search
Back to results

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

Primary Purpose

Niemann-Pick Type C Disease

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Adrabetadex
Sponsored by
Mandos LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Niemann-Pick Type C Disease focused on measuring Niemann-Pick, NPC, NPC Type 1 (NPC1)

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Arkansas System
  • Loma Linda University Health System
  • Children's Hospital Los Angeles
  • Children's Hospital of Orange County
  • Children's Hospital Colorado
  • Children's National Hospital
  • Rare Disease Research, LLC
  • Rush University Medical Center
  • Gillette Children's Specialty Healthcare
  • Children's Specialty Center of Nevada
  • NYU Langone Medical Center
  • Cincinnati Children's Hospital Medical Center
  • Oregon Health & Science University
  • The Children's Hospital at TriStar Centennial
  • Vanderbilt University Medical Center
  • Dell Children's Medical Center of Central Texas
  • Carilion Medical Center, Carilion Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Adrabetadex

Arm Description

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

Outcomes

Primary Outcome Measures

Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Full Information

First Posted
August 19, 2018
Last Updated
October 7, 2022
Sponsor
Mandos LLC
search

1. Study Identification

Unique Protocol Identification Number
NCT03643562
Brief Title
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Official Title
Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2022
Overall Recruitment Status
Completed
Study Start Date
June 18, 2018 (Actual)
Primary Completion Date
November 2, 2021 (Actual)
Study Completion Date
November 2, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mandos LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Niemann-Pick Type C Disease
Keywords
Niemann-Pick, NPC, NPC Type 1 (NPC1)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Continued treatment for children at least 4 years of age
Masking
None (Open Label)
Allocation
N/A
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Adrabetadex
Arm Type
Experimental
Arm Description
Participants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks.
Intervention Type
Drug
Intervention Name(s)
Adrabetadex
Other Intervention Name(s)
VTS-270
Intervention Description
Administered via lumbar puncture (LP) and IT infusion
Primary Outcome Measure Information:
Title
Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame
Up to 134 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Study Lead
Organizational Affiliation
Mandos LLC
Official's Role
Study Director
Facility Information:
Facility Name
University of Arkansas System
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
Loma Linda University Health System
City
Loma Linda
State/Province
California
ZIP/Postal Code
92354
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Children's National Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Gillette Children's Specialty Healthcare
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101
Country
United States
Facility Name
Children's Specialty Center of Nevada
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89109
Country
United States
Facility Name
NYU Langone Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10017
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
The Children's Hospital at TriStar Centennial
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Dell Children's Medical Center of Central Texas
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
Carilion Medical Center, Carilion Clinic
City
Roanoke
State/Province
Virginia
ZIP/Postal Code
24014
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

We'll reach out to this number within 24 hrs