X-linked Adrenoleukodystrophy Clinical Trial
Official title:
Registry of X-linked Adrenoleukodystrophy
This study is a observational study conducted through recruiting X-linked adrenoleukodystrophy (X-ALD) patients, to build a comprehensive evaluation and long-term follow-up platform for X-ALD patients, and to provide a theoretical basis for the treatment and management of X-ALD patients.
In this study, X-ALD patients will be selected as study participants. Through a longitudinal collection of genetic, imaging and clinical symptoms data of the patients and carriers. The purpose of this study is to build a comprehensive evaluation and long-term follow-up platform for X-ALD patients. Through the long-term follow-up of imaging and clinical symptoms progress and outcome of X-ALD patients, combined with genetic data, we will improve the relationships between genes and phenotypes, and explore the deep insight in the pathophysiological mechanism of X-ALD, to provide a theoretical basis for the treatment and management of X-ALD patients. ;
Status | Clinical Trial | Phase | |
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Not yet recruiting |
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Recruiting |
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N/A | |
Completed |
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Phase 1 | |
Completed |
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N/A | |
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N/A | |
Completed |
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N/A | |
Active, not recruiting |
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LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
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Completed |
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Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy
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Phase 2 | |
Recruiting |
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Disease Progression in Women With X-linked Adrenoleukodystrophy
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Recruiting |
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Recruiting |
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Recruiting |
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Quality of Life in Women With X-linked Adrenoleukodystrophy
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Withdrawn |
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Phase 1/Phase 2 | |
Withdrawn |
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Phase 1 | |
No longer available |
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Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
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N/A |