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：102］
Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2018-001145-14-FR | 27/03/2019 | 30 April 2019 | Clinical 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 | 20 | Phase 3 | United States;France;Netherlands;Germany;Italy;United Kingdom | |||
| 2 | NCT03852498 | January 24, 2019 | 28 October 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) | Drug: Genetic | bluebird bio | Recruiting | N/A | 17 Years | Male | 20 | Phase 3 | United States;France;Italy;United Kingdom | |
| 3 | NCT03196765 | August 2018 | 18 March 2019 | 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. | Not recruiting | 4 Years | 18 Years | Male | 0 | Phase 1/Phase 2 | Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom | |
| 4 | NCT03513328 | June 15, 2018 | 3 June 2019 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders | Bone 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;Adrenoleukodystrophy | Drug: Thiotepa--single daily dose;Drug: Thiotepa--escalated dose | University of Florida | Live Like Bella Pediatric Cancer Research | Recruiting | 6 Months | 38 Years | All | 40 | Phase 1/Phase 2 | United States |
| 5 | NCT03231878 | December 8, 2017 | 8 April 2019 | A 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 Adrenoleukodystrophy | Adrenoleukodystrophy | Drug: MIN-102;Drug: Placebos | Minoryx Therapeutics, S.L. | Not recruiting | 18 Years | 65 Years | Male | 105 | Phase 2/Phase 3 | United States;France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2017-000748-16-NL | 23/11/2017 | 15 October 2018 | Study 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 patients | A 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 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. | Authorised | Female: no Male: yes | 105 | Phase 2;Phase 3 | France;United States;Hungary;Poland;Spain;Germany;Netherlands;Italy;United Kingdom | |||
| 7 | EUCTR2017-000748-16-HU | 04/09/2017 | 2 October 2017 | Study 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 patients | A 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 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. | Authorised | Female: no Male: yes | 105 | Phase 2;Phase 3 | France;United States;Hungary;Spain;Netherlands;Germany;Italy;United Kingdom | |||
| 8 | NCT02595489 | March 2016 | 17 June 2019 | A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy | A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy | X-linked Adrenoleukodystrophy | Dietary Supplement: vitamin D3 | Stanford University | National Institute of Neurological Disorders and Stroke (NINDS);Hugo W. Moser Research Institute at Kennedy Krieger, Inc.;ALD Connect, Inc. | Recruiting | 18 Months | 25 Years | Male | 20 | Phase 1 | United States |
| 9 | NCT03864523 | January 2016 | 30 September 2019 | Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy | Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy: a Phase II, Single-arm, Multicentric Clinical Trial | Adrenomyeloneuropathy;X-linked Adrenoleukodystrophy | Drug: Pioglitazone | Onofre, Aurora Pujol, M.D. | Instituto de Salud Carlos III;Fundacion Hesperia;ELA España Association | Not recruiting | 18 Years | 65 Years | All | 18 | Phase 2 | Spain |
| 10 | EUCTR2015-002805-13-GB | 27/11/2015 | 15 October 2018 | Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Longterm 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 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, Inc | Authorised | Female: no Male: yes | 50 | Phase 3 | France;United States;Argentina;Australia;Algeria;United Kingdom | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT02410239 | June 2015 | 16 December 2017 | MT2014-14 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 Adrenoleukodystrophy | Biological: Mesenchymal Stem Cells | Masonic Cancer Center, University of Minnesota | Not recruiting | 4 Years | N/A | All | 0 | Phase 1 | ||
| 12 | NCT02233257 | February 2015 | 5 February 2018 | Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy | Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy | X-linked Adrenoleukodystrophy | Drug: Lorenzo's Oil | University of Minnesota - Clinical and Translational Science Institute | Not recruiting | 18 Months | 18 Years | Male | N/A | United States | ||
| 13 | NCT02961803 | October 2014 | 16 December 2017 | 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 | Not recruiting | 18 Years | 60 Years | Male | 67 | Phase 2/Phase 3 | France;Germany;Spain | |
| 14 | NCT02254863 | September 2014 | 17 June 2019 | UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells | Adrenoleukodystrophy;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 Mucopolysaccharidoses | Biological: DUOC-01 | Joanne Kurtzberg, MD | The Marcus Foundation | Recruiting | N/A | 22 Years | All | 12 | Phase 1 | United States |
| 15 | EUCTR2014-000698-38-ES | 13/08/2014 | 2 October 2017 | 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 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 SAS | Not Recruiting | Female: no Male: yes | 60 | Phase 2;Phase 3 | Spain | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT02171104 | July 10, 2014 | 7 October 2019 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis 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 Disorders | Biological: 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 Minnesota | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States | |
| 17 | EUCTR2011-006113-34-ES | 24/10/2013 | 26 November 2013 | Effect 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] | 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 ONOFRE | Authorised | Female: yes Male: yes | Phase 2 | Spain | ||||
