X-linked Adrenoleukodystrophy
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CLINICAL_TRIALS trial_result · 2026-08-26Clinical Trial Results: A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) [Phase 1, Phase 2]
Phase 1/2 study evaluating SBT101 gene therapy in adult adrenomyeloneuropathy (AMN) has been terminated; results posted.
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CLINICAL_TRIALS research · 2026-03-02Clinical Trial Update: Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy [Phase 4]
Clinical trial update on a Phase 4 study evaluating treatments for leg symptoms in women with X-linked adrenoleukodystrophy, including restl…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: Longitudinal Study of Neurodegenerative Disorders
This clinical trial update describes a longitudinal study of several neurodegenerative and lysosomal storage disorders. The study is current…
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CLINICAL_TRIALS trial_result · 2025-10-31Clinical Trial Update: A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) [Phase 1, Phase 2]
A Phase 1/2 study evaluating the SBT101 gene therapy for Adrenomyeloneuropathy (a form of X-linked adrenoleukodystrophy) has been terminated…
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CLINICAL_TRIALS trial_result · 2025-10-30Clinical Trial Update: Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
A SwanBio Therapeutics study on disease progression in adults with inherited forms of spastic paraplegia has been terminated, involving Adre…
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CLINICAL_TRIALS research · 2025-09-24Clinical Trial Update: Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
A recruiting clinical trial from Assistance Publique - Hôpitaux de Paris is examining macrophage activation patterns in four leukodystrophie…
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CLINICAL_TRIALS research · 2025-09-09Clinical Trial Update: IT and IV Lentiviral Gene Therapy for X-ALD [NA]
Clinical trial update indicates a lentiviral gene therapy delivered intrathecally and intravenously for X-linked Adrenoleukodystrophy is cur…
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CLINICAL_TRIALS trial_result · 2025-02-20Clinical Trial Update: Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This clinical trial update provides long-term follow-up data for participants with cerebral adrenoleukodystrophy who were treated with the L…
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CLINICAL_TRIALS trial_result · 2025-02-17Clinical Trial Update: A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN [Phase 1]
A Phase 1 pharmacodynamics study of VK0214 in male subjects with adrenomyeloneuropathy (AMN) associated with X-linked adrenoleukodystrophy (…
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CLINICAL_TRIALS research · 2025-01-03Clinical Trial Update: Quality of Life in Women with X-linked Adrenoleukodystrophy
This registry entry notes a recruiting clinical trial evaluating quality of life in women with X-linked Adrenoleukodystrophy.
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CLINICAL_TRIALS withdrawal · 2024-12-16Clinical Trial Update: Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) [Phase 2]
A Phase 2 clinical trial evaluating PXL770 for Adrenomyeloneuropathy (AMN), a form of X-linked adrenoleukodystrophy, has been withdrawn.
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CLINICAL_TRIALS trial_result · 2024-07-12Clinical Trial Update: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases [Phase 2]
This Phase 2 clinical trial update reports the termination of a haploidentical hematopoietic stem cell transplantation study using reduced-i…
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CLINICAL_TRIALS trial_result · 2019-09-10Clinical Trial Update: Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy [Phase 2]
Phase 2 trial update on pioglitazone in patients with adrenomyeloneuropathy and X-linked adrenoleukodystrophy has completed.
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CLINICAL_TRIALS trial_result · 2018-08-29Clinical Trial Update: Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy [NA]
Clinical trial update for an exercise study in women with X-linked adrenoleukodystrophy; trial completed with results posted.
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CLINICAL_TRIALS withdrawal · 2014-02-14Clinical Trial Update: Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) [Phase 1]
Phase 1 safety and pharmacodynamic study of sobetirome in X-linked adrenoleukodystrophy has been withdrawn.
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CLINICAL_TRIALS trial_result · 2011-08-10Clinical Trial Update: The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) [NA]
Completed clinical trial evaluating the effect of bezafibrate on very long chain fatty acid (VLCFA) levels in X-linked adrenoleukodystrophy …