DDrare
Disease
Drug
Target Gene/Pathway
問合せ先
EN
20. 副腎白質ジストロフィー
Adrenoleukodystrophy
Clinical trials
/
Disease details
臨床試験数
:
61
/
薬物数
:
90
- (
DrugBank
:
31
) /
標的遺伝子数
:
23
-
標的パスウェイ数
:
126
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No.
TrialID
Date_
enrollment
Date_
registration
Public_title
Scientific_title
Condition
Intervention
Primary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
Phase
Countries
1
NCT01043640
(
ClinicalTrials.gov
)
December 2009
5/1/2010
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal Disorders
Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Man ...
Drug:
Campath
-1H
;Drug:
Cyclophosphamide
;Drug:
Busulfan
;Procedure: Allogeneic stem cell transplantation;Drug:
Cyclosporine
A
;Drug:
Mycophenolate
Mofetil
Drug:
Campath
-1H
;Drug:
Cyclophosphamide
;Drug:
Busulfan
;Procedure: Allogeneic stem cell transplantati ...
Masonic Cancer Center, University of Minnesota
NULL
Completed
N/A
21 Years
All
46
Phase 2
United States
2
NCT00176904
(
ClinicalTrials.gov
)
January 1995
12/9/2005
Stem Cell Transplant for Inborn Errors of Metabolism
Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation
Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Gaucher's Disease;Fucosidosis;Wolman Disease;Niemann-Pick Disease;Batten Disease;GM1 Gangliosidosis;Tay Sachs Disease;Sandhoff Disease
Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Gaucher's Disease;Fuco ...
Procedure: Stem Cell Transplant;Drug:
Busulfan
,
Cyclophosphamide
, Antithymocyte Globulin
Masonic Cancer Center, University of Minnesota
NULL
Completed
N/A
N/A
All
135
Phase 2/Phase 3
United States