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 20. 副腎白質ジストロフィー [臨床試験数:31,薬物数:60(DrugBank:27),標的遺伝子数:17,標的パスウェイ数:23] 

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
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PhaseCountries
1EUCTR2018-001145-14-FR27/03/201930 April 2019Clinical 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]
Product Name: Lenti-D Drug Product
Pharmaceutical Form: Dispersion for infusion
Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA
Concentration unit: Other
Concentration type: not less then
Concentration number: 5000000-
bluebird bio, Inc.Authorised Female: no
Male: yes
20Phase 3United States;France;Netherlands;Germany;Italy;United Kingdom
2NCT03852498January 24, 201928 October 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: Geneticbluebird bioRecruitingN/A17 YearsMale20Phase 3United States;France;Italy;United Kingdom
3NCT03196765August 201818 March 2019Safety, 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.Not recruiting4 Years18 YearsMale0Phase 1/Phase 2Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom
4NCT03513328June 15, 20183 June 2019Conditioning 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 ResearchRecruiting6 Months38 YearsAll40Phase 1/Phase 2United States
5NCT03231878December 8, 20178 April 2019A 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.Not recruiting18 Years65 YearsMale105Phase 2/Phase 3United States;France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
Inclusion_
agemin
Inclusion_
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Target_
size
PhaseCountries
6EUCTR2017-000748-16-NL23/11/201715 October 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 - ADVANCEADRENOMYELONEUROPATHY 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
CAS Number: 146062-44-4
Current Sponsor code: MIN-102
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 15-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Minoryx Therapeutics S.L.AuthorisedFemale: no
Male: yes
105Phase 2;Phase 3France;United States;Hungary;Poland;Spain;Germany;Netherlands;Italy;United Kingdom
7EUCTR2017-000748-16-HU04/09/20172 October 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 - ADVANCEADRENOMYELONEUROPATHY 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
CAS Number: 146062-44-4
Current Sponsor code: MIN-102
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 15-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Minoryx Therapeutics S.L.AuthorisedFemale: no
Male: yes
105Phase 2;Phase 3France;United States;Hungary;Spain;Netherlands;Germany;Italy;United Kingdom
8NCT02595489March 201617 June 2019A 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.Recruiting18 Months25 YearsMale20Phase 1United States
9NCT03864523January 201630 September 2019Effect 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 AssociationNot recruiting18 Years65 YearsAll18Phase 2Spain
10EUCTR2015-002805-13-GB27/11/201515 October 2018Longterm 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 ProductCerebral 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
Pharmaceutical Form: Dispersion for infusion
INN or Proposed INN: N/A
Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA
Concentration unit: Other
Concentration type: not less then
Concentration number: 5000000-
bluebird bio, IncAuthorisedFemale: no
Male: yes
50Phase 3France;United States;Argentina;Australia;Algeria;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
Inclusion_
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Target_
size
PhaseCountries
11NCT02410239June 201516 December 2017MT2014-14 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 MinnesotaNot recruiting4 YearsN/AAll0Phase 1
12NCT02233257February 20155 February 2018Expanded Access for Lorenzo's Oil (GTO/GTE) in AdrenoleukodystrophyExpanded Access for Lorenzo's Oil (GTO/GTE) in AdrenoleukodystrophyX-linked AdrenoleukodystrophyDrug: Lorenzo's OilUniversity of Minnesota - Clinical and Translational Science InstituteNot recruiting18 Months18 YearsMaleN/AUnited States
13NCT02961803October 201416 December 2017MD1003-AMN MD1003 in AdrenomyeloneuropathyMD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled StudyAdrenomyeloneuropathy;Adrenoleukodystrophy;AMNDrug: MD1003 100 mg capsule;Drug: PlaceboMedDay Pharmaceuticals SANot recruiting18 Years60 YearsMale67Phase 2/Phase 3France;Germany;Spain
14NCT02254863September 201417 June 2019UCB 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
15EUCTR2014-000698-38-ES13/08/20142 October 2017MD1003 IN ADRENOMYELONEUROPATHYMD1003 IN ADRENOMYELONEUROPATHY: A RANDOMIZED DOUBLE BLIND PLACEBO CONTROLLED STUDY - MD1003-AMNAdrenomyeloneuropathy
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
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: D-BIOTIN
Current Sponsor code: MD1003
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
MEDDAY SASNot RecruitingFemale: no
Male: yes
60Phase 2;Phase 3Spain
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
16NCT02171104July 10, 20147 October 2019MT2013-31: 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 MinnesotaRecruitingN/A55 YearsAll100Phase 2United States
17EUCTR2011-006113-34-ES24/10/201326 November 2013Effect of pioglitazone in patienst with Adrenomyeloneuropathy.Effect of pioglitazone administred to patients with Adrenomyeloneuropathy: A phase II, Singlearm, Monocentric Trial. - Pioglitazone in AdrenomyeloneuropathyX-linked adrenoleukodystrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Pharmaceutical Form: Tablet
INN or Proposed INN: PIOGLITAZONE
CAS Number: 111025-46-8
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 30-
AURORA PUJOL ONOFREAuthorisedFemale: yes
Male: yes
Phase 2Spain
18NCT01586455April 20138 January 2018Human 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 CollegeNot recruitingN/A55 YearsAll43Phase 1United States
19NCT01787578April 201319 February 2015Safety 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. ScanlanNot recruiting18 Years64 YearsMale0Phase 1United States
20NCT01495260September 201118 March 2019A 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 HesperiaNot recruiting18 Years64 YearsAll13Phase 2Spain
No.TrialIDDate_
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21NCT01372228April 201130 September 2019Phase 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 infusionUniversity of LouisvilleDuke UniversityNot recruitingN/AN/AAll30Phase 1/Phase 2United States
22NCT01165060July 201019 February 2015The 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 FoundationNot recruiting18 YearsN/AMale10N/ANetherlands
23NCT01043640December 200916 December 2017Allogeneic 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 MinnesotaNot recruitingN/A21 YearsAll46Phase 2United States
24EUCTR2004-002200-14-GB17/10/20061 May 2012Randomised 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 AdrenomyeloneuropathyAdrenomyeloneuropathyProduct Name: modified cobratoxin
Product Code: RPI78M for injection
Pharmaceutical Form: Solution for injection
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
ReceptoPharm Inc.AuthorisedFemale: yes
Male: yes
United Kingdom
25NCT00383448September 200622 July 2019HSCT 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 MinnesotaNot recruitingN/A70 YearsAll38Phase 2United States
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
26NCT00545597March 200519 February 2015A 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)Not recruiting18 YearsN/ABoth240Phase 3United States
27NCT00004450August 19987 April 2015Randomized 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.Not recruiting4 YearsN/AMale60N/A
28NCT00004418April 199819 February 2015Effect 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;Drug: glyceryl trioleateHugo W. Moser Research Institute at Kennedy Krieger, Inc.Not recruiting18 Months6 YearsMale30Phase 2/Phase 3United States
29NCT00004442September 19977 April 2015Study 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, CincinnatiNot recruitingN/A5 YearsBoth25N/A
30NCT00176904January 199519 October 2017Stem 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 MinnesotaNot recruitingN/AN/AAll135Phase 2/Phase 3United States
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31NCT00007020January 199219 February 2015Compassionate 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 Metabolism. This Study Was Previously Registered by the NCRR and Identified as NCRR-M01RR08084-0009Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;CholestasisDrug: Cholic AcidsAsklepion Pharmaceuticals, LLCChildren's Hospital Medical Center, CincinnatiNot recruitingN/AN/ABoth79Phase 3United States

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