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	NCT03196765 (ClinicalTrials.gov)	August 2018	5/5/2017	Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy	Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD)	X-Linked Adrenoleukodystrophy	Drug: Sobetirome (NV1205)	NeuroVia, Inc.	NULL	Withdrawn	4 Years	18 Years	Male	0	Phase 1;Phase 2	Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom
2	EUCTR2017-001684-21-GB (EUCTR)	20/04/2018	26/10/2017	Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)	Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)	Childhood Cerebral Adrenoleukodystrophy (CCALD);Therapeutic area: Diseases [C] - Hormonal diseases [C19]		Neurov Acquisition LLC	NULL	Not Recruiting			Female: no Male: yes	25	Phase 1	France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom
3	EUCTR2017-001684-21-FR (EUCTR)		23/03/2018	Phase 1, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)	Phase 1, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)	Childhood Cerebral Adrenoleukodystrophy (CCALD);Therapeutic area: Diseases [C] - Hormonal diseases [C19]		NeuroVia, Inc.	NULL	NA			Female: no Male: yes	25	Phase 1	France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom

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

NCT03196765
(ClinicalTrials.gov)

August 2018

5/5/2017

Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD)

X-Linked Adrenoleukodystrophy

Drug: Sobetirome (NV1205)

NeuroVia, Inc.

NULL

Withdrawn

4 Years

18 Years

Male

Phase 1;Phase 2

Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom

EUCTR2017-001684-21-GB
(EUCTR)

20/04/2018

26/10/2017

Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)

Childhood Cerebral Adrenoleukodystrophy (CCALD);Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Neurov Acquisition LLC

NULL

Not Recruiting

Female: no
Male: yes

Phase 1

France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom

EUCTR2017-001684-21-FR
(EUCTR)

23/03/2018

Phase 1, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD)

Childhood Cerebral Adrenoleukodystrophy (CCALD);Therapeutic area: Diseases [C] - Hormonal diseases [C19]

NeuroVia, Inc.

NULL

Female: no
Male: yes

Phase 1

France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom