UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia
Title of the study (Brief title)	The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia
Region	Japan

Condition
Condition	Sideroblastic anemia (congenital and acquired)
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To examine the safety and efficacy of 5-aminolevulinic acid (ALA) therapy for congenital and acquired sideroblastic anemia
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	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.).
Key secondary outcomes	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.

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	Food
Interventions/Control_1	Food supplement containing 150 mg of ALA phosphate was given per day for 24 weeks. For the evaluation of efficacy and safety, medical examinations as well as urine and blood tests will be performed prior to administration of ALA, then at 2 and 4 weeks, and thereafter every month (up to 12 months).
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	Patients with sideroblastic anemia (congenital and acquired), who agreed to participate in the study.
Key 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)
Target sample size	5

Research contact person
Name of lead principal investigator	Harigae Hideo
Organization	Tohoku University Graduate School of Medicine
Division name	Department of Hematolgy and Rheumatology
Address	2-1 Seiryo-cho, Aoba-ku, Sendai
TEL	022-717-7165
Email	harigae@med.tohoku.ac.jp

Public contact
Name of contact person	Fujiwara Tohru
Organization	Tohoku University Graduate School of Medicine
Division name	Department of Hematolgy and Rheumatology
Address	2-1 Seiryo-cho, Aoba-ku, Sendai
TEL	022-717-7165
Homepage URL
Email	fujiwara-to@apple.email.ne.jp

Sponsor
Institute	Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine
Institute
Department

Funding Source
Organization	Other
Organization
Division
Category of Funding Organization	Other
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	2016 Year 08 Month 05 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2016 Year 08 Month 05 Day
Anticipated trial start date	2016 Year 08 Month 20 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 08 Month 04 Day
Last modified on	2018 Year 08 Month 06 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000027057