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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-000654-59-FR
(EUCTR)
31/03/202021/07/2020A study to evaluate the effects of a new drug called MIN-102 on diseaseprogression in boys with cerebral X-linked adrenoleukodystrophy (cALD)An open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophy (cALD) to assess the effect of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT) Cerebral X-linked 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: 5-[4-[2-(5-(1-hydroxyethyl)-2- pyridinyl)ethoxy]benzyl]-2,4- thiazolidinedione hydrochloride
INN or Proposed INN: Leriglitazone
Other descriptive name: 5-[[4-[2-[5-(1-HYDROXYETHYL)-2-PYRIDINYL]ETHOXY]PHENYL]METHYL]-2,4-THIAZOLIDINEDIONE HYDROCHLORIDE
Minoryx Therapeutics S.L.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
13Phase 2United States;France;Spain;Germany
2NCT04303416
(ClinicalTrials.gov)
March 9, 20206/3/2020Plasma Exchange With Albumin in AMN PatientsEffect of Plasma Exchange With Albumin in Patients With Adrenomyeloneuropathy: Unicentric, Single Arm, Proof of Concept Study.Adrenomyeloneuropathy;AdrenoleukodystrophyDrug: Albumin solutionOnofre, Aurora Pujol, M.D.NULLNot yet recruiting18 Years65 YearsMale5Phase 2;Phase 3Spain
3EUCTR2019-004733-17-ES
(EUCTR)
25/02/202020/12/2019Plasma exchange by albumin replacement in AdrenomyeloneuropathyEffect of plasma exchange by albumin replacement in Adrenomyeloneuropathy: unicentric, single arm, proof of concept trial Adrenomyeloneuropathy
MedDRA version: 20.0;Level: LLT;Classification code 10069075;Term: Adrenomyeloneuropathy without cerebral involvement;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Aurora Pujol OnofreNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
5Phase 2Spain
4EUCTR2018-001145-14-DE
(EUCTR)
23/01/202027/11/2018Clinical 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.NULLNot RecruitingFemale: no
Male: yes
35Phase 3United States;France;Netherlands;Germany;United Kingdom;Italy
5NCT04528706
(ClinicalTrials.gov)
September 13, 201931/7/2020A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)Cerebral AdrenoleukodystrophyDrug: MIN-102Minoryx Therapeutics, S.L.NULLRecruiting2 Years12 YearsMale13Phase 2France;Germany;Spain
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-000654-59-ES
(EUCTR)
25/06/201930/04/2019A study to evaluate the effects of a new drug called MIN-102 on the progression of brain lesions in boys with cerebral X-linked adrenoleukodystrophy (cALD)An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions. Cerebral X-linked 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Minoryx Therapeutics S.L.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
10Phase 2Spain;Germany
7EUCTR2018-001145-14-NL
(EUCTR)
24/06/201925/02/2019Clinical 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
35Phase 3France;United States;Germany;Netherlands;Italy;United Kingdom
8EUCTR2018-001145-14-GB
(EUCTR)
17/04/201923/10/2018Clinical 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
35Phase 3United States;France;Netherlands;Germany;Italy;United Kingdom
9EUCTR2018-001145-14-FR
(EUCTR)
27/03/201923/10/2018Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy.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]
bluebird bio, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
20Phase 3United States;France;Netherlands;Germany;Italy;United Kingdom
10NCT03852498
(ClinicalTrials.gov)
January 24, 201913/2/2019Clinical 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)Drug: Lenti-D; elivaldogene autotemcelbluebird bioNULLRecruitingN/A17 YearsMale35Phase 3United States;France;Germany;Italy;Netherlands;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03196765
(ClinicalTrials.gov)
August 20185/5/2017Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With AdrenoleukodystrophyPhase 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 AdrenoleukodystrophyDrug: Sobetirome (NV1205)NeuroVia, Inc.NULLWithdrawn4 Years18 YearsMale0Phase 1;Phase 2Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom
12NCT03513328
(ClinicalTrials.gov)
June 15, 201819/4/2018Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchRecruiting3 Months39 YearsAll40Phase 1;Phase 2United States
13EUCTR2017-000748-16-PL
(EUCTR)
12/06/201802/05/2018Study to evaluate effect of MIN-102 compared to Placebo after 96 weeks of randomized, double-blind Treatment followed by open-label extension study to evaluate effect of long term treatment with MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patientsA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTINATIONAL, MULTICENTER STUDY WITH OPEN-LABEL TREATMENT EXTENSION TO ASSESS THE EFFECT OF MIN-102 ON THE PROGRESSION OF ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY - ADVANCE ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Minoryx Therapeutics S.L.NULLNot Recruiting Female: no
