An observational study in X-Linked Adrenoleukodystrophy, sponsored by NeuroVia, Inc.. Status unknown at 3 sites in United States. Open to male participants aged 2 Years to 13 Years. Per ClinicalTrials.gov, last updated 2018-08-08.
Sponsored by NeuroVia, Inc. · Observational
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to patient treatment are necessary.
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to the patient treatment are necessary.
Patients with X-ALD will be recruited and invited to attend a baseline visit. After eligible patients are enrolled, retrospective and baseline data will be collected. After enrollment, patients will be seen approximately every 6 months until they meet withdrawal criteria or the Sponsor terminates the study.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's planned enrollment of 100 is below the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →NeuroVia, Inc. is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients will be selected from participating sites, referring physicians, and outreach communities
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Males patients 2-13 years of age with a confirmed diagnosis of X-ALD that fall into one of the following:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Loes score
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Time frame: baseline and 24 weeks
Plasma VLCFA levels
Change from baseline plasma VLCFA levels
Time frame: baseline and 24 weeks
Neurological symptoms
Development of new neurological symptoms throughout the study
Time frame: baseline and 24 weeks
Plan to share: No
No publications or documents are linked to this record.
Started Feb 2018 (actual). Primary completion is estimated for Jun 2021, 39 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in May 2018. You cannot join it, but the record below documents what was studied.
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NeuroVia, Inc.