testa-collo. |
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E.4 | Principal exclusion criteria |
· Previous irradiation of the oral mucosa and / or oropharynx, larynx, hypopharynx · Contraindications to the administration of full doses of CT · Chronic use of steroids or immunosuppressive drugs · Pregnancy. · Inability by the patient (or parents / legal guardians), based on PI's opinion, to adhere to the requirements of the protocol · Presence of other medical conditions besides oncological therapies potentially causing mucositis (eg. reflux, rheumatologic diseases). |
· Precedente irradiazione delle mucose del cavo orale e/o dell’orofaringe, laringe, ipofaringe · Controindicazioni alla somministrazione di dosi piene di CT · Uso cronico di farmaci steroidi o immunosoppressori · Gravidanza. · Impossibilità da parte del paziente (o dei genitori/tutori legali), previo parere da parte del PI, di aderire ai requisiti dello Studio · Presenza di altre condizioni cliniche oltre le terapie oncologiche in grado di causare mucosite (es. reflusso, malattie reumatologiche). |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint of this study is the feasibility of SAMITAL® administration, defined as the ability of children, especially younger ones, to assume the product with intact or damaged oral mucosa. The primary endpoint includes: · Evaluation of the compliance of the enrolled patients · Evaluation of DLT for each dose-escalation. |
L’endpoint primario di questo studio è la fattibilità della somministrazione di SAMITAL®, definita come capacità dei bambini, in particolare quelli più piccoli, di assumere il prodotto per via orale a mucosa integra o danneggiata. L’endpoint primario include: · Valutazione della compliance dei pazienti arruolati · Valutazione delle DLT per ogni dose-escalation. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At each scheduled visit as per flow-chart |
Ad ogni visita programmata come da flow-chart |
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E.5.2 | Secondary end point(s) |
Assesment of mucositis severity and of pain; Determination of plasma concentration curves of the active ingredients of Samital; Evaluation of palatability of IP assessed with Likert with age-appropriate visual-analogical scale |
Valutazione della gravit¿ della mucosite e valutazione del dolore; Determinazione delle curve di concentrazione plasmatica dei principi attivi di Samital nel tempo; Valutazione della palatabilit¿ dell'IP secondo metodo likert con scale visivo-analogiche appropriate all'et¿ |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At each scheduled visit; Predose, after 30 minutes, 24 hours, 96 hours, 168 hours; The day before start of tretment and after 7 and 14 days |
Ad ogni visita programmata; Predose, dopo 30 minuti, 24 ore, 96 ore e 168 ore; Il giorno prima della somministrazione e dopo 7 e 14 giorni dall'inizio del trattamento |
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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 | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Palatability |
Palatabilit¿ |
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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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | 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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.5.1 | Number of sites anticipated in the EEA | 1 |
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 | Information not present in EudraCT |
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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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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |