DDrare: Database of Drug Development for Rare Diseases

20. 副腎白質ジストロフィー
［臨床試験数：49，薬物数：86（DrugBank：29），標的遺伝子数：18，標的パスウェイ数：112］

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 5-[4-[2-(5-(1-hydroxyethyl)-2- pyridinyl)ethoxy]benzyl]-2,4- thiazolidinedione hydrochloride; 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride; 5-[[4-[2-[5-(1-HYDROXYETHYL)-2-PYRIDINYL]ETHOXY]PHENYL]METHYL]-2,4-THIAZOLIDINEDIONE HYDROCHLORIDE; ALBUMIN; AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA; Acetylcysteine; Albumin; Albumin solution; Albunorm® 5%,; Alemtuzumab; Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA; Bezafibrate; Biotin; Busulfan; Busulfan, Cyclophosphamide, Antithymocyte Globulin; CALD HR-D (High-Risk, Regimen C); CALD HR-D (High-Risk, Regimen D); CALD SR-A (Standard-Risk, Regimen A); CALD SR-B (Standard-Risk, Regimen B); Campath; Campath-1H; Chenodeoxycholic acid; Cholic Acid; Cholic Acids; Cholic acid; Clofarabine; Cyclophosphamide; Cyclosporine; Cyclosporine A; D-BIOTIN; DUOC-01; Device: Cyclosporine A; Elivaldogene autotemcel; Elivaldogenum tavalentivecum; Glyceryl Trierucate; Glyceryl Trioleate; Glyceryl trierucate; Glyceryl trierucate/glyceryl trioleate; Glyceryl trioleate; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cell infusion; Human Placental Derived Stem Cell; Hydroxyurea; IMD; IMD Preparative Regimen; Interferon beta; Lenti-D; Lenti-D Drug Product; Leriglitazone; Lipoic acid; Lorenzo's Oil; Lorenzo's oil; MD1003; MD1003 100 mg capsule; MIN-102; Melphalan; Mesenchymal Stem Cells; Modified cobratoxin; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Mycophenylate mofetil; N-acetylcysteine; NV1205; Osteopetrosis Haploidentical Only Preparative Regimen; Osteopetrosis Only Preparative Regimen; PIOGLITAZONE; PLERIXAFOR; Pioglitazone; Plerixafor; Procedure: Allogeneic stem cell transplantation; Procedure: Stem Cell Transplant; Procedure: Total body Irradiation; QTXEW2-04-AF, EW2, GC-1, QRX-431; RPI78M for injection; Sobetirome; Sobetirome (NV1205); Stem Cell Transplantation; Thalidomide; Thiazolidinedione; Thiotepa; Thiotepa--escalated dose; Thiotepa--single daily dose; Ursodiol; Vitamin D3; Vitamin E

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	NCT01372228 (ClinicalTrials.gov)	April 2011	10/6/2011	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders	Hurler Syndrome (MPS I);Hurler-Scheie Syndrome;Hunter Syndrome (MPS II);Sanfilippo Syndrome (MPS III);Krabbe Disease (Globoid Leukodystrophy);Metachromatic Leukodystrophy (MLD);Adrenoleukodystrophy (ALD and AMN);Sandhoff Disease;Tay Sachs Disease;Pelizaeus Merzbacher (PMD);Niemann-Pick Disease;Alpha-mannosidosis	Biological: hematopoietic stem cell infusion	Talaris Therapeutics Inc.	Duke University	Active, not recruiting	N/A	N/A	All	30	Phase 1;Phase 2	United States

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

NCT01372228
(ClinicalTrials.gov)

April 2011

10/6/2011

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Hurler Syndrome (MPS I);Hurler-Scheie Syndrome;Hunter Syndrome (MPS II);Sanfilippo Syndrome (MPS III);Krabbe Disease (Globoid Leukodystrophy);Metachromatic Leukodystrophy (MLD);Adrenoleukodystrophy (ALD and AMN);Sandhoff Disease;Tay Sachs Disease;Pelizaeus Merzbacher (PMD);Niemann-Pick Disease;Alpha-mannosidosis

Biological: hematopoietic stem cell infusion

Talaris Therapeutics Inc.

Duke University

Active, not recruiting

N/A

All

Phase 1;Phase 2

United States