20. 副腎白質ジストロフィー Adrenoleukodystrophy Clinical trials / Disease details
臨床試験数 : 61 / 薬物数 : 90 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05200104 (ClinicalTrials.gov) | December 2022 | 6/1/2022 | Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) | An Open-label Phase 2a Study to Assess the Pharmacokinetics and Pharmacodynamic of PXL065 Parameters After 12 Weeks of Treatment in Male Subjects With Adrenomyeloneuropathy (AMN) | ALD (Adrenoleukodystrophy) | Drug: PXL065 | Poxel SA | NULL | Not yet recruiting | 18 Years | 65 Years | Male | 12 | Phase 2 | NULL |
| 2 | NCT05146284 (ClinicalTrials.gov) | December 2022 | 16/11/2021 | Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) | A Randomized Open-label Phase 2a Study to Assess PXL770 After 12 Weeks of Treatment in Male Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) | Adrenomyeloneuropathy | Drug: PXL770 | Poxel SA | NULL | Not yet recruiting | 18 Years | 65 Years | Male | 24 | Phase 2 | NULL |
| 3 | NCT05394064 (ClinicalTrials.gov) | June 30, 2022 | 16/5/2022 | A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) | A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients With Adrenomyeloneuropathy | AMN;AMN Gene Mutation;X-ALD | Genetic: SBT101;Procedure: Imitation Procedure | SwanBio Therapeutics, Inc. | NULL | Recruiting | 18 Years | 65 Years | Male | 16 | Phase 1/Phase 2 | United States |
| 4 | EUCTR2021-006223-18-DE (EUCTR) | 03/05/2022 | 17/01/2022 | Trial on patients with adrenomyeloneuropathy to assess PXL770's behaviour, a new potential medicine, in the body | A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN) - START770 | Adrenomyeloneuropathy (AMN);Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: PXL770 Product Code: PXL770 Other descriptive name: PXL770 | Poxel S.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 24 | Phase 2 | Germany | ||
| 5 | EUCTR2015-002805-13-NL (EUCTR) | 07/09/2021 | 30/04/2021 | 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lenti-D Drug Product INN or Proposed INN: Elivaldogene autotemcel Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA | bluebird bio, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 60 | Phase 3 | France;United States;Argentina;Brazil;Australia;Netherlands;United Kingdom | ||
| 6 | NCT05003648 (ClinicalTrials.gov) | September 2021 | 15/7/2021 | Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy | Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance | Adrenoleukodystrophy;Restless Legs Syndrome | Drug: Pramipexole;Drug: Placebo | Massachusetts General Hospital | European Leukodystrophy Association | Not yet recruiting | 18 Years | 75 Years | Female | 24 | Phase 4 | United States;Netherlands |
| 7 | NCT04973657 (ClinicalTrials.gov) | June 22, 2021 | 28/6/2021 | A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN | Phase 1b, Multi-center, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability, PD and PK of VK0214, in Subjects With the Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) | Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) | Drug: VK0214;Other: Placebo | Viking Therapeutics, Inc. | NULL | Recruiting | 18 Years | N/A | All | 36 | Phase 1 | United States |
| 8 | EUCTR2019-000654-59-FR (EUCTR) | 31/03/2020 | 21/07/2020 | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 13 | Phase 2 | United States;France;Spain;Germany | ||
| 9 | NCT04303416 (ClinicalTrials.gov) | March 9, 2020 | 6/3/2020 | Plasma Exchange With Albumin in AMN Patients | Effect of Plasma Exchange With Albumin in Patients With Adrenomyeloneuropathy: Unicentric, Single Arm, Proof of Concept Study. | Adrenomyeloneuropathy;Adrenoleukodystrophy | Drug: Albumin solution | Onofre, Aurora Pujol, M.D. | NULL | Completed | 18 Years | 65 Years | Male | 5 | Phase 2/Phase 3 | Spain |
| 10 | EUCTR2019-004733-17-ES (EUCTR) | 25/02/2020 | 20/12/2019 | Plasma exchange by albumin replacement in Adrenomyeloneuropathy | Effect 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] | Trade Name: Albunorm® 5%, Product Name: Albumin INN or Proposed INN: ALBUMIN Other descriptive name: ALBUMIN | Aurora Pujol Onofre | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Phase 2 | Spain | ||
| 11 | 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 | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | France;United States;Netherlands;Germany;United Kingdom;Italy | ||
| 12 | NCT04528706 (ClinicalTrials.gov) | September 13, 2019 | 31/7/2020 | A 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 Adrenoleukodystrophy | Drug: MIN-102 | Minoryx Therapeutics, S.L. | NULL | Recruiting | 2 Years | 12 Years | Male | 13 | Phase 2 | Argentina;France;Germany;Spain |
