Study Stopped
Sponsor had determined that the benefit-risk profile of adrabetadex was not favorable and the clinical development program for adrabetadex was discontinued.
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
1 other identifier
interventional
27
1 country
17
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jun 2018
Typical duration for phase_3
17 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 18, 2018
CompletedFirst Submitted
Initial submission to the registry
August 19, 2018
CompletedFirst Posted
Study publicly available on registry
August 23, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 2, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
November 2, 2021
CompletedResults Posted
Study results publicly available
January 17, 2024
CompletedJanuary 17, 2024
December 1, 2023
3.4 years
August 19, 2018
November 17, 2023
December 22, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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.
Baseline up to Week 134
Study Arms (1)
Adrabetadex
EXPERIMENTALParticipants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks.
Interventions
Administered via lumbar puncture (LP) and IT infusion
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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:
- has laboratory values that would preclude participation
- has suspected infection of the central nervous system (CNS)
- has a spinal deformity that could impact performance of repeated LPs
- has a serious skin infection in the lumbar region or evidence of obstructive or normal pressure hydrocephalus
- is unable to comply with the study requirements
- has a medical condition that might increase the risk of participation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Mandos LLClead
Study Sites (17)
University of Arkansas System
Little Rock, Arkansas, 72205, United States
Loma Linda University Health System
Loma Linda, California, 92354, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
Rare Disease Research, LLC
Atlanta, Georgia, 30318, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101, United States
Children's Specialty Center of Nevada
Las Vegas, Nevada, 89109, United States
NYU Langone Medical Center
New York, New York, 10017, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Dell Children's Medical Center of Central Texas
Austin, Texas, 78723, United States
Carilion Medical Center, Carilion Clinic
Roanoke, Virginia, 24014, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
This study was terminated early when the Sponsor had determined that the benefit-risk profile of adrabetadex was not favorable and the clinical development program for adrabetadex was discontinued.
Results Point of Contact
- Title
- Executive Vice President, Regulatory Affairs
- Organization
- Mandos, LLC
Study Officials
- STUDY DIRECTOR
Clinical Study Lead
Mandos LLC
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 19, 2018
First Posted
August 23, 2018
Study Start
June 18, 2018
Primary Completion
November 2, 2021
Study Completion
November 2, 2021
Last Updated
January 17, 2024
Results First Posted
January 17, 2024
Record last verified: 2023-12
Data Sharing
- IPD Sharing
- Will not share