20. Adrenoleukodystrophy Clinical trials / Disease details
Clinical trials : 53 / Drugs : 88 - (DrugBank : 31) / Drug target genes : 23 - Drug target pathways : 123
Showing 1 to 10 of 53 diseases
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 |
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1 | NCT05200104 (ClinicalTrials.gov) | July 2022 | 6/1/2022 | Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy(AMN) Form of X-linked Adrenoleukodystr ... | 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) An Open-label Phase 2a Study to Assess the Pharmacokinetics and Pharmacodynamic of PXL065 Parameters ... | ALD (Adrenoleukodystrophy) | Drug: PXL065 | Poxel SA | NULL | Not yet recruiting | 18 Years | 65 Years | Male | 12 | Phase 2 | NULL |
2 | NCT05146284 (ClinicalTrials.gov) | July 2022 | 16/11/2021 | Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy(AMN) Form of X-linked Adrenoleukodystr ... | 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) A Randomized Open-label Phase 2a Study to Assess PXL770 After 12 Weeks of Treatment in Male Subjects ... | Adrenomyeloneuropathy | Drug: PXL770 | Poxel SA | NULL | Not yet recruiting | 18 Years | 65 Years | Male | 24 | Phase 2 | NULL |
3 | 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 AdrenoleukodystrophyWho Were Treated With Lenti-D Drug ... | Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Longterm Follow-up of Subjects With Cerebral AdrenoleukodystrophyWho Were Treated With Lenti-D Drug ... | 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] Cerebral Adrenoleukodystrophy(CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term ... | 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 Product Name: Lenti-D Drug Product INN or Proposed INN: Elivaldogene autotemcel Other descriptive name ... | 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 | ||
4 | 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 Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait ... | 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 |
5 | 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) Phase 1b, Multi-center, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, ... | Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) | Drug: VK0214;Other: Placebo | Viking Therapeutics, Inc. | NULL | Active, not recruiting | 18 Years | N/A | All | 36 | Phase 1 | United States |
6 | 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) A study to evaluate the effects of a new drug called MIN-102 on diseaseprogression in boys with cere ... | 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) An open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystro ... | 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] Cerebral X-linked Adrenoleukodystrophy(cALD) MedDRA version: 20.0;Level: PT;Classification code 1005 ... | 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 Product Name: 5-[4-[2-(5-(1-hydroxyethyl)-2- pyridinyl)ethoxy]benzyl]-2,4- thiazolidinedione hydroch ... | Minoryx Therapeutics S.L. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 13 | Phase 2 | Germany;Spain;France;United States | ||
7 | 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. Effect of Plasma Exchange With Albumin in Patients With Adrenomyeloneuropathy: Unicentric, Single Ar ... | Adrenomyeloneuropathy;Adrenoleukodystrophy | Drug: Albumin solution | Onofre, Aurora Pujol, M.D. | NULL | Active, not recruiting | 18 Years | 65 Years | Male | 5 | Phase 2/Phase 3 | Spain |
8 | 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 Effect of plasma exchange by albumin replacement in Adrenomyeloneuropathy: unicentric, single arm, p ... | 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] Adrenomyeloneuropathy MedDRA version: 20.0;Level: LLT;Classification code 10069075;Term: Adrenomyelo ... | Trade Name: Albunorm® 5%, Product Name: Albumin INN or Proposed INN: ALBUMIN Other descriptive name: ALBUMIN Trade Name: Albunorm® 5%, Product Name: Albumin INN or Proposed INN: ALBUMIN Other descriptive name: AL ... | Aurora Pujol Onofre | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | Spain | ||
9 | 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) Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adren ... | 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) A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarab ... | 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] Cerebral Adrenoleukodystrophy(CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term ... | 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 Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name ... | 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 | ||
10 | 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) A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy(Cald) to Ass ... | 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) An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystro ... | Cerebral Adrenoleukodystrophy | Drug: MIN-102 | Minoryx Therapeutics, S.L. | NULL | Recruiting | 2 Years | 12 Years | Male | 13 | Phase 2 | France;Germany;Spain |