E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
patients developing acute GvHD grade I |
pazienti che hanno sviluppato aGvHD acuta di grado I |
|
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 | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000802 |
E.1.2 | Term | Acute GVH disease |
E.1.2 | System Organ Class | 100000004870 |
|
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 determine if methylprednisolone 1 mg/kg/day at the onset of grade I acute GvHD may reduce the progression rate to acute GVHD grade > 1 in patients who have undergone allogeneic SCT. |
Determinare se methylprednisolone 1 mg/kg/day all esordio della GVHd acuta di grado I puo` ridurre il tasso di progressione della GVHd a gradi >1 in pazienti sottoposti a trapianto di cellule staminali SCT |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Prior allo-SCT using either BM, PB or cord blood from related or unrelated donors. - Patients developing aGvHD after prophylactic or planned donor lymphocyte infusion (DLI) are also eligible. - Signed informed consent - Skin rash on 10-50% of body surface compatible with aGvHD and not directly attributable to other etiologies. Biopsy confirmation of aGVHD is strongly encouraged but not mandatory and in any case should not delay enrollment (see Appendix). - Patients must not have previous systemic corticosteroid therapy higher than 0,5 mg/kg/day MP (0.6 mg/kg/day prednisone) within 48 hours prior aGVHD onset. |
 Precedente trapianto allogenico con BM, PB o sangue da cordone da donatore relato o non relato.  I pazienti che hanno sviluppato aGvHD dopo infusione pianificata o profilattica di linfociti da donatore (DLI) sono eleggibili.  Consenso informato firmato  Rash cutaneo sul 10-50% della superficie corporea compatibile con aGvHD e non attribuibile ad altre patologie.  La biopsia di conferma della aGVHD e` fortemente incoraggiata ma non obbligatoria e in caso non deve essere successive alla data di arruolamento.  I pazienti non devono essere precedentemente trattati con con terapie sistemiche con cortisonici oltre 0,5 mg/kg/day methylprednisolone (0.6 mg/kg/day prednisone) nelle 48 ore precedenti l inizio della aGVHD. |
|
E.4 | Principal exclusion criteria |
- Any life-threatening infection or condition. - Evidence of hematological relapse or residual disease at the time of enrollment. - Patients who have undergone unscheduled DLI for relapse. - Other investigational immunosuppressive regimens for GvHD prophylaxis within 30 days. - Patients receiving MP >0.5 mg/kg/day within 2 days of onset of aGVHD. - Patients receiving steroids for a non-GvHD related condition are eligible only after tapering to ≤0.5 mg/kg/day for at least 48 hours. |
 Qualsiasi infezione o condizioni che rende il paziente in pericolo di vita.  Evidenza di ricaduta ematologica o residuo di malattia all arruolamento.  Pazienti che hanno ricevuto DLI per ricaduta.  Qualsiasi regime investigativo immunosoppressivo per la profilassi della GvHD negli ultimi 30 giorni.  Pazienti che hanno ricevuto methylprednisolone >0.5 mg/kg/day nei 2 giorni precedenti l esordio della aGVHD.  Pazienti che hanno ricevuto steroidi per condizioni con correlate alla GvHD sono eleggibili solo dopo diminuzione a ≤0.5 mg/kg/day per almeno 48 ore. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
- Progression rate to acute GVHD grade > 1 in the two study arms. |
Tasso di progressione della GVHD acuta di grado >1 nei due bracci di trattamento. |
|
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 | 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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
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 | 16 |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |