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.



Eligible Ages
Over 4 Years
Eligible Genders
Accepts 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 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-╬▓-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

Study Design

Phase 3
Study Type
Intervention Model
Single Group Assignment
Intervention Model Description
Continued treatment for children at least 4 years of age
Primary Purpose
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Participants receive prescribed adrabetadex by intra-thecal injection
  • Drug: Adrabetadex
    administered via lumbar puncture and intra-thecal infusion
    Other names:
    • VTS270
    • VTS-270

Recruiting Locations

University of Arkansas System
Little Rock, Arkansas 72205

Loma Linda University Health System
Loma Linda, California 92354

Children's Hospital Colorado
Aurora, Colorado 80045

Rush University Medical Center
Chicago, Illinois 60612

Gillette Children's Specialty Healthcare
Saint Paul, Minnesota 55101

The Children's Hospital at TriStar Centennial
Nashville, Tennessee 37203

Carilion Medical Center, Carilion Clinic
Roanoke, Virginia 24014

More Details

Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company

Study Contact

Medical Information Call Center


Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.