Prospective, open-label, treatment protocol to provide expanded access to VTS-270 to eligible Niemann-Pick type C disease (NPC) patient



Eligible Ages
Over 4 Years
Eligible Genders
Accepts Healthy Volunteers

Inclusion Criteria

  1. Patient is male or female and at least 4 years of age at time of screening.
  2. Patient has a confirmed diagnosis of NPC.
  3. Patient or parent/guardian must provide written informed consent to participate in the treatment protocol. In addition to parental consent, assent to participate must also be sought from minor children.
  4. Patient has the ability to undergo LP and IT drug administration.
  5. 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 treatment protocol. Alternatively, patients may elect to discontinue miglustat use and be eligible for entry into the treatment protocol after undergoing a minimum 6-week washout period prior to study Day 1.
  6. If a patient has a history of seizures, the condition must be adequately controlled, i.e., the pattern of seizure activity must be stable and the patient must be on a stable dose and regimen of antiepileptic medication during the 1 month prior to screening, with no change in dose or regimen up to and including the day of the first dose of VTS-270 (Day 1).
  7. Patients not currently receiving VTS-270 agree to discontinue any other investigational treatment for at least 1 month prior to first dose of VTS-270 (Day 1).
  8. Patients who are females of childbearing potential (not surgically sterile) must use a medically acceptable method of contraception and must agree to continue use of this method for the duration of the treatment protocol and for 30 days after participation in the treatment protocol. Acceptable methods of contraception include barrier method with spermicide, intrauterine device, steroidal contraceptive in conjunction with a barrier method, abstinence, or same-sex partner.
  9. Patient or parent/guardian must possess the ability, in the clinician's opinion, to understand and comply with treatment protocol requirements, including clinical outcome measurements and instructions, for the entire duration of the treatment protocol.
  10. Caregiver, parent, guardian, or responsible adult must be able and willing to accompany the patient to treatment protocol visits.

Exclusion Criteria

  1. Patient weighs less than 15 kg.
  2. Patient has uncontrolled psychosis.
  3. Patient 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.
  4. Patient has received treatment with any investigational product (other than VTS-270) within 1 month prior to Day 1 of treatment.
  5. Patient is a female who is pregnant or nursing.
  6. Patient has suspected infection of the CNS or any systemic infection.
  7. Patient has a spinal deformity that, in the opinion of the clinician, could impact the ability to perform repeated LPs.
  8. Patient, in the opinion of the clinician, has a serious skin infection in the lumbar region within 2 months of entry into the treatment protocol.
  9. Patient has neutropenia, defined as an absolute neutrophil count of less than 1.0 × 109/L.
  10. Patient has thrombocytopenia (platelet count of less than 75 × 109/L).
  11. Patient has an activated partial thromboplastin time (aPTT) or prothrombin time (PT) prolonged by greater than 1.5 times the upper limit of normal (ULN) or a known history of a bleeding disorder.
  12. Patient has evidence of obstructive hydrocephalus or normal pressure hydrocephalus.
  13. Patient has recently used anticoagulants (in past 2 weeks prior to first dose of VTS-270 [Day 1].
  14. Patient, in the opinion of the clinician, is unable to comply with the treatment protocol or has a medical condition (e.g., cardiovascular, respiratory, hematologic, neurologic, renal) that would potentially increase the risk of participation

Study Design

Study Type
Expanded Access

Recruiting Locations

University of Arkansas for Medical Sciences
Little Rock, Arkansas 72205

Loma Linda University Health System
Loma Linda, California 92354

Children's Hospital Los Angeles
Los Angeles, California 90027

Children's Hospital of Orange County
Orange, California 92868

Children's Hospital Colorado
Aurora, Colorado 80045

Children's National Hospital
Washington, District of Columbia 20010

Rare Disease Research
Atlanta, Georgia 30318

Rush University Medical Center
Chicago, Illinois 60612

Gillette Children's Specialty Healthcare
Saint Paul, Minnesota 55101

Children's Specialty Center of Nevada
Las Vegas, Nevada 89109

NYU Langone Medical Center
New York, New York 10017

Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229

Oregon Health & Science University
Portland, Oregon 97239

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

Vanderbilt University Medical Center
Nashville, Tennessee 37232

Dell Children's Medical Center of Central Texas
Austin, Texas 78723

Carilion Medical Center
Roanoke, Virginia 24014

More Details

Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company

Study Contact

Christopher Hitchens

Detailed Description

This is a prospective, open-label, treatment protocol to provide expanded access to VTS-270 to eligible Niemann-Pick type C disease (NPC) patients. Eligible patients will receive VTS-270 via intrathecal (IT) lumbar puncture (LP) every 2 weeks, and assessments of safety and exploratory efficacy measures will be performed at prescribed intervals. Patients who have not previously been treated with VTS-270 will start at a dose of 900 mg, and patients who have been previously receiving VTS-270 will receive their current stable dose of VTS-270. Treatment will continue until the clinician considers VTS-270 to be no longer beneficial, the patient withdraws from the treatment protocol, VTS-270 receives marketing authorization in the US, or the development program is discontinued. Patients participating in this treatment protocol should not expect additional access to VTS-270 beyond this treatment protocol.


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.