Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
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|ClinicalTrials.gov Identifier: NCT03643562|
Recruitment Status : Active, not recruiting
First Posted : August 23, 2018
Last Update Posted : July 29, 2021
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.
The study design will be like the expanded access by Rush University, but for children at least 4 years of age at screening.
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)|
|Estimated Enrollment :||60 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|
|Estimated Primary Completion Date :||October 2021|
|Estimated Study Completion Date :||November 2021|
Participants receive prescribed adrabetadex by intra-thecal injection
administered via lumbar puncture and intra-thecal infusion
- Clinician Global Impression of Change (Clinician-CGIC) [ Time Frame: within approximately 3 years ]The Clinician-CGIC is evaluated using a 7-point Likert scale ranging from 1 (marked improvement) from screening to 7 (marked worsening from screening) at the participant's last visit
- Composite Niemann Pick Type C Severity Scale (NPC-SS) [ Time Frame: within approximately 3 years ]Each of four NPC-SS components (ambulation, cognition, fine motor, and swallowing) are rated on a scale from 0 (better) to 5 (worse). The highest (worst) possible score is 20.
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|