Autosomal dominant polycystic kidney and liver disease
Conditions
Brief summary
% of treatment stop due to leucopenia, % of treatment stop due to liver function distrubances, % of treatment stop due to subjective intolerance of treatment, Number of infections, Patient-reported outcome measures (PROs): Validated questionnaires to assess quality of life (QoL) and symptom burden (e.g. abdominal discomfort, fatigue, physical limitation) will be completed at baseline, week 8, week 32, week 56 and week 60.
Detailed description
Log-transformed annualised relative change of height adjusted total kidney volume (htTKV) and ht total liver volume (htTLV) (%/year) from baseline to week 56 of treatment, Change in eGFR from baseline eGFR to week 52 of treatment
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| % of treatment stop due to leucopenia, % of treatment stop due to liver function distrubances, % of treatment stop due to subjective intolerance of treatment, Number of infections, Patient-reported outcome measures (PROs): Validated questionnaires to assess quality of life (QoL) and symptom burden (e.g. abdominal discomfort, fatigue, physical limitation) will be completed at baseline, week 8, week 32, week 56 and week 60. | — |
Secondary
| Measure | Time frame |
|---|---|
| Log-transformed annualised relative change of height adjusted total kidney volume (htTKV) and ht total liver volume (htTLV) (%/year) from baseline to week 56 of treatment, Change in eGFR from baseline eGFR to week 52 of treatment | — |