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	NCT02233257 (ClinicalTrials.gov)	August 2011	5/5/2014	Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy	Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy	X-linked Adrenoleukodystrophy	Drug: Lorenzo's Oil	University of Minnesota	NULL	No longer available	18 Months	18 Years	Male			United States
2	NCT00545597 (ClinicalTrials.gov)	March 2005	16/10/2007	A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy	A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy	Adrenomyeloneuropathy;Adrenoleukodystrophy	Drug: Lorenzo's oil	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Food and Drug Administration (FDA)	Terminated	18 Years	N/A	Both	240	Phase 3	United States
3	NCT00004418 (ClinicalTrials.gov)	April 1998	18/10/1999	Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy	Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy	Adrenoleukodystrophy	Drug: glyceryl trierucate /glyceryl trioleate	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.	NULL	Terminated	18 Months	8 Years	Male	126	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

NCT02233257
(ClinicalTrials.gov)

August 2011

5/5/2014

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

Drug: Lorenzo's Oil

University of Minnesota

NULL

No longer available

18 Months

18 Years

Male

United States

NCT00545597
(ClinicalTrials.gov)

March 2005

16/10/2007

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

Adrenomyeloneuropathy;Adrenoleukodystrophy

Drug: Lorenzo's oil

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Food and Drug Administration (FDA)

Terminated

18 Years

N/A

Both

240

Phase 3

United States

NCT00004418
(ClinicalTrials.gov)

April 1998

18/10/1999

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy

Adrenoleukodystrophy

Drug: glyceryl trierucate /glyceryl trioleate

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

NULL

Terminated

18 Months

8 Years

Male

126

Phase 2

United States