Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease
Niemann-Pick Disease, Type C
Drug: Sham Procedure Control
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease|
- NPC Clinical Severity Score [ Time Frame: 52 weeks ]Data for NPC score rating will be provided to a centralized independent blinded rater who will analyze all NPC information for all subjects and assign the NPC Clinical Severity Score.
- Clinician and Caregiver Clinical Global Impression of Change [ Time Frame: 52 weeks ]The CGIC will be evaluated using a 7-point Likert scale.
- Time to get up and go test [ Time Frame: 52 weeks ]
- 9-hole peg test [ Time Frame: 52 weeks ]
- Percentage of patients with clinical worsening [ Time Frame: from week 26 through week 52 based on first dose being week 1 ]
- European Quality of Life-5 Dimensions Quality of Life Rating (EQ-5D QoL) [ Time Frame: 52 weeks ]
- Cerebrospinal fluid (CSF) biomarkers [ Time Frame: 52 weeks ]CSF will be collected from subjects receiving study drug and will be stored for possible biomarker analysis.
- Plasma protein biomarkers [ Time Frame: 52 weeks ]Plasma samples will be collected and stored for possible biomarker analysis
|Study Start Date:||September 2015|
|Estimated Study Completion Date:||March 2018|
|Estimated Primary Completion Date:||March 2017 (Final data collection date for primary outcome measure)|
Part A: Three (3) different VTS-270 lumbar intrathecal doses (900 mg, 1200 mg, and 1800 mg) administered IT every 2 weeks for 8 weeks in 9 subjects; 3 subjects will receive sham treatment. Dose for Part B to be selected for the remainder of the study.
Part B: Approximately 51 subjects, including 12 from Part A, will receive lumbar intrathecal infusions of VTS-270 or sham (randomized 2:1) every 2 weeks for a total of 52 weeks.
Part C: Open-label extension with IT treatment every 2 weeks.
Lumber intrathecal infusion of VTS-270 (dose to be selected after Part A)
Sham Comparator: Sham Procedure Control
Sixteen (16) subjects, including 3 from Part A.
Drug: Sham Procedure Control
No experimental drug administered to patients. All intrathecal administrations are simulated.
Other Name: Sham group
Non-clinical studies and a Phase 1 clinical trial suggest that intrathecal administration of 2-hydroxypropyl- β-cyclodextrin (VTS-270) in patients with neurologic manifestations of Niemann-Pick Type C1 (NPC1) disease has the potential to slow the rate of progression of their neurologic disease.
Niemann-Pick Type C1 (NPC1) disease is a rare, neurodegenerative, inherited, autosomal recessive lysosomal lipid storage disorder primarily in children and teenagers. The disease is characterized by the inability to properly metabolize cholesterol and other lipids within the cell due to mutations in the NPC1 gene causing unesterified cholesterol to accumulate in the brain, liver and spleen.
This study is a multicenter, multinational, prospective, randomized, double-blind, sham-controlled, 3-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every 2 weeks, with a planned enrollment of approximately 51 subjects with NPC1 disease. The study will be conducted in three parts: Parts A, B, and C.
Part A will evaluate 3 different dose levels of VTS-270 in 9 subjects and 3 sham control subjects to determine the dose level for Parts B and C.
Part B will evaluate the safety and efficacy of the dose selected from Part A compared to sham control in 51 subjects, including the 12 subjects from Part A.
Part C will be an open-label extension phase of the study to subjects who either complete Part B or are subjects in Part B who have met rescue therapy criteria.
Subjects in Part C will receive treatment with VTS-270 until the product is licensed or the program is terminated.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02534844
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