Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
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|ClinicalTrials.gov Identifier: NCT03643562|
Recruitment Status : Completed
First Posted : August 23, 2018
Last Update Posted : May 24, 2022
This study was amended from extended access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.
Participants who were already taking adrabetadex will receive their stable dose. Participants who have not ever taken it will start by receiving 400 mg.
Participants will receive treatment every 2 weeks until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason. Participants will not receive additional study treatment after their participation in this protocol.
|Condition or disease||Intervention/treatment||Phase|
|Niemann-Pick Type C Disease||Drug: Adrabetadex||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||27 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Continued treatment for children at least 4 years of age|
|Masking:||None (Open Label)|
|Official Title:||Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)|
|Actual Study Start Date :||June 18, 2018|
|Actual Primary Completion Date :||November 2, 2021|
|Actual Study Completion Date :||November 2, 2021|
Participants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks.
Administered via lumbar puncture (LP) and IT infusion
Other Name: VTS-270
- Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to 134 Weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03643562
|Study Director:||Clinical Study Lead||Mandos LLC|