Rare renal diseases linked to monoclonal gammopathy MedDRA version: 20.0 Level: SOC Classification code 10038359 Term: Renal and urinary disorders System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Trade Name: Darzalex, daratumumab
Pharmaceutical Form: Injection
Sponsors
Helsinki University Central Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Males or females = 18 years of age • Subject has provided informed consent prior to initiation of the study or subject’s legally acceptable representative has provided informed consent prior to the study when the subject has any kind of condition that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent. • Renal biopsy confirmed MGRS-disease. o Renal biopsy must not be older than 3 months before informed consent o Renal transplant patients are allowed • Amount of proteinuria = 500 mg/24 h OR eGFR = 20 ml/min prior to the study • Previous anticlonal treatment is allowed if deemed ineffective (no formal time required) Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 25
Exclusion criteria
Exclusion criteria: • Myeloma or systemic AL amyloidosis (smoldering myeloma sized plasma cell clone is allowed when in association with a documented MGRS condition and AHL amyloidosis and AH amyloidosis are included), • Cancer that requires treatment, • MGRS related to B-cell malignant disorders, • Known HIV infection, active hepatitis C infection (subjects with hepatitis C that achieve a sustained virologic response after antiviral therapy are allowed), or hepatitis B infection (subjects with hepatitis B surface antigen or core antibody that achieve sustained virologic response (PCR negativity in HBVNh) with antiviral therapy are permitted with a requirement for regular monitoring for reactivation for the duration of treatment on the study), • Pregnancy or breastfeeding, • No cyclophosphamide within 6 months of enrollment, or oral high-dose prednisone or equivalent within 6 weeks of enrollment; • prednisone or its equivalent at a dosage of =10 mg daily for a condition unrelated to MGRS (e.g. asthma or gout) allowed. • mycophenolate mofetil (MMF), calcineurin inhibitors (CNI) or azathioprine treated patients are eligible if proteinuria not improving or if kidney function declining despite treatment with these medications. Once therapy with daratumumab started, these medications need to be discontinued unless they are used as immunosuppressive medication due to renal transplantation. • In patients who previously received rituximab, reconstitution of B cells (CD19 normalized, Ly-B-CD19 lab.code 8329) required, • Inability to use daratumumab and to comply with the study protocol as assessed by treating nephrologist and/or hematologist (e.g. severe psychiatric illness, severe lung disease, known allergy to daratumumab)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Rate of complete renal remission (proteinuria 50% reduction in 24-h proteinuria and 50% reduction in 24-h proteinuria and 50 % from baseline to EOT. ;Primary end point(s): Renal (changes in eGFR and amount of proteinuria) and hematological (sFLCs and MRD) endpoints;Timepoint(s) of evaluation of this end point: at 6 and 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Rate of complete renal remission (proteinuria 50% reduction in 24-h proteinuria and 50 % from baseline to EOT. ;Timepoint(s) of evaluation of this end point: At 6 and 12 months | — |
Countries
Finland
Contacts
Public ContactKati Kaartinen
Helsinki University Central Hospital
Outcome results
None listed