E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Multiple myeloma |
Multiples Myelom |
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E.1.1.1 | Medical condition in easily understood language |
Plasma cell myeloma; cancer of the blood-forming system |
Plasmozytom; Krebserkrankung des blutbildenden Systems |
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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 | LLT |
E.1.2 | Classification code | 10028228 |
E.1.2 | Term | Multiple myeloma |
E.1.2 | System Organ Class | 100000054086 |
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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 |
The primary endpoint of the study is chronic GvHD and progression-free survival at 2 years after transplantation. |
Das primäre Studienziel ist die chronische GvHD und das progressionsfreie Überleben 2 Jahre nach allogener Stammzelltransplantation |
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E.2.2 | Secondary objectives of the trial |
- to evaluate the incidence of acute and chronic graft-versus-host disease at 2-years, the 2-year risk of non-relapse mortality, the 2-year progressive-free, and overall survival in patients who received a toxicity-reduced conditioning regimen combined of thiotepa and busulfan followed by allogeneic stem cell transplantation from matched or mismatched, related or unrelated donor,and cyclophosphamide as post-transplant GvHD prophylaxis
- to determine toxicity and safety of cyclophosphamide as GvHD prophylaxis |
• Inzidenz einer akuten und chronischen GvHD nach 2 Jahren, Nicht-rezidiv bedingte Sterblichkeit nach 2 Jahren, progressionsfreies Überleben nach 2 Jahren und Gesamtüberleben bei Patienten, welche eine dosisreduzierte Konditionierung (Thiotepa und Busul-fan) vor einer allogenen Stammzelltransplantation (passender oder nichtpassender Familien- bzw. Fremdspender) und Cyclophosphamide als GvHD Prophylaxe nach der Stammzelltransplantation erhal-ten haben • Erfassen von Toxizität und Sicherheit von Cyclo-phosphamid als GvHD Prophylaxe
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Multiple myeloma newly diagnosed with deletion 17p or translocation 4;14 or multiple myeloma with 1. or 2. relapse after autologous stem cell transplantation - Patients age: 18 - 65 years at time of inclusion (female and male) -Performance status ECOG < 2 - Availability of haploidentical, matched or mismatched relative or unrelated donor - Patients understand and voluntarily sign an informed consent |
1. Neu diagnostiziertes Multiples Myelom mit Deletion 17p oder Translokation 4;14 oder Multiples Myelom im 1. oder 2. Rezidiv nach einer autologen Stamm-zelltransplantation 2. Männer und Frauen zwischen 18 und 65 Jahren zum Zeitpunkt des Einschlusses 3. ECOG-Leistungsstatus von ≤ 2 4. Vorhandensein von einem haploidenten, passenden oder nicht passenden Familien - bzw. Fremdspender 5. Fähigkeit des Patienten die Patienteninformation in-haltlich zu verstehen und die Einwilligungserklärung eigenständig und freiwillig zu unterschreiben.
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E.4 | Principal exclusion criteria |
-Multiple Myeloma newly diagnosed patients without detection of deletion 17p or translocation 4;14 -Severe active infection or other uncontrolled severe condition-ing -Severe renal, hepatic, pulmonary or cardiac disease, such as: • Total bilirubin, SGPT or SGOT > 3 times upper the normal level • Left ventricular ejection fraction < 30 % • Creatinine clearance < 30 ml/min • DLCO < 35 % and/or receiving supplementary continuous ox-ygen -Positive serology for HIV -Pregnant or lactating women (positive serum pregnancy test) - Age < 18 and > 65 years. - Uncontrolled invasive fungal infection at time of screening (baseline) - Serious psychiatric or psychological disorders - Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment
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1. Neu diagnostiziertes Multiples Myelom ohne Deletion 17p oder Translokation 4;14 2. Schwere aktive Infektionen oder andere unkontrol-lierte schwere Erkrankungen 3. Schwere renale, hepatische, pulmonale oder kardiale Erkrankungen, wie z. B. • Total Bilirubin, SGPT oder SGOT > 3 x des oberen Normwertes • Linksventrikuläre Ejektionsfraktion < 30 % • Kreatinin Clearance < 30 ml/min • DLCO < 35 % und/oder ständige Sauerstoffzufuhr 4. HIV – Positivität (Serologie) 5. Schwangere oder stillende Frauen (positiver Serum Schwangerschaftstest) 6. Patientenalter < 18 und > 65 Jahre zum Zeitpunkt des Einschlusses 7. Unkontrollierte invasive Pilzinfektion zum Zeitpunkt des Screening (Baseline) 8. Schwere psychiatrische oder psychologische Er-krankungen 9. Teilnahme an einer anderen klinischen Studie mit ei-nem nichtzugelassenem Prüfpräparat 28 Tage vor Studieneinschluss
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E.5 End points |
E.5.1 | Primary end point(s) |
Chronic GvHD and progression-free survival at 2 years after allogeneic SCT |
chronische GvHD und das progressionsfreie Überleben 2 Jahre nach allogener Stammzelltransplantation |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
2 years after allogeneic SCT |
2 Jahre nach allogener Stammzelltransplantation |
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E.5.2 | Secondary end point(s) |
1. NRM at 2 years after allogeneic SCT 2. Incidence of acute GvHD on Day +100 after allogeneic SCT 3. Incidence of chronic GvHD at 1 and 2 years after allogeneic SCT 4. Toxicity according to NCI 5. Complete remission rate (including sCR and MRD negativity) 6. Overall and progression-free survival at 2 years |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. NRM at 2 years after allogeneic SCT 2. Incidence of acute GvHD on Day +100 after allogeneic SCT 3. Incidence of chronic GvHD at 1 and 2 years after allogeneic SCT 4. Toxicity according to NCI 5. Complete remission rate (including sCR and MRD negativity) 6. Overall and progression-free survival at 2 years |
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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 | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
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 | 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 | 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 | 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 | Yes |
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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LVLS |
Letzte Nachuntersuchung des letzten Patienten |
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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 | 4 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |