UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000021407 |
|---|---|
| Receipt number | R000022716 |
| Scientific Title | A study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemia |
| Date of disclosure of the study information | 2016/03/15 |
| Last modified on | 2019/09/11 10:00:13 |
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Basic information
Public title
A study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemia
Acronym
Efficacy and safety of 5-ALA phosphate in patients with sideroblastic anemia
Scientific Title
A study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemia
Scientific Title:Acronym
Efficacy and safety of 5-ALA phosphate in patients with sideroblastic anemia
Region
| Japan |
Condition
Condition
sideroblastic anemia
Classification by specialty
| Medicine in general | Hematology and clinical oncology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
Investigate the efficacy and safety of 5-aminolevulinic acid phosphate in a few patients with sideroblastic anemia.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Others
Developmental phase
Not applicable
Assessment
Primary outcomes
Efficacy (improvement of the hemoglobin value) at 12 weeks after the initiation of the treatment.
Key secondary outcomes
Safety until 8 weeks after the discontinuation of the treatment.
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
Orally intake a 150 mg 5-aminolevulinic acid phosphate capsule once a day for up to 24 weeks.
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
Individuals with sideroblastic anemia willing to give written informed consent for study
participation
Key exclusion criteria
Platelet count less than 100,000/micro L.
Neutrophil count less than 1500/micro L.
Target sample size
5
Research contact person
Name of lead principal investigator
| 1st name | Akifumi |
| Middle name | |
| Last name | Takaori-Kondo |
Organization
Graduate School of Medicine, Kyoto University
Division name
Department of Hematology and Oncology
Zip code
606-8507
Address
54 Shogo-in Kawaharacho, Sakyo-ku, Kyoto
TEL
075-751-3150
atakaori@kuhp.kyoto-u.ac.jp
Public contact
Name of contact person
| 1st name | Hiroshi |
| Middle name | |
| Last name | Kawabata |
Organization
Graduate School of Medicine, Kyoto University
Division name
Department of Hematology and Oncology
Zip code
606-8507
Address
54 Shogo-in Kawaharacho, Sakyo-ku, Kyoto
TEL
075-751-4964
Homepage URL
http://www.kuhp.kyoto-u.ac.jp/~hemonc/research/trial.html
hkawabat@kuhp.kyoto-u.ac.jp
Sponsor or person
Institute
Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University
Institute
Department
Personal name
Funding Source
Organization
Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University
Organization
Division
Category of Funding Organization
Self funding
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
SBI Pharmaceuticals Co., Ltd
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Kyoto University Graduate School and Faculty of Medicine, Ethics Committee
Address
Yoshida Konoe-cho, Sakyo-ku, Kyoto
Tel
075-753-4680
ethcom@kuhp.kyoto-u.ac.jp
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
京都大学医学部附属病院（京都府）
Other administrative information
Date of disclosure of the study information
| 2016 | Year | 03 | Month | 15 | Day |
Related information
URL releasing protocol
https://upload.umin.ac.jp/cgi-open-bin/ctr/ctr.cgi?function=brows&action=brows&recptno=R000022716
Publication of results
Unpublished
Result
URL related to results and publications
https://upload.umin.ac.jp/cgi-open-bin/ctr/ctr.cgi?function=brows&action=brows&recptno=R000022716
Number of participants that the trial has enrolled
4
Results
Totally 4 patients (2 with hereditary sideroblastic anemia and 2 with MDS-RS) were enrolled after written informed consent. Two patients took ALA for 12 weeks and the other 2 took it for 12 weeks. None of these patients responded to the ALA treatment according to our response criteria. No treatment-related adverse events were reported.
Results date posted
| 2019 | Year | 09 | Month | 11 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Totally 4 patients (2 with hereditary sideroblastic anemia and 2 with MDS-RS) were enrolled after written informed consent.One was male and the other 3 were female.
Participant flow
Totally 4 patients were enrolled after written informed consent. Two patients took ALA for 12 weeks and the other 2 took it for 12 weeks.
Adverse events
A compression fracture occurred in one elderly patient just after the initiation of the treatment. However, this patient already had severe lumbago at the point of enrollment; therefore, we speculated that the fracture was not the side-effect. No other adverse events were reported.
Outcome measures
As the primary endpoint, efficacy (improvement of the hemoglobin value >1g/dL) at 12 weeks after the initiation of the treatment was evaluated. None of these patients responded to the ALA treatment according to our response criteria.
Secondary endpoint was safety of this treatment. A compression fracture occurred in one elderly patient just after the initiation of the treatment. However, this patient already had severe lumbago at the point of enrollment; therefore, we speculated that the fracture was not the side-effect. No other adverse events were reported.
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
No longer recruiting
Date of protocol fixation
| 2016 | Year | 02 | Month | 26 | Day |
Date of IRB
| 2016 | Year | 02 | Month | 26 | Day |
Anticipated trial start date
| 2016 | Year | 03 | Month | 15 | Day |
Last follow-up date
| 2018 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
| 2019 | Year | 03 | Month | 31 | Day |
Other
Other related information
Management information
Registered date
| 2016 | Year | 03 | Month | 09 | Day |
Last modified on
| 2019 | Year | 09 | Month | 11 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000022716
| Research Plan | |
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| Registered date | File name |
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