| 13 | EUCTR2019-000654-59-DE (EUCTR) | 13/08/2019 | 19/07/2019 | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 13 | Phase 2 | France;United States;Argentina;Spain;Germany | ||
| 14 | EUCTR2019-000654-59-ES (EUCTR) | 25/06/2019 | 30/04/2019 | A 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] | 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 10 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Spain;Germany | ||
| 15 | 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 | ||
| 16 | EUCTR2018-001145-14-IT (EUCTR) | 19/04/2019 | 02/08/2021 | 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 inferior or equal to 17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) - ALD-104 | 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;Netherlands;Germany;United Kingdom;Italy | ||
| 17 | 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 | ||
| 18 | EUCTR2018-001145-14-FR (EUCTR) | 27/03/2019 | 23/10/2018 | 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 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 | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;France;Netherlands;Germany;Italy;United Kingdom | ||
| 19 | NCT03852498 (ClinicalTrials.gov) | January 24, 2019 | 13/2/2019 | A 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) | Genetic: Lenti-D | bluebird bio | NULL | Active, not recruiting | N/A | 17 Years | Male | 35 | Phase 3 | United States;France;Germany;Italy;Netherlands;United Kingdom |
| 20 | NCT03727555 (ClinicalTrials.gov) | October 30, 2018 | 25/9/2018 | Lentiviral Gene Therapy for X-ALD | Lentiviral Gene Therapy for X-linked Adrenoleukodystrophy (X-ALD) | X-linked Adrenoleukodystrophy | Genetic: Intracerebral LV gene therapy | Shenzhen Geno-Immune Medical Institute | NULL | Recruiting | N/A | N/A | All | 10 | N/A | China |
| 21 | NCT03196765 (ClinicalTrials.gov) | August 2018 | 5/5/2017 | Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy | Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Childhood Cerebral Adrenoleukodystrophy (CCALD) | X-Linked Adrenoleukodystrophy | Drug: Sobetirome (NV1205) | NeuroVia, Inc. | NULL | Withdrawn | 4 Years | 18 Years | Male | 0 | Phase 1/Phase 2 | Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom |
| 22 | EUCTR2011-001953-10-DE (EUCTR) | 13/07/2018 | 11/12/2017 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy | A 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. | NULL | Not Recruiting | Female: no Male: yes | 32 | Phase 2;Phase 3 | United States;France;Germany;United Kingdom | ||
| 23 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | 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 | Active, not recruiting | 3 Months | 39 Years | All | 6 | Phase 1/Phase 2 | United States |
| 24 | EUCTR2017-000748-16-PL (EUCTR) | 12/06/2018 | 02/05/2018 | 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 INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride | Minoryx Therapeutics S.L. | NULL | Not Recruiting | Female: no Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Hungary;Spain;Poland;Netherlands;Germany;Italy;United Kingdom | ||
| 25 | EUCTR2017-001684-21-GB (EUCTR) | 20/04/2018 | 26/10/2017 | Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD) | Phase I/II, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD) | Childhood Cerebral Adrenoleukodystrophy (CCALD);Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Product Name: Sobetirome Product Code: NV1205 INN or Proposed INN: Sobetirome Other descriptive name: QTXEW2-04-AF, EW2, GC-1, QRX-431 | Neurov Acquisition LLC | NULL | Not Recruiting | Female: no Male: yes | 25 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom | ||
| 26 | EUCTR2017-000748-16-DE (EUCTR) | 02/01/2018 | 22/06/2017 | 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 INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride | Minoryx Therapeutics S.L. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 105 | Phase 2;Phase 3 | France;United States;Hungary;Poland;Spain;Netherlands;Germany;United Kingdom;Italy | ||
| 27 | NCT03231878 (ClinicalTrials.gov) | December 8, 2017 | 24/7/2017 | 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. | NULL | Active, not recruiting | 18 Years | 65 Years | Male | 105 | Phase 2/Phase 3 | United States;France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom |
| 28 | EUCTR2017-000748-16-NL (EUCTR) | 23/11/2017 | 10/07/2017 | 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 INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride | Minoryx Therapeutics S.L. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Hungary;Poland;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 29 | EUCTR2017-000748-16-GB (EUCTR) | 14/09/2017 | 19/06/2017 | 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 INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride | Minoryx Therapeutics S.L. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 105 | Phase 2;Phase 3 | France;United States;Hungary;Poland;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 30 | EUCTR2017-000748-16-HU (EUCTR) | 04/09/2017 | 30/06/2017 | 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 INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride | Minoryx Therapeutics S.L. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 105 | Phase 2;Phase 3 | France;United States;Hungary;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 31 | EUCTR2017-000748-16-ES (EUCTR) | 25/08/2017 | 04/07/2017 | 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 INN or Proposed INN: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride | Minoryx Therapeutics S.L. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 105 | Phase 2;Phase 3 | France;United States;Hungary;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 32 | NCT02595489 (ClinicalTrials.gov) | November 21, 2016 | 29/10/2015 | 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. | Completed | 18 Months | 25 Years | Male | 21 | Phase 1 | United States |
| 33 | NCT02698579 (ClinicalTrials.gov) | January 22, 2016 | 17/2/2016 | Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Cerebral Adrenoleukodystrophy (CALD);Adrenoleukodystrophy (ALD);X-Linked Adrenoleukodystrophy (X-ALD) | Genetic: Lenti-D | bluebird bio | NULL | Enrolling by invitation | N/A | N/A | Male | 60 | United States;Argentina;Australia;Brazil;France;Germany;Netherlands;United Kingdom;Algeria | |
| 34 | NCT03864523 (ClinicalTrials.gov) | January 2016 | 16/9/2016 | 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 | Completed | 18 Years | 65 Years | All | 18 | Phase 2 | Spain |
| 35 | EUCTR2011-001953-10-FR (EUCTR) | 08/07/2015 | 24/06/2015 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of childhood cerebral adrenoleukodystrophy | A 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 Product | bluebird bio, Inc. | NULL | Not Recruiting | Female: no Male: yes | 15 | Phase 1;Phase 2;Phase 3 | United States;France;Argentina;Australia;Germany;United Kingdom | ||
| 36 | NCT02410239 (ClinicalTrials.gov) | June 2015 | 27/3/2015 | 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 | NULL | Withdrawn | 4 Years | N/A | All | 0 | Phase 1 | NULL |
| 37 | NCT02204904 (ClinicalTrials.gov) | April 2015 | 28/7/2014 | Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD) | A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in Males =17 Years of Age Undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy | X-Linked Adrenoleukodystrophy (X-ALD);Cerebral Adrenoleukodystrophy (CALD);Adrenoleukodystrophy (ALD) | Genetic: Allo-HSCT | bluebird bio | NULL | Terminated | N/A | 17 Years | Male | 59 | United States;Argentina;Canada;Germany;Italy;Netherlands;United Kingdom | |
| 38 | NCT02559830 (ClinicalTrials.gov) | January 2015 | 12/8/2015 | Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy | A Phase I/II Clinical Trial of Lentiviral Hematopoietic Stem Cell Gene Therapy for Treatment of Developed Metachromatic Leukodystrophy and Adrenoleukodystrophy | Metachromatic Leukodystrophy;Adrenoleukodystrophy | Genetic: transduced CD34+ hematopoietic stem cell | Shenzhen Second People's Hospital | Shenzhen University;Guangzhou Women and Children's Medical Center | Recruiting | 1 Year | 16 Years | All | 50 | Phase 1/Phase 2 | China |
| 39 | NCT02961803 (ClinicalTrials.gov) | October 2014 | 9/11/2016 | 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 | NULL | Completed | 18 Years | 60 Years | Male | 67 | Phase 2/Phase 3 | France;Germany;Spain |
| 40 | NCT02254863 (ClinicalTrials.gov) | September 2014 | 23/9/2014 | 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 | 1 Week | 22 Years | All | 40 | Phase 1 | United States |
| 41 | EUCTR2014-000698-38-ES (EUCTR) | 13/08/2014 | 13/06/2014 | 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 INN or Proposed INN: D-BIOTIN | MEDDAY SAS | NULL | Not Recruiting | Female: no Male: yes | 60 | Phase 2;Phase 3 | Spain | ||
| 42 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | 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 | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 43 | EUCTR2011-006113-34-ES (EUCTR) | 24/10/2013 | 12/09/2013 | 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] | INN or Proposed INN: PIOGLITAZONE | AURORA PUJOL ONOFRE | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | Spain | |||
| 44 | NCT01896102 (ClinicalTrials.gov) | August 21, 2013 | 22/3/2013 | A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) | A 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) | Genetic: Lenti-D Drug Product | bluebird bio | NULL | Completed | N/A | 17 Years | Male | 32 | Phase 2/Phase 3 | United States;Argentina;Australia;France;Germany;United Kingdom |
| 45 | NCT01787578 (ClinicalTrials.gov) | April 2013 | 6/2/2013 | 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 | NULL | Withdrawn | 18 Years | 64 Years | Male | 0 | Phase 1 | United States |
| 46 | NCT01586455 (ClinicalTrials.gov) | April 2013 | 25/4/2012 | 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 | NULL | Completed | 0 Years | 55 Years | All | 43 | Phase 1 | United States |
| 47 | NCT01495260 (ClinicalTrials.gov) | September 2011 | 28/11/2011 | 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 | Completed | 18 Years | 64 Years | All | 13 | Phase 2 | Spain |
| 48 | NCT02233257 (ClinicalTrials.gov) | August 2011 | 5/5/2014 | 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 | NULL | No longer available | 18 Months | 18 Years | Male | United States | ||
| 49 | NCT01372228 (ClinicalTrials.gov) | April 2011 | 10/6/2011 | 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 | Talaris Therapeutics Inc. | Duke University | Terminated | N/A | N/A | All | 30 | Phase 1/Phase 2 | United States |
| 50 | NCT01165060 (ClinicalTrials.gov) | July 2010 | 13/7/2010 | 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 | Completed | 18 Years | N/A | Male | 10 | N/A | Netherlands |
| 51 | NCT01043640 (ClinicalTrials.gov) | December 2009 | 5/1/2010 | 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 | NULL | Completed | N/A | 21 Years | All | 46 | Phase 2 | United States |
| 52 | EUCTR2004-002200-14-GB (EUCTR) | 17/10/2006 | 18/07/2005 | 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 | ReceptoPharm Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | United Kingdom | ||
| 53 | NCT00383448 (ClinicalTrials.gov) | September 2006 | 29/9/2006 | 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 | NULL | Completed | N/A | 70 Years | All | 38 | Phase 2 | United States |
| 54 | NCT00545597 (ClinicalTrials.gov) | March 2005 | 16/10/2007 | 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) | Terminated | 18 Years | N/A | Both | 240 | Phase 3 | United States |
| 55 | NCT00004450 (ClinicalTrials.gov) | August 1998 | 18/10/1999 | 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. | Completed | 4 Years | N/A | Male | 60 | N/A | NULL | |
| 56 | NCT00004418 (ClinicalTrials.gov) | April 1998 | 18/10/1999 | 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/glyceryl trioleate | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | NULL | Terminated | 18 Months | 8 Years | Male | 126 | Phase 2 | United States |
| 57 | NCT00004442 (ClinicalTrials.gov) | September 1997 | 18/10/1999 | 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 | Terminated | N/A | 5 Years | Both | 25 | N/A | NULL | |
| 58 | NCT00176904 (ClinicalTrials.gov) | January 1995 | 12/9/2005 | 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 | NULL | Completed | N/A | N/A | All | 135 | Phase 2/Phase 3 | United States |
| 59 | NCT00007020 (ClinicalTrials.gov) | January 1992 | 6/12/2000 | 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 | Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;Cholestasis | Drug: Cholic Acids | Travere Therapeutics, Inc. | Children's Hospital Medical Center, Cincinnati | Completed | N/A | N/A | All | 85 | Phase 3 | United States |
| 60 | EUCTR2015-002805-13-FR (EUCTR) | 24/07/2017 | 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 INN or Proposed INN: elivaldogenum tavalentivecum Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA | bluebird bio, Inc. | NULL | NA | Female: no Male: yes | 25 | Phase 3 | United States;France;Argentina;Australia;Algeria;United Kingdom | |||
| 61 | EUCTR2017-001684-21-FR (EUCTR) | 23/03/2018 | Phase 1, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD) | Phase 1, Open-Label, Dose Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of NV1205 in Pediatric Male Subjects with Childhood Cerebral Adrenoleukodystrophy (CCALD) | Childhood Cerebral Adrenoleukodystrophy (CCALD);Therapeutic area: Diseases [C] - Hormonal diseases [C19] | Product Name: Sobetirome Product Code: NV1205 INN or Proposed INN: Sobetirome Other descriptive name: QTXEW2-04-AF, EW2, GC-1, QRX-431 | NeuroVia, Inc. | NULL | NA | Female: no Male: yes | 25 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Argentina;Ukraine;Australia;Russian Federation;Chile;Colombia;United Kingdom |