Male: yes
105Phase 2;Phase 3France;United States;Hungary;Spain;Poland;Netherlands;Germany;Italy;United Kingdom
14EUCTR2017-001684-21-GB
(EUCTR)
20/04/201826/10/2017Phase 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 LLCNULLNot Recruiting Female: no
Male: yes
25Phase 1France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom
15EUCTR2017-000748-16-DE
(EUCTR)
02/01/201822/06/2017Study to evaluate effect of MIN-102 compared to Placebo after 96 weeks of randomized, double-blind Treatment followed by open-label extension study to evaluate effect of long term treatment with MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patientsA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTINATIONAL, MULTICENTER STUDY WITH OPEN-LABEL TREATMENT EXTENSION TO ASSESS THE EFFECT OF MIN-102 ON THE PROGRESSION OF ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY - ADVANCE ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Product Code: MIN-102
INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Minoryx Therapeutics S.L.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
105Phase 2;Phase 3United States;France;Hungary;Poland;Spain;Netherlands;Germany;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT03231878
(ClinicalTrials.gov)
December 8, 201724/7/2017A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 (IMP) on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked AdrenoleukodystrophyAdrenoleukodystrophyDrug: MIN-102;Drug: PlacebosMinoryx Therapeutics, S.L.NULLActive, not recruiting18 Years65 YearsMale105Phase 2;Phase 3United States;France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom
17EUCTR2017-000748-16-NL
(EUCTR)
23/11/201710/07/2017Study to evaluate effect of MIN-102 compared to Placebo after 96 weeks of randomized, double-blind Treatment followed by open-label extension study to evaluate effect of long term treatment with MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patientsA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTINATIONAL, MULTICENTER STUDY WITH OPEN-LABEL TREATMENT EXTENSION TO ASSESS THE EFFECT OF MIN-102 ON THE PROGRESSION OF ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY - ADVANCE ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Minoryx Therapeutics S.L.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
105Phase 2;Phase 3France;United States;Hungary;Poland;Spain;Germany;Netherlands;Italy;United Kingdom
18EUCTR2017-000748-16-GB
(EUCTR)
14/09/201719/06/2017Study to evaluate effect of MIN-102 compared to Placebo after 96 weeks of randomized, double-blind Treatment followed by open-label extension study to evaluate effect of long term treatment with MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patientsA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTINATIONAL, MULTICENTER STUDY WITH OPEN-LABEL TREATMENT EXTENSION TO ASSESS THE EFFECT OF MIN-102 ON THE PROGRESSION OF ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY - ADVANCE ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Product Code: MIN-102
INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Minoryx Therapeutics S.L.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
105Phase 2;Phase 3France;United States;Hungary;Poland;Spain;Netherlands;Germany;Italy;United Kingdom
19EUCTR2017-000748-16-HU
(EUCTR)
04/09/201730/06/2017Study to evaluate effect of MIN-102 compared to Placebo after 96 weeks of randomized, double-blind Treatment followed by open-label extension study to evaluate effect of long term treatment with MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patientsA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTINATIONAL, MULTICENTER STUDY WITH OPEN-LABEL TREATMENT EXTENSION TO ASSESS THE EFFECT OF MIN-102 ON THE PROGRESSION OF ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY - ADVANCE ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Product Code: MIN-102
INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Minoryx Therapeutics S.L.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
105Phase 2;Phase 3France;United States;Hungary;Spain;Netherlands;Germany;Italy;United Kingdom
20EUCTR2017-000748-16-ES
(EUCTR)