| 18 | NCT01586455 | April 2013 | 8 January 2018 | Human Placental-Derived Stem Cell Transplantation | A 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 Disorders | Mucopolysaccharidosis 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 Leukemia | Drug: Human Placental Derived Stem Cell | New York Medical College | Not recruiting | N/A | 55 Years | All | 43 | Phase 1 | United States | |
| 19 | NCT01787578 | April 2013 | 19 February 2015 | Safety 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;Adrenomyeloneuropathy | Drug: Sobetirome | Thomas S. Scanlan | Not recruiting | 18 Years | 64 Years | Male | 0 | Phase 1 | United States | |
| 20 | NCT01495260 | September 2011 | 18 March 2019 | A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants | A 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 E | Adrenomyeloneuropathy | Drug: N-acetylcysteine;Drug: lipoic acid;Drug: vitamin E | Onofre, Aurora Pujol, M.D. | Ministerio de Sanidad, Servicios Sociales e Igualdad;Fundacion Hesperia | Not recruiting | 18 Years | 64 Years | All | 13 | Phase 2 | Spain |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT01372228 | April 2011 | 30 September 2019 | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | Hurler 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-mannosidosis | Biological: hematopoietic stem cell infusion | University of Louisville | Duke University | Not recruiting | N/A | N/A | All | 30 | Phase 1/Phase 2 | United States |
| 22 | NCT01165060 | July 2010 | 19 February 2015 | The 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;Adrenomyeloneuropathy | Drug: Bezafibrate | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | The Stop ALD Foundation | Not recruiting | 18 Years | N/A | Male | 10 | N/A | Netherlands |
| 23 | NCT01043640 | December 2009 | 16 December 2017 | Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders | Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders | Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal Disorders | Drug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate Mofetil | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | 21 Years | All | 46 | Phase 2 | United States | |
| 24 | EUCTR2004-002200-14-GB | 17/10/2006 | 1 May 2012 | Randomised Placebo Controlled Trial of a Parenteral Modified Cobratoxin in Adrenomyeloneuropathy - Modified Cobratoxin Therapy in Adrenomyeloneuropathy | Randomised Placebo Controlled Trial of a Parenteral Modified Cobratoxin in Adrenomyeloneuropathy - Modified Cobratoxin Therapy in Adrenomyeloneuropathy | Adrenomyeloneuropathy | Product 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. | Authorised | Female: yes Male: yes | United Kingdom | |||||
| 25 | NCT00383448 | September 2006 | 22 July 2019 | HSCT for High Risk Inherited Inborn Errors | Treatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoietic Stem Cell Transplantation | Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Tay Sachs Disease;Sandhoffs Disease;Wolman Disease;I-Cell Disease;Sanfilippo Syndrome;GM1 Gangliosidosis | Drug: Clofarabine;Procedure: Total body Irradiation;Drug: Melphalan;Biological: Hematopoietic Stem Cell Transplantation;Drug: Alemtuzumab;Drug: mycophenylate mofetil;Device: Cyclosporine A;Drug: Hydroxyurea | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | 70 Years | All | 38 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT00545597 | March 2005 | 19 February 2015 | A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy | A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy | Adrenomyeloneuropathy;Adrenoleukodystrophy | Drug: Lorenzo's oil | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Food and Drug Administration (FDA) | Not recruiting | 18 Years | N/A | Both | 240 | Phase 3 | United States |
| 27 | NCT00004450 | August 1998 | 7 April 2015 | Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy | Adrenoleukodystrophy | Drug: glyceryl trierucate;Drug: glyceryl trioleate;Drug: interferon beta;Drug: thalidomide | FDA Office of Orphan Products Development | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Not recruiting | 4 Years | N/A | Male | 60 | N/A | ||
| 28 | NCT00004418 | April 1998 | 19 February 2015 | Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy | Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy | Adrenoleukodystrophy | Drug: glyceryl trierucate;Drug: glyceryl trioleate | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Not recruiting | 18 Months | 6 Years | Male | 30 | Phase 2/Phase 3 | United States | |
| 29 | NCT00004442 | September 1997 | 7 April 2015 | Study of Bile Acids in Patients With Peroxisomal Disorders | Infantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;Adrenoleukodystrophy | Drug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiol | Children's Hospital Research Foundation University of Cincinnati | Children's Hospital Medical Center, Cincinnati | Not recruiting | N/A | 5 Years | Both | 25 | N/A | ||
| 30 | NCT00176904 | January 1995 | 19 October 2017 | Stem Cell Transplant for Inborn Errors of Metabolism | Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation | Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Gaucher's Disease;Fucosidosis;Wolman Disease;Niemann-Pick Disease;Batten Disease;GM1 Gangliosidosis;Tay Sachs Disease;Sandhoff Disease | Procedure: Stem Cell Transplant;Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | N/A | All | 135 | Phase 2/Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT00007020 | January 1992 | 19 February 2015 | Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid | Investigation 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-M01RR08084-0009 | Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;Cholestasis | Drug: Cholic Acids | Asklepion Pharmaceuticals, LLC | Children's Hospital Medical Center, Cincinnati | Not recruiting | N/A | N/A | Both | 79 | Phase 3 | United States |