DDrare: Database of Drug Development for Rare Diseases

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_ gender	Target_ size	Phase	Countries
1	EUCTR2018-001145-14-DE (EUCTR)	23/01/2020	27/11/2018	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD)	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR	bluebird bio, Inc.	NULL	Not Recruiting	Female: no Male: yes	35	Phase 3	United States;France;Netherlands;Germany;United Kingdom;Italy
2	EUCTR2018-001145-14-NL (EUCTR)	24/06/2019	25/02/2019	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD)	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: Lenti-D Drug Product Product Code: Not Applicable INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA	bluebird bio, Inc.	NULL	Authorised-recruitment may be ongoing or finished	Female: no Male: yes	35	Phase 3	France;United States;Germany;Netherlands;Italy;United Kingdom
3	EUCTR2018-001145-14-GB (EUCTR)	17/04/2019	23/10/2018	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD).	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA	bluebird bio, Inc.	NULL	Authorised-recruitment may be ongoing or finished	Female: no Male: yes	35	Phase 3	United States;France;Netherlands;Germany;Italy;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

EUCTR2018-001145-14-DE
(EUCTR)

23/01/2020

27/11/2018

Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD)

A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD)
MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: Lenti-D Drug Product
INN or Proposed INN: elivaldogene autotemcel
Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA
INN or Proposed INN: plerixafor
Other descriptive name: PLERIXAFOR

bluebird bio, Inc.

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

United States;France;Netherlands;Germany;United Kingdom;Italy

EUCTR2018-001145-14-NL
(EUCTR)

24/06/2019

25/02/2019

Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD)

A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

Product Name: Lenti-D Drug Product
Product Code: Not Applicable
INN or Proposed INN: elivaldogene autotemcel
Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA

bluebird bio, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

Phase 3

France;United States;Germany;Netherlands;Italy;United Kingdom

EUCTR2018-001145-14-GB
(EUCTR)

17/04/2019

23/10/2018

Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD).

A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

bluebird bio, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

Phase 3

United States;France;Netherlands;Germany;Italy;United Kingdom