Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
NCT ID: NCT03643562
Last Updated: 2024-01-17
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
27 participants
INTERVENTIONAL
2018-06-18
2021-11-02
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Adrabetadex
Participants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks.
Adrabetadex
Administered via lumbar puncture (LP) and IT infusion
Interventions
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Adrabetadex
Administered via lumbar puncture (LP) and IT infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Is male or female and at least 4 years of age at time of screening.
* Has a confirmed diagnosis of NPC and exhibits neurologic symptoms.
* Has written informed consent/assent to participate.
* Has the ability to undergo LP and IT drug administration.
* If taking miglustat (Zavesca®), must have been on a stable dose for the past 6 weeks and be willing to remain on a stable dose for the duration of participation in the study, or discontinue miglustat use at least 6 weeks before entry into the study (Day 1).
* If has a history of seizures, the condition is adequately controlled as per protocol requirements.
* Agrees to discontinue any investigational treatments (other than adrabetadex) for at least 1 month before first dose on Day 1.
* If engaging in heterosexual sex, agrees to use a protocol-defined method of contraception throughout the study, and until 30 days after completing the study.
* Has a responsible adult who the investigator determines is able and willing to comply with study requirements and a parent/guardian who will accompany the participant to study visits.
Exclusion Criteria
* Weighs less than 15 kg.
* Has a history of hypersensitivity reactions to any product containing 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) or has a history of hypersensitivity reactions or allergy to anesthesia/sedation.
* Has received treatment with any investigational product (other than adrabetadex) within 1 month prior to Day 1 of treatment.
* Is pregnant or nursing.
* Has systemic infection or uncontrolled psychosis.
* Has known history of a bleeding disorder.
* Has used anticoagulants within 2 months of entry into the study.
* Per protocol, or in the opinion of the investigator:
1. has laboratory values that would preclude participation
2. has suspected infection of the central nervous system (CNS)
3. has a spinal deformity that could impact performance of repeated LPs
4. has a serious skin infection in the lumbar region or evidence of obstructive or normal pressure hydrocephalus
5. is unable to comply with the study requirements
6. has a medical condition that might increase the risk of participation
4 Years
ALL
No
Sponsors
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Mandos LLC
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Study Lead
Role: STUDY_DIRECTOR
Mandos LLC
Locations
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University of Arkansas System
Little Rock, Arkansas, United States
Loma Linda University Health System
Loma Linda, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States
Children's Specialty Center of Nevada
Las Vegas, Nevada, United States
NYU Langone Medical Center
New York, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Dell Children's Medical Center of Central Texas
Austin, Texas, United States
Carilion Medical Center, Carilion Clinic
Roanoke, Virginia, United States
Countries
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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VTS-270-001
Identifier Type: -
Identifier Source: org_study_id
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