E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Active and untreated Waldenstrom Macroglobulinemia |
Macroglobulinemia di Waldenstrom e malattia attiva non precedentemente trattata |
|
E.1.1.1 | Medical condition in easily understood language |
Waldenstrom Macroglobulinemia |
Macroglobulinemia di Waldenstrom |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10054693 |
E.1.2 | Term | Von Waldenstrom macroglobulinemia |
E.1.2 | System Organ Class | 100000004864 |
|
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 |
To assess the 1-year complete remission (CR) rate |
Valutare il tasso di risposta completa a 1 anno (CR) |
|
E.2.2 | Secondary objectives of the trial |
• To assess the safety of the R-COMP-14 chemotherapy
• To assess the disease-free survival
• To assess the overall survival |
• Valutare la sicurezza del regime chemioterapico R-COMP-14
• Valutare la sopravvivenza libera da malattia (DFS)
• Valutare la sopravvivenza globale (OS) |
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Gene expression profiling
Single nucleotide polymorphisms (SNP) analysis |
Gene expression profiling
Analisi di single nucleotide polymorphisms (SNP) |
|
E.3 | Principal inclusion criteria |
• Signed informed consent prior to any study procedures being performed
• Diagnosis of untreated Waldenstrom Macroglobulinemia
• Age >18 and <80 years.
• Karnofsky score ≥ 70% or WHO ≤1.
• Adequate renal, pulmonary and hepatic function:
Serum creatinine < 2 mg/dl;
Sat O2 ≥ 96%;
ALT/AST ≤ 2.5 x N.
• Fertile female must use adequate contraceptive precautions.
• Patients with active disease. |
• Firma del consenso informato prima di effettuare qualsiasi procedura
dello studio.
• Diagnosi di Macroglobulinemia di Waldenstrom non trattata.
• Età >18 and <80 anni.
• Karnofsky score ≥ 70% or WHO ≤1.
• Funzionalità renale, polmonare ed epatica adeguata:
Creatinina sierica < 2 mg/dl;
Sat O2 ≥ 96%;
ALT/AST ≤ 2.5 x N.
• Test di gravidanza su siero o urine negativo nei 10-14 giorni
precedenti e
a 24 ore dall'infusione e utilizzo di sistemi anticoncezionali adeguati.
• Pazienti con malattia attiva. |
|
E.4 | Principal exclusion criteria |
• People unable or unwilling to give informed consent.
• Age < 18 or > 80 years.
• HIV positivity.
• HCV positivity with high viral load..
• HBs Ag positivity.
• Pregnancy or nursing female
• Current uncontrolled infection.
• Patients with a history of other malignancies within 2 years prior to
study entry.
• Treatment with any other IMP, or participating in another clinical trial
within 30 days prior to entering this study.
• Lacking of at least one criteria of active disease. |
• Pazienti incapaci di fornire volontariamente il consenso informato
• Età< 18 or > 80 anni.
• Positività HIV.
• Positività HCV con alta carica virale.
• Positività HBs Ag.
• Donne in gravidanza o in allattamento
• Infezioni ricorrenti incontrollate.
• Pazienti con una storia di altre patologie neoplastiche nei due anni
precedenti l'entrata nello studio.
• Trattamento con IMP, o partecipazione a un'altro protocollo clinico nei
30 giorni precedenti all'entrata nello studio. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
To assess the 1-year complete remission (CR) rate. |
Valutare il tasso di risposta completa a 1 anno (CR). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
• To assess the safety of the R-COMP-14 chemotherapy
• To assess the disease-free survival
• To assess the overall survival |
• Valutare la sicurezza del regime chemioterapico R-COMP-14
• Valutare la sopravvivenza libera da malattia (DFS).
• Valutare la sopravvivenza globale (OS). |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | Yes |
E.6.4 | Safety | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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 | 1 |
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 | 10 |
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
|
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |