A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-?-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease

Vtesse, Inc0 sites51 target enrollmentApril 13, 2016

Overview

No summary available.

Registry

who.int

Start Date

April 13, 2016

End Date

TBD

Last Updated

8 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

Vtesse, Inc

•1\. Male or female subjects, ages 6 to 21 years of age at time of screening with onset of neurological symptoms prior to 15 years of age.
•2\. Diagnosis of NPC1 determined by one of the following:
•a. Two NPC1 mutations;
•b. Positive filipin staining or oxysterol testing and at least one NPC1 mutation;
•c. Vertical supranuclear gaze palsy (VSNGP) in combination with either:
•i. One NPC1 mutation, or
•ii. Positive filipin staining or oxysterol levels consistent with NPC1 disease and no NPC2 mutations.
•3\. Subject or parent/guardian must provide written informed consent to participate in the study. In addition to parental consent, assent to participate must also be sought from minor children.
•4\. Ability to undergo a LP and IT drug administration under monitored anesthesia care (conscious sedation) or if medically necessary, general anesthesia.
•5\. An NPC Clinical Severity Score of 1 through 4, inclusive, in two or more of the following NPC components on the NPC Clinical Severity Scale: ambulation, fine motor skills, or swallowing and a score of 0 through 4 on the cognition component.

•1\. Exclusion criteria as assessed by NPC Clinical Severity Scale:
•a. Unable to walk, wheelchair dependent (ambulation NPC Clinical Severity Score\=5\)
•b. Needs a nasogastric tube or gastric tube for all feedings (swallowing NPC Clinical Severity Score\=5\)
•c. Severe dysmetria (fine motor NPC Clinical Severity Score\=5\)
•d. Minimal cognitive function (cognition NPC Clinical Severity Score\=5\)
•2\. Body weight \< 15 kg.
•3\. Prior treatment at any time with HP\-?\-CD for NPC1 disease. Note: Treatment of other medical conditions with drug preparations containing HP\-?\-CD as an excipient (inactive ingredient) is acceptable and will not exclude a subject from this trial.
•4\. Uncontrolled seizures, specifically: greater than 1 seizure over a 2\-month period (quantified over the 6 months prior to enrollment), subjects requiring antiepileptic medication changes (other than dose adjustment for weight) in the 6 months prior to screening.
•5\. Subjects requiring more than 2 antiepileptic medications to control seizures.
•6\. Subjects on typical or atypical antipsychotics for treatment of psychosis.