25/08/201704/07/2017Study to evaluate effect of MIN-102 compared to Placebo after 96 weeks of randomized, double-blind Treatment followed by open-label extension study to evaluate effect of long term treatment with MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patientsA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTINATIONAL, MULTICENTER STUDY WITH OPEN-LABEL TREATMENT EXTENSION TO ASSESS THE EFFECT OF MIN-102 ON THE PROGRESSION OF ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY - ADVANCE ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Product Code: MIN-102
INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
Minoryx Therapeutics S.L.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
105Phase 2;Phase 3France;United States;Hungary;Spain;Netherlands;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT02595489
(ClinicalTrials.gov)
March 201629/10/2015A Pilot Study of Vitamin D in Boys With X-linked AdrenoleukodystrophyA Pilot Study of Vitamin D in Boys With X-linked AdrenoleukodystrophyX-linked AdrenoleukodystrophyDietary Supplement: vitamin D3Stanford UniversityNational Institute of Neurological Disorders and Stroke (NINDS);Hugo W. Moser Research Institute at Kennedy Krieger, Inc.;ALD Connect, Inc.Active, not recruiting18 Months25 YearsMale21Phase 1United States
22NCT02698579
(ClinicalTrials.gov)
January 201617/2/2016Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductLong-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductCerebral Adrenoleukodystrophy (CALD);Adrenoleukodystrophy (ALD);X-Linked Adrenoleukodystrophy (X-ALD)Drug: Lenti-D; elivaldogene autotemcelbluebird bioNULLEnrolling by invitationN/AN/AMale50United States;Argentina;Australia;Brazil;France;United Kingdom;Algeria
23NCT03864523
(ClinicalTrials.gov)
January 201616/9/2016Effect of Pioglitazone Administered to Patients With AdrenomyeloneuropathyEffect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy: a Phase II, Single-arm, Multicentric Clinical TrialAdrenomyeloneuropathy;X-linked AdrenoleukodystrophyDrug: PioglitazoneOnofre, Aurora Pujol, M.D.Instituto de Salud Carlos III;Fundacion Hesperia;ELA España AssociationCompleted18 Years65 YearsAll18Phase 2Spain
24EUCTR2011-001953-10-FR
(EUCTR)
08/07/201524/06/2015Clinical study to assess the efficacy and safety of gene therapy for the treatment of childhood cerebral adrenoleukodystrophyA phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of childhood cerebral adrenoleukodystrophy (CCALD) Childhood Cerebral Adrenoleukodystrophy (CCALD)
MedDRA version: 18.0;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 Productbluebird bio, Inc.NULLNot RecruitingFemale: no
Male: yes
15Phase 1;Phase 2;Phase 3United States;France;Argentina;Australia;Germany;United Kingdom
25NCT02410239
(ClinicalTrials.gov)
June 201527/3/2015MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)MT2014-14 Intrathecal Administration of Mesenchymal Stem Cells (IT-MSC) for the Treatment of Advanced Cerebral Adrenoleukodystrophy (cALD)Cerebral AdrenoleukodystrophyBiological: Mesenchymal Stem CellsMasonic Cancer Center, University of MinnesotaNULLWithdrawn4 YearsN/AAll0Phase 1NULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT02961803
(ClinicalTrials.gov)
October 20149/11/2016MD1003-AMN MD1003 in AdrenomyeloneuropathyMD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled StudyAdrenomyeloneuropathy;Adrenoleukodystrophy;AMNDrug: MD1003 100 mg capsule;Drug: PlaceboMedDay Pharmaceuticals SANULLCompleted18 Years60 YearsMale67Phase 2;Phase 3France;Germany;Spain
27NCT02254863
(ClinicalTrials.gov)
September 201423/9/2014UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsAugmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like CellsAdrenoleukodystrophy;Batten Disease;Mucopolysaccharidosis II;Leukodystrophy, Globoid Cell;Leukodystrophy, Metachromatic;Neimann Pick Disease;Pelizaeus-Merzbacher Disease;Sandhoff Disease;Tay-Sachs Disease;Brain Diseases, Metabolic, Inborn;Alpha-Mannosidosis;Sanfilippo MucopolysaccharidosesBiological: DUOC-01Joanne Kurtzberg, MDThe Marcus FoundationRecruitingN/A22 YearsAll12Phase 1United States
28EUCTR2014-000698-38-ES
(EUCTR)
13/08/201413/06/2014MD1003 IN ADRENOMYELONEUROPATHYMD1003 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 SASNULLNot RecruitingFemale: no
Male: yes
60Phase 2;Phase 3Spain
29NCT02171104
(ClinicalTrials.gov)
July 10, 201420/6/2014MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGMucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic DisordersBiological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D)Masonic Cancer Center, University of MinnesotaNULLRecruitingN/A55 YearsAll100Phase 2United States
30EUCTR2011-006113-34-ES
(EUCTR)
