Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease (NCT01747135) | Clinical Trial Compass
CompletedPhase 1
Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
United States14 participantsStarted 2013-01
Plain-language summary
Background:
\- Hydroxypropyl beta cyclodextrin (HPBCD) is being tested for a disease called Niemann-Pick disease type C1 (NPC1). NPC1 is a genetic disorder that results in gradual loss of nervous system function. Cholesterol and other fats have trouble moving out of the brain cells, which makes the cells work poorly and leads to symptoms. There is no treatment currently approved in the US for NPC1. Researchers want to test if it is safe to use HPBCD for NPC1. They want to see if it can help brain cells process cholesterol better.
Objectives:
\- To test the safety and effectiveness of HPBCD for NPC1.
Eligibility:
\- Individuals between 2 and 25 years of age who have been diagnosed with NPC1 and who have not already received HPBCD in an attempt to treat NPC1.
Design:
* Participants will be screened with a physical exam and medical history. They will provide blood and urine samples for screening. They will also have neurological tests, including tests of hearing, speech and movement.
* Participants will have a lumbar puncture (also called a spinal tap) every month to deliver the drug to the spinal fluid that surrounds the brain. The length of the trial will be determined by the safety and efficacy information that is obtained.
* Treatment will be monitored with frequent blood and urine tests, cerebral spinal fluid tests, hearing and neurological exams.
Who can participate
Age range2 Years – 25 Years
SexALL
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Inclusion criteria
✓. Aged greater than or equal to 2 and less than or equal to 25 years old at time of enrollment, either gender and any ethnicity.
✓. Diagnosis of NPC1 based upon one of the following:
✓. Two NPC1 mutations;
✓. Positive filipin staining and at least one NPC1 mutation;
✓. Vertical supranuclear gaze palsy (VSNGP) in combination with either:
✓. Patients with at least one neurological manifestation of NPC1. For example, but not limited to, hearing loss, vertical supranuclear gaze palsy, ataxia, dementia, dystonia, seizures, dysarthria, or dysphagia.
✓. Ability to travel to the National Institutes of Health Clinical Center (NIH CC) repeatedly for evaluation and follow-up.
✓. If taking miglustat, the patient must have been taking a constant dose of the medication for no less than 3 months prior to baseline evaluation and must be willing to maintain that dose level for the duration of the trial.
Exclusion criteria
What they're measuring
1
24-hydroxycholesterol Area under the curve
Timeframe: Days
Trial details
NCT IDNCT01747135
SponsorVtesse, LLC, a Mallinckrodt Pharmaceuticals Company
. Aged below 2 or above 25 years of age at enrollment in the trial.
✕. Subjects will be excluded if their weight would result in an endotoxin level that would exceed 0.2 EU/kg for either the saline or drug dosing.
✕. Severe manifestations of NPC1 that would interfere with the patient's ability to comply with the requirements of this protocol.
✕. Neurologically asymptomatic patients.
✕. Patients who have received any form of cyclodextrin in an attempt to treat NPC1. Treatment with another drug preparation for another medical indication that contains cyclodextrin as an excipient, will not exclude a patient.
✕. History of hypersensitivity reactions to cyclodextrin or components of the formulation.
✕. Pregnancy or breastfeeding at any time during the study.
✕. Patients with suspected infection of the CNS or any systemic infection.