E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028228 |
E.1.2 | Term | Multiple myeloma |
|
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 compare the efficacy of palifermin relative to placebo when given either pre- and post-high dose chemotherapy or pre-high dose chemotherapy only with regard to the severity of oral mucositis WHO grades 0/1, 2, 3 or 4 |
|
E.2.2 | Secondary objectives of the trial |
To assess the effect of palifermin on the incidence and duration of ulcerative oral mucositis WHO grades 2, 3 and 4 and of severe mucositis WHO grades 3 and 4 To evaluate the impact of palifermin on patient-reported mouth and throat soreness MTS |
|
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
- Multiple myeloma MM subjects scheduled to receive high-dose Melphalan 200 mg/m2 if creatinine clearance 8805; 30 mL/min or 140 mg/m2 if creatinine clearance 30 mL/min , in a one day schedule day -2 followed by autologous PBSCT day 0 - Age 8805; 18 years and 8804; 70 years - BMI 8804; 35 - ECOG performance status 8804; 2, or an ECOG status of 3 if the reason for a status of 3 is due exclusively due to MM e.g. pathological fracture - Adequate pulmonary function as measured by a corrected carbon monoxide CO diffusing capacity DLCO 8805; 60 of predicted - Minimum of 2.0 x 106 CD34 cells/kg collected for autologous transplantation - Adequate hematological function ANC 8805; 1.5 x 109/L and platelet count 8805; 100 x 109/L - Total bilirubin 8804; 2 mg/dL - Aspartate aminotransferase AST and/or alanine aminotransferase ALT 8804; 4.0 x IULN - Negative serum or urine pregnancy test for women of child bearing potential within 14 days prior to enrolment - Each subject must give informed consent directly or through a legally acceptable representative before participating in any study specific procedure, or receiving any study medication |
|
E.4 | Principal exclusion criteria |
- History of or concurrent malignancy other than MM, with the exception of curatively treated basal cell or squamous cell carcinoma of the skin, in situ cervical carcinoma, or other surgically cured malignancy, without evidence of disease for 3 years - Prior treatment with palifermin, or other fibroblast or keratinocyte growth factors eg, KGF-2 - Prior autologous or allogeneic transplants - Oral abnormalities defined as baseline oral assessment of WHO grade 0 - Receiving dialysis - Twenty-eight days or less between receiving any other investigational drug or device and randomization into this study - Subject of child-bearing potential is evidently pregnant eg, positive HCG test or is breast feeding - Subject has not agreed to using adequate contraceptive precautions - Known to be sero-positive for human immunodeficiency virus HIV , hepatitis B virus HBSAg , or hepatitis C virus HCV - Unwilling or unable to complete the patient-reported outcome questionnaires - Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and/or to comply with study procedures |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Maximum severity of oral mucositis WHO grades 0/1, 2, 3 or 4 |
|
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 | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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 | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Yes |
E.8.2.3 | Other | No |
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.8.5 | The trial involves multiple Member States | Yes |
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 | Information not present in EudraCT |
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 | 10 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 10 |
E.8.9.2 | In all countries concerned by the trial months | 0 |