24/10/201312/09/2013Effect of pioglitazone in patienst with Adrenomyeloneuropathy.Effect of pioglitazone administred to patients with Adrenomyeloneuropathy: A phase II, Singlearm, Monocentric Trial. - Pioglitazone in Adrenomyeloneuropathy X-linked adrenoleukodystrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]INN or Proposed INN: PIOGLITAZONEAURORA PUJOL ONOFRENULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Spain
No.TrialIDDate_
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31NCT01586455
(ClinicalTrials.gov)
April 201325/4/2012Human Placental-Derived Stem Cell TransplantationA Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant DisordersMucopolysaccharidosis I;Mucopolysaccharidosis VI;Adrenoleukodystrophy;Niemann-Pick Disease;Metachromatic Leukodystrophy;Wolman Disease;Krabbe's Disease;Gaucher's Disease;Fucosidosis;Batten Disease;Severe Aplastic Anemia;Diamond-Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Myelodysplastic Syndrome;Acute Myelogenous Leukemia;Acute Lymphocytic LeukemiaDrug: Human Placental Derived Stem CellNew York Medical CollegeNULLActive, not recruitingN/A55 YearsAll43Phase 1United States
32NCT01787578
(ClinicalTrials.gov)
April 20136/2/2013Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)A Prospective Safety, Tolerance, Pharmacodynamics and Pharmacokinetics Study of Sobetirome in Male Subjects Diagnosed With X-linked Adrenoleukodystrophy (X-ALD)X-Linked Adrenoleukodystrophy;AdrenomyeloneuropathyDrug: SobetiromeThomas S. ScanlanNULLWithdrawn18 Years64 YearsMale0Phase 1United States
33NCT01495260
(ClinicalTrials.gov)
September 201128/11/2011A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of AntioxidantsA Clinical Trial for Adrenomyeloneuropathy (AMN): Validation of Biomarkers of Oxidative Stress, and Efficacy, Tolerance and Safety of a Mixture of the Antioxidants N-acetylcysteine, Lipoic Acid and Vitamin EAdrenomyeloneuropathyDrug: N-acetylcysteine;Drug: lipoic acid;Drug: vitamin EOnofre, Aurora Pujol, M.D.Ministerio de Sanidad, Servicios Sociales e Igualdad;Fundacion HesperiaCompleted18 Years64 YearsAll13Phase 2Spain
34NCT02233257
(ClinicalTrials.gov)
August 20115/5/2014Expanded Access for Lorenzo's Oil (GTO/GTE) in AdrenoleukodystrophyExpanded Access for Lorenzo's Oil (GTO/GTE) in AdrenoleukodystrophyX-linked AdrenoleukodystrophyDrug: Lorenzo's OilUniversity of MinnesotaNULLNo longer available18 Months18 YearsMaleUnited States
35NCT01372228
(ClinicalTrials.gov)
April 201110/6/2011Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersPhase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersHurler 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-mannosidosisBiological: hematopoietic stem cell infusionTalaris Therapeutics Inc.Duke UniversityActive, not recruitingN/AN/AAll30Phase 1;Phase 2United States
No.TrialIDDate_
enrollment
Date_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
36NCT01165060
(ClinicalTrials.gov)
July 201013/7/2010The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)Effect of Bezafibrate on Very Long Chain Fatty Acid Metabolism in Men With X-linked Adrenoleukodystrophy (X-ALD)X-linked Adrenoleukodystrophy;AdrenomyeloneuropathyDrug: BezafibrateAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)The Stop ALD FoundationCompleted18 YearsN/AMale10N/ANetherlands
37NCT01043640
(ClinicalTrials.gov)
December 20095/1/2010Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersAllogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic DisordersMucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal DisordersDrug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate MofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A21 YearsAll46Phase 2United States
38EUCTR2004-002200-14-GB
(EUCTR)
17/10/200618/07/2005Randomised Placebo Controlled Trial of a Parenteral Modified Cobratoxin in Adrenomyeloneuropathy - Modified Cobratoxin Therapy in AdrenomyeloneuropathyRandomised Placebo Controlled Trial of a Parenteral Modified Cobratoxin in Adrenomyeloneuropathy - Modified Cobratoxin Therapy in Adrenomyeloneuropathy AdrenomyeloneuropathyProduct Name: modified cobratoxin
Product Code: RPI78M for injection
ReceptoPharm Inc.NULLNot RecruitingFemale: yes
Male: yes
20Phase 3United Kingdom
39NCT00383448
(ClinicalTrials.gov)
September 200629/9/2006HSCT for High Risk Inherited Inborn ErrorsTreatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoietic Stem Cell TransplantationAdrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Tay Sachs Disease;Sandhoffs Disease;Wolman Disease;I-Cell Disease;Sanfilippo Syndrome;GM1 GangliosidosisDrug: Clofarabine;Procedure: Total body Irradiation;Drug: Melphalan;Biological: Hematopoietic Stem Cell Transplantation;Drug: Alemtuzumab;Drug: mycophenylate mofetil;Device: Cyclosporine A;Drug: HydroxyureaMasonic Cancer Center, University of MinnesotaNULLCompletedN/A70 YearsAll38Phase 2United States
