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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03643562
Recruitment Status : Completed
First Posted : August 23, 2018
Last Update Posted : May 24, 2022
Information provided by (Responsible Party):
Mandos LLC

Brief Summary:

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Continued treatment for children at least 4 years of age
Masking: None (Open Label)
Primary Purpose: Treatment
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

Arm Intervention/treatment
Experimental: Adrabetadex
Participants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks.
Drug: Adrabetadex
Administered via lumbar puncture (LP) and IT infusion
Other Name: VTS-270

Primary Outcome Measures :
  1. Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to 134 Weeks ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

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 identifier (NCT number): NCT03643562

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Sponsors and Collaborators
Mandos LLC
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Study Director: Clinical Study Lead Mandos LLC
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Responsible Party: Mandos LLC Identifier: NCT03643562    
Other Study ID Numbers: VTS-270-001
First Posted: August 23, 2018    Key Record Dates
Last Update Posted: May 24, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mandos LLC:
NPC Type 1 (NPC1)
Additional relevant MeSH terms:
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Niemann-Pick Disease, Type C
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders