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

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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	NCT05146284 (ClinicalTrials.gov)	December 2022	16/11/2021	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 ...	Adrenomyeloneuropathy	Drug: PXL770	Poxel SA	NULL	Not yet recruiting	18 Years	65 Years	Male	24	Phase 2	NULL
2	NCT05200104 (ClinicalTrials.gov)	December 2022	6/1/2022	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 ...	ALD (Adrenoleukodystrophy)	Drug: PXL065	Poxel SA	NULL	Not yet recruiting	18 Years	65 Years	Male	12	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 Adrenomyeloneuropat ...	A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrat ...	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 adrenomyeloneuropathyto assess PXL770's behaviour, a new potential medicine, ...	A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters ...	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 AdrenoleukodystrophyWho Were Treated With Lenti-D Drug ...	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 ...	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
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 ...	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, ...	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 cere ...	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 1005 ...	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	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 Ar ...	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, p ...	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: AL ...	Aurora Pujol Onofre	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	5	Phase 2	Spain

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