UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Studies of efficacy and safety of simultaneous administration of febuxostat and inosine for patients with genetic anemia, and changes of blood and urine purine compounds
Title of the study (Brief title)	Effect of febuxostat and inosine on anemia
Region	Japan

Condition
Condition	genetic anemia
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Examine the effects of simultaneous administration of febuxostat and inosine on genetic anemia, and changes of blood and urine purine compounds.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Difference of serum haptoglobin between before and after the treatment
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Treatment with febuxostat 20 mg and inosine 0.5 g twice a day for 14 days
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	80 years-old >=
Gender	Male and Female
Key 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.
Key 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.
Target sample size	6

Research contact person
Name of lead principal investigator	Naoyuki Kamatani
Organization	Tsukuba International Clinical Pharmacology Clinic
Division name	Doctors' office
Address	1-21-16 Kan-nondai Tsukuba City, Ibaraki Prefecture
TEL	029-839-1150
Email	kamatani@msb.biglobe.ne.jp

Public contact
Name of contact person	Masaki Suzuki
Organization	Tsukuba International Clinical Pharmacology Clinic
Division name	Division for Volunteers
Address	1-21-16 Kan-nondai Tsukuba City, Ibaraki Prefecture
TEL	029-839-1150
Homepage URL
Email	m-suzuki@tsukuba-icp.jp

Sponsor
Institute	Tsukuba International Clinical Pharmacology Clinic
Institute
Department

Funding Source
Organization	StaGen Co. LTD
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2017 Year 02 Month 27 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2016 Year 10 Month 24 Day
Anticipated trial start date	2016 Year 12 Month 10 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 11 Month 08 Day
Last modified on	2017 Year 05 Month 09 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000028453