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	NCT02961803 (ClinicalTrials.gov)	October 2014	9/11/2016	MD1003-AMN MD1003 in Adrenomyeloneuropathy	MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study	Adrenomyeloneuropathy;Adrenoleukodystrophy;AMN	Drug: MD1003 100 mg capsule;Drug: Placebo	MedDay Pharmaceuticals SA	NULL	Completed	18 Years	60 Years	Male	67	Phase 2;Phase 3	France;Germany;Spain
2	EUCTR2014-000698-38-ES (EUCTR)	13/08/2014	13/06/2014	MD1003 IN ADRENOMYELONEUROPATHY	MD1003 IN ADRENOMYELONEUROPATHY: A RANDOMIZED DOUBLE BLIND PLACEBO CONTROLLED STUDY - MD1003-AMN	Adrenomyeloneuropathy MedDRA version: 17.0;Level: LLT;Classification code 10069075;Term: Adrenomyeloneuropathy without cerebral involvement;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: biotin Product Code: MD1003 INN or Proposed INN: D-BIOTIN	MEDDAY SAS	NULL	Not Recruiting			Female: no Male: yes	60	Phase 2;Phase 3	Spain

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

NCT02961803
(ClinicalTrials.gov)

October 2014

9/11/2016

MD1003-AMN MD1003 in Adrenomyeloneuropathy

MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study

Adrenomyeloneuropathy;Adrenoleukodystrophy;AMN

Drug: MD1003 100 mg capsule;Drug: Placebo

MedDay Pharmaceuticals SA

NULL

Completed

18 Years

60 Years

Male

Phase 2;Phase 3

France;Germany;Spain

EUCTR2014-000698-38-ES
(EUCTR)

13/08/2014

13/06/2014

MD1003 IN ADRENOMYELONEUROPATHY

MD1003 IN ADRENOMYELONEUROPATHY: A RANDOMIZED DOUBLE BLIND PLACEBO CONTROLLED STUDY - MD1003-AMN

Adrenomyeloneuropathy
MedDRA version: 17.0;Level: LLT;Classification code 10069075;Term: Adrenomyeloneuropathy without cerebral involvement;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: biotin
Product Code: MD1003
INN or Proposed INN: D-BIOTIN

MEDDAY SAS

NULL

Not Recruiting

Female: no
Male: yes

Phase 2;Phase 3

Spain