JPRN-jRCTs031230310
Recruiting
Phase 2
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
akazawa Hideyuki0 sites5 target enrollmentAugust 28, 2023
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- akazawa Hideyuki
- Enrollment
- 5
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •(1\) Common criteria of acquired chronic pure red cell aplasia (PRCA): the diagnosis referred to a clinical guide for PRCA by a Japanese National Research Group on Idiopathic Bone Marrow Filure Syndromes of the Ministry of Health, Labour, and Welfare, revised in 2021\.
- •(2\) Hemoglobin (Hb) level of less than 9 g/dL within 4 weeks before informed consent
- •(3\) At least one of the indications listed below (a) to (d):
- •(a) a refractory disease: anemia being refractory to cyclosporine (CsA) treatment with a full dose for preceding \>\= months
- •(b) an intolerant disease: having CsA treatment being withdrawn due to its adverse events (ex. renal toxicity, hypertension)
- •(c) an intoerant disease: withholding CsA treatment due to its adverse events (ex. renal toxicity, hypertension)
- •(d) a relapsed disease: anemia being relapsing due to dose reduction or cesation of CsA treatment after clinical response to CsA
- •(4\) Age 18 years or above
- •(5\) Written informed consent
Exclusion Criteria
- •Exclusion criterias:
- •(1\) A woman during pregnancy or breastfeeding, or a man or woman without consent to contraception until 12 weeks after last dose of administration of the study drug
- •(2\) Clinical symptoms of interstitial lung disease such as cough, dyspnea, or fever
- •(3\) A medical history of sirolimus or other mTOR inhibitors
- •(4\) A medical history of immunosuppressive therapy (cyclosporine, corticosteroid, cyclophosphamide, etc.) for acquired chronic pure red cell aplasia within 5 weeks before consent.
- •(5\) A major surgery, requiring red blood cell transfusion, such as a surgery of a body cavity or a surgery with 3 or more sutures (including, biopsy), being scheduled between 2 weeks prior to the first dose of the study drug and 1 year after the consent
- •(6\) A hematopoietic stem cell transplantation scheduled within 6 months of the consent
- •(7\) Any of the laboratory results within 4 weeks of the consent; platelet count \< 75,000/mm3, neutrophil count \< 1,000/mm3, of white blood cell count \< 2,500/mm3
- •(8\) AST or ALT \> 3 times the upper limit of the reference range within 4 weeks of the consent
- •(9\) Child\-Pugh C
Outcomes
Primary Outcomes
Not specified
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