NIPH Clinical Trials Search

Sirolimus for Relapsed or Refractory PRCA Extension Study

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	acquired chronic pure red cell aplasia
Date of first enrollment	10/11/2025
Target sample size	35
Countries of recruitment
Study type	Interventional
Intervention(s)	-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 of sirolimus is adjusted based on the clinical judgment of the principal investigator or sub-investigator, but in principle, it is adjusted in accordance with the following guidelines. (1) Continue administration to achieve maximum effect while red blood cell count or reticulocyte count is trending upward. (2) If more than 18 months have passed since the start of the SOARER-A study investigational drug or more than 12 months have passed since the start of the pilot study with sirolimus, and more than 3 months have passed since hemoglobin levels reached maximum effect, reduced doses may be administered as maintenance therapy.The maximum effect of hemoglobin levels is defined by either a) or b) below. a)If hemoglobin levels remain above the lower limit of the facility standard during any two-month period without blood transfusions, the first day of that period shall be considered the date of maximum effect. b)If the increase in hemoglobin levels is less than 1.5 g/dL before and after any two-month period without blood transfusions, the first day of that period shall be considered the date of maximum effect. (3) Unless the dose reduction is due to adverse events, the reduction should be limited to 1 mg or less per dose, and the same dose should be maintained for at least 3 months after the reduction. If recurrence is suspected during maintenance therapy, consider increasing the sirolimus dose promptly and take other measures to prevent the progression of anemia. -As a general rule, test drugs should be administered to each subject at the same time in the morning (between 7:00 and 9:00 a.m. as a guideline) and either after meals or on an empty stomach, whichever is consistent for each subject.If a dose is missed, the missed dose (one day's worth) should be administered orally as soon as it is noticed. However, if the dose is missed on one day, it should not be administered on that day, but rather the next day's dose should be administered.

Outcome(s)

Primary Outcome

long term safety: frequency of adverse events

Secondary Outcome

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

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	Not applicable
Gender	Both
Include 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.
Exclude 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