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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 : Active, not recruiting
First Posted : August 23, 2018
Last Update Posted : July 29, 2021
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.

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 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
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : November 2021

Arm Intervention/treatment
Experimental: Adrabetadex
Participants receive prescribed adrabetadex by intra-thecal injection
Drug: Adrabetadex
administered via lumbar puncture and intra-thecal infusion
Other Names:
  • VTS270
  • VTS-270

Primary Outcome Measures :
  1. 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

  2. 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.

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 lumbar puncture (LP) and intra-thecal (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:

Patients 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 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 before 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: July 29, 2021
Last Verified: July 2021
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 Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
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