40NCT00545597
(ClinicalTrials.gov)
March 200516/10/2007A Phase III Trial of Lorenzo's Oil in AdrenomyeloneuropathyA Phase III Trial of Lorenzo's Oil in AdrenomyeloneuropathyAdrenomyeloneuropathy;AdrenoleukodystrophyDrug: Lorenzo's oilEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Food and Drug Administration (FDA)Terminated18 YearsN/ABoth240Phase 3United States
No.TrialIDDate_
enrollment
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registration
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sponsor
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agemin
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PhaseCountries
41NCT00004450
(ClinicalTrials.gov)
August 199818/10/1999Randomized Study of Beta Interferon and Thalidomide in Patients With AdrenoleukodystrophyAdrenoleukodystrophyDrug: glyceryl trierucate;Drug: glyceryl trioleate;Drug: interferon beta;Drug: thalidomideFDA Office of Orphan Products DevelopmentHugo W. Moser Research Institute at Kennedy Krieger, Inc.Completed4 YearsN/AMale60N/ANULL
42NCT00004418
(ClinicalTrials.gov)
April 199818/10/1999Effect of Glycerol Trierucate on Clinical Course of AdrenoleukodystrophyStudy of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With AdrenoleukodystrophyAdrenoleukodystrophyDrug: glyceryl trierucate/glyceryl trioleateHugo W. Moser Research Institute at Kennedy Krieger, Inc.NULLTerminated18 Months8 YearsMale126Phase 2United States
43NCT00004442
(ClinicalTrials.gov)
September 199718/10/1999Study of Bile Acids in Patients With Peroxisomal DisordersInfantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;AdrenoleukodystrophyDrug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiolChildren's Hospital Research Foundation University of CincinnatiChildren's Hospital Medical Center, CincinnatiTerminatedN/A5 YearsBoth25N/ANULL
44NCT00176904
(ClinicalTrials.gov)
January 199512/9/2005Stem Cell Transplant for Inborn Errors of MetabolismTreatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow TransplantationAdrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Gaucher's Disease;Fucosidosis;Wolman Disease;Niemann-Pick Disease;Batten Disease;GM1 Gangliosidosis;Tay Sachs Disease;Sandhoff DiseaseProcedure: Stem Cell Transplant;Drug: Busulfan, Cyclophosphamide, Antithymocyte GlobulinMasonic Cancer Center, University of MinnesotaNULLCompletedN/AN/AAll135Phase 2;Phase 3United States
45NCT00007020
(ClinicalTrials.gov)
January 19926/12/2000Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidInvestigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid MetabolismInfantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;CholestasisDrug: Cholic AcidsTravere Therapeutics, Inc.Children's Hospital Medical Center, CincinnatiCompletedN/AN/AAll85Phase 3United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
46EUCTR2011-001953-10-DE
(EUCTR)
11/12/2017Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophyA phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of 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
Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA
bluebird bio, Inc.NULLNAFemale: no
Male: yes
32Phase 2;Phase 3United States;France;Germany;United Kingdom
47EUCTR2017-001684-21-FR
(EUCTR)
23/03/2018Phase 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.NULLNA Female: no
Male: yes
25Phase 1France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom
48EUCTR2015-002805-13-FR
(EUCTR)
24/07/2017Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductLongterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product 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: elivaldogenum tavalentivecum
Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA
bluebird bio, Inc.NULLNAFemale: no
Male: yes
25Phase 3United States;France;Argentina;Australia;Algeria;United Kingdom
49EUCTR2019-000654-59-DE
(EUCTR)
19/07/2019A study to evaluate the effects of a new drug called MIN-102 on diseaseprogression in boys with cerebral X-linked adrenoleukodystrophy (cALD)An open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophy (cALD) to assess the effect of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT) Cerebral X-linked 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: 5-[4-[2-(5-(1-hydroxyethyl)-2- pyridinyl)ethoxy]benzyl]-2,4- thiazolidinedione hydrochloride
INN or Proposed INN: Leriglitazone
Other descriptive name: 5-[[4-[2-[5-(1-HYDROXYETHYL)-2-PYRIDINYL]ETHOXY]PHENYL]METHYL]-2,4-THIAZOLIDINEDIONE HYDROCHLORIDE
Minoryx Therapeutics S.L.NULLNAFemale: no
Male: yes
13Phase 2United States;France;Spain;Germany