E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Multiple myeloma patients with acute dialysis dependent renal failure resulting from cast nephropathy. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028566 |
E.1.2 | Term | Myeloma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028569 |
E.1.2 | Term | Myelomatosis multiple |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10038436 |
E.1.2 | Term | Renal failure acute |
E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018875 |
E.1.2 | Term | Haemodialysis |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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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 objective of the study is to address the hypothesis that extended HD, using the Gambro HCO 1100, increases the percentage of patients with cast nephropathy becoming independent of dialysis. |
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E.2.2 | Secondary objectives of the trial |
1. Investigation of the efficiency of extended HD using the Gambro HCO 1100 to result in sustained reductions in sFLC concentrations versus a standard dialysis.
2 Comparison of the duration of HD before renal recovery
3 Investigation of multiple myeloma response to chemotherapy and suitability for stem cell transplantation.
4 Mortality
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Dialysis dependent acute renal failure (eGFR <15mls/min/1.73m2) • Fulfils diagnostic criteria for the diagnosis of symptomatic de novo multiple myeloma1 • Abnormal serum FLC ratio • Myeloma kidney demonstrated on a renal biopsy (cast nephropathy) • Ability to give informed consent to partake in study
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E.4 | Principal exclusion criteria |
• Known advanced chronic renal failure (CKD stage IV; eGFR <30mls/min/1.73m2) or evidence of significant chronic damage on renal biopsy • Amyloidosis or light chain deposition disease on renal biopsy • Previous treatment of multiple myeloma with chemotherapy • Haemodynamic instability that precludes unsupported dialysis • Significant cardiac disease (myocardial infarction with in 6 months; unstable angina; NYHA class III or IV heart failure; clinically significant pericardial disease; cardiac amyloidosis) • Advanced disease or significant co-morbidity: with poor short term prognosis, necessitating palliation and no active or disease specific treatment. • Inability to give informed consent • History of allergic reaction to compounds containing boron or mannitol • History of peripheral neuropathy or neuropathic pain (Grade 2 or Higher) • Clinically significant liver dysfunction (bilirubin >1.8mg/dl (30umol/L) • Known HIV infection • Active uncontrolled infection * Pregnant and lactating women
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary analysis for the trial is to compare the experimental treatment to the control treatment in terms of independence of dialysis at three months. The proportion of patients independent of dialysis will be compared using relative risks and chi-square tests. The analysis will be carried out on an intention-to-treat basis. |
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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 | 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 | 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) | 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 | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Randomised trial of a medical device (haemodialyser) not the IMP (Velcade). |
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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 | Yes |
E.8.2.3.1 | Comparator description |
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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 | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 | 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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Interim analysis will initially be carried out 3 months after the first 25 patients and then at 3 months after the recruitment of 50% of target. Results will be presented to an independent Data Monitoring Committee. The interim analysis will present recruitment data and data on outcomes and adverse events. Given the small size of the study, the interim analysis will be purely descriptive with the primary aim to assess patient safety and that it is ethical to continue. |
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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 | 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 |