acquired chronic pure red cell aplasia pure red cell aplasia
Conditions
Interventions
-When starting treatment with sirolimus, the dosage administered at the end of the previous trial should be prescribed as a general rule.
-The maximum dose of sirolimus is 4.0 mg per day.
-The dosage
D000284
Sponsors
Nakazawa Hideyuki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) Patients with acquired chronic pure red cell aplasia who have completed 52-week participation in "A pilot study of the long-term safety and efficacy of oral sirolimus in patients with relapsed or refractory acquired chronic pure red cell aplasia (A pilot study of sirolimus for aPRCA)" , or patients with acquired chronic pure red cell aplasia who have completed 52-week participation in the "Sirolimus for acquired chronic pure red cell aplasia relapsed/refractory to cyclosporine A: a randomised, double-blind, placebo-controlled, multicenter phase III clinical trial (Sirolimus for acquired chronic pure red cell aplasia (PRCA) relapsed/refractory to cyclosporine A: a randomised, double-blind, placebo-controlled, multicenter phase III clinical trial )" (2) Patients deemed by the principal investigator or sub-investigator to have clinical benefit and appropriate tolerability (3) Patients who have received adequate explanation regarding participation in this study and have provided written informed consent based on their own free will.
Exclusion criteria
Exclusion criteria: (1) Patients scheduled to undergo hematopoietic stem cell transplantation within two months of obtaining consent (2) Patients exhibiting adverse events 1) to 5) that constitute criteria for discontinuation at the time of obtaining consent 1) Sirolimus pneumonitis or suspected sirolimus pneumonitis 2) Myocarditis or pericardial effusion 3) AST or ALT exceeding three times the upper limit of the facility's reference range 4) Child-Pugh classification at stage C 5) Grade 4 adverse events (3) Patients who cannot obtain consent for contraception from the time of obtaining written consent until 12 weeks after the end of investigational drug administration (4) Other patients deemed unsuitable by the principal investigator or other study staff
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| long term safety: frequency of adverse events | — |
Secondary
| Measure | Time frame |
|---|---|
| Long-term efficacy 1. Change in hemoglobin (Hb) levels and reticulocyte counts between baseline (0 weeks) and 52 weeks or at the time of discontinuation 2. Trend in hemoglobin levels and reticulocyte counts 3. Trend in sirolimus dosage 4. Dosage reduction and reasons for reduction 5. Trend in blood transfusion volume 6. Presence or absence of recurrence/relapse of erythroblastopenia and timing 7. Sirolimus serum concentration at the time of relapse 8. Trends in white blood cell count and differential, and platelet count 9. Trends in ferritin levels 10 Trends in transaminase levels 11.Trends in serum creatinine and eGFR levels, and urinary test abnormalities | — |
Contacts
Public ContactHideyuki Nakazawa
Shinshu University Hospital
Outcome results
None listed