genetic anemia
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1. Japanese with 20 to 80 years of age 2. Patients diagnosed as purine metabolism abnormalities, hemoglobinopathies, or red blood cell membrane abnormalities such as stomatocytosis with hemolytic anemia including adenylate kinase deficiency or red cell adenosine deaminase overproduction or genetic enzyme deficiencies in glycolysis system such as pyruvate kinase deficiency, glucose phosphate isomerase deficiency, phosphofructokinase deficiency and phosphoglycerate kinase deficiency. 3. Patients with hemoglobin concentration of 10.0 mg/dL or lower as determined within 2 months before the first day of the drug administration. 4. Patients with lower haptoglobin concentration than the normal range as determined within 2 months before the first day of the drug administration.
Exclusion criteria
Exclusion criteria: 1. Subjects taking azathioprine or mercaptopurine 2. Subject who has or had renal function disorder. 3. Subject with a history of gout, hyperuricemia or urolithiasis. 4. Subject who has or had hypersensitivity, idiosyncracy (allergy) to drug. 5. Subjects to whom either transfusion or surgical treatment such as splenectomy was made within 3 months before the initiation of the drug therapy. 6. Subject who was judged not to be appropriate by the principal investigator or sub investigators by other reasons.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Difference of serum haptoglobin between before and after the treatment | — |
Countries
Japan
Contacts
Tsukuba International Clinical Pharmacology Clinic Division for Volunteers