ALA ( DrugBank: - )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
180ATR−X症候群1
286遺伝性鉄芽球性貧血2

180. ATR−X症候群


臨床試験数 : 1 薬物数 : 3 - (DrugBank : 2) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-jRCT2051220062
14/07/202214/07/2022Physician-initiated clinical trial in patients with 5-aminolevulinic acid-induced ATR-X syndrome.Exploratory study to investigate the safety and efficacy of NPJ005 in patients with ATR-X syndrome. - IACT21003 ATR-X syndrome
infant, intellectual disability, epigenetics, guanine quadruplex;D008607
Containing 5-aminolevulinic acid (5-ALA) hydrochloride and sodium ferrous citrate (SFC)
Tablets (25 mg 5-ALA hydrochloride/39.2 mg SFC per tablet, NPJ005) for 24 weeks
Wada TakahitoNULLRecruiting>= 2age oldNot applicableMale5Phase 2Japan

286. 遺伝性鉄芽球性貧血


臨床試験数 : 7 薬物数 : 20 - (DrugBank : 10) / 標的遺伝子数 : 8 - 標的パスウェイ数 : 43
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-UMIN000023490
2016/08/2005/08/2016The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic AnemiaThe Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia - The Safety and Efficacy of 5-Aminolevulinic Acid (ALA) Therapy for Sideroblastic Anemia Sideroblastic anemia (congenital and acquired)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).Department of Hematolgy and Rheumatology, Tohoku University Graduate School of MedicineNULLComplete: follow-up complete20years-old80years-oldMale and Female5Not applicableJapan
2JPRN-UMIN000021407
2016/03/1515/03/2016A study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemiaA study of efficacy and safety of 5-aminolevulinic acid phosphate in patients with sideroblastic anemia - Efficacy and safety of 5-ALA phosphate in patients with sideroblastic anemia sideroblastic anemiaOrally intake a 150 mg 5-aminolevulinic acid phosphate capsule once a day for up to 24 weeks.Department of Hematology and Oncology, Graduate School of Medicine, Kyoto UniversitySBI Pharmaceuticals Co., LtdComplete: follow-up continuing20years-old80years-oldMale and Female5Not applicableJapan