A single center study to evaluate the safety and tolerability of oral Azathioprine in patients with ADPKD

Condition studied

Autosomal dominant polycystic kidney and liver disease

Investigational medicinal product(s)

Eligibility

Primary endpoint

% 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.

Endpoint detail

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