The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia
Not Applicable
Completed
- Conditions
- Sideroblastic anemia (congenital and acquired)
- Registration Number
- JPRN-UMIN000023490
- Lead Sponsor
- Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- All
- Target Recruitment
- 5
Inclusion Criteria
Not provided
Exclusion Criteria
1) Thrombocytopenia (less than 100,000), neutropenia (less than 1,500) 2)Patients who do not understand the study. 3) Serious complication (renal insufficiency with Ccr < 30 ml/min, liver insufficiency with total bilirubin >2 mg/dL, hypoxia requiring home oxygen therapy)
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To evaluate if ALA responds to the therapy, hemoglobin levels at 12 and 24 weeks will be compared with the data at pre-treatment. Effectiveness is defined as more than 1g/dL increase in hemoglobin level (if the case undergoes regular red blood cell transfusion, the effectiveness is defined as achieving transfusion withdrawal.).
- Secondary Outcome Measures
Name Time Method Onset of adverse event is evaluated at 2 weeks, 4 weeks, and thereafter every 4 weeks up to 12 months. The grade is defined according o the Common Terminology Criteria for Adverse Events (CTCAE) v4 guideline.