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An Open-label Treatment Protocol for VTS-270 in Patients With Neurologic Manifestations of NPC

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03643562
Expanded Access Status : Available
First Posted : August 23, 2018
Last Update Posted : February 27, 2019
Information provided by (Responsible Party):
Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company

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

Condition or disease Intervention/treatment
Niemann-Pick Type C Disease Drug: VTS270

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.

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)

Intervention Details:
  • Drug: VTS270
    Lumber intrathecal infusion of VTS-270

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

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

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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Contact: Christopher Hitchens 800-556-3314

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United States, Arkansas
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
United States, California
Loma Linda University Health System
Loma Linda, California, United States, 92354
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Children's Hospital of Orange County
Orange, California, United States, 92868
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, District of Columbia
Children's National Hospital
Washington, District of Columbia, United States, 20010
United States, Georgia
Rare Disease Research
Atlanta, Georgia, United States, 30318
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Minnesota
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
United States, Nevada
Children's Specialty Center of Nevada
Las Vegas, Nevada, United States, 89109
United States, New York
NYU Langone Medical Center
New York, New York, United States, 10017
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Tennessee
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States, 37203
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
Dell Children's Medical Center of Central Texas
Austin, Texas, United States, 78723
United States, Virginia
Carilion Medical Center
Roanoke, Virginia, United States, 24014
Sponsors and Collaborators
Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company

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Responsible Party: Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company Identifier: NCT03643562     History of Changes
Other Study ID Numbers: VTS-270-001
First Posted: August 23, 2018    Key Record Dates
Last Update Posted: February 27, 2019
Last Verified: August 2018

Keywords provided by Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company:

Additional relevant MeSH terms:
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Neurologic Manifestations
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Nervous System Diseases
Signs and Symptoms
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central 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