Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Phase 3

Terminated

Brief Summary: This study was amended from expanded 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.

Recruitment & Eligibility

Inclusion Criteria

To be included in the study, a participant must meet the following 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

Participants will be excluded from the study if they meet any of the following 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

Arm && Interventions

Group	Intervention	Description
Adrabetadex	Adrabetadex	Participants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Baseline up to Week 134	An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

Secondary Outcome Measures

Name	Time	Method

Locations (17): Rare Disease Research, LLC
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Atlanta, Georgia, United States
Rush University Medical Center
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Chicago, Illinois, United States
Gillette Children's Specialty Healthcare
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Saint Paul, Minnesota, United States
University of Arkansas System
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Little Rock, Arkansas, United States
Children's Hospital Los Angeles
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Los Angeles, California, United States
Children's Specialty Center of Nevada
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Las Vegas, Nevada, United States
NYU Langone Medical Center
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New York, New York, United States
Children's National Hospital
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Washington, District of Columbia, United States
The Children's Hospital at TriStar Centennial
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Nashville, Tennessee, United States
Children's Hospital of Orange County
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Orange, California, United States
Loma Linda University Health System
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Loma Linda, California, United States
Cincinnati Children's Hospital Medical Center
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Cincinnati, Ohio, United States
Oregon Health & Science University
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Portland, Oregon, United States
Dell Children's Medical Center of Central Texas
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Austin, Texas, United States
Vanderbilt University Medical Center
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Nashville, Tennessee, United States
Carilion Medical Center, Carilion Clinic
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Roanoke, Virginia, United States
Children's Hospital Colorado
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Aurora, Colorado, United States