VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
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|ClinicalTrials.gov Identifier: NCT02534844|
Recruitment Status : Active, not recruiting
First Posted : August 28, 2015
Last Update Posted : July 29, 2021
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.
This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords).
In Parts A/B, two out of every three patients will receive the study drug. The third patient will receive 1 to 2 small needle pricks at the location where the LP and IT injection is normally made (sham control).
In Part C, all participants will receive study drug, as described in the Part C registration record.
Start date for this record is the first day a participant was enrolled in Parts A/B. The trial is actually continuing until the last primary outcome measure of safety data are collected from Part C participants. The last primary outcome measure of safety, along with final adverse events results will be posted in the separate Part C registration record.
|Condition or disease||Intervention/treatment||Phase|
|Niemann-Pick Disease, Type C||Drug: Parts A/B: Adrabetadex Other: Parts A/B: Sham Control||Phase 2 Phase 3|
Non-clinical studies and a Phase 1 clinical trial suggest that intrathecal administration of 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 plans to enroll about 51 participants with NPC1 disease. It will be conducted in three parts: Parts A, B, and C.
- Part A will evaluate 3 different dose levels of VTS-270 in 12 participants to determine the dose level for Parts B and C.
- In Part B, 39 more participants will join the original 12 to evaluate the safety and effectiveness of the dose selected from Part A compared to sham control.
- Part C will be an open-label extension phase of the study for Part B participants who either complete Part B or have met rescue therapy criteria, as well as participants entering Part C from other trials.
Participants in Part C will receive treatment with VTS-270 until the product is licensed or the program is terminated (anticipated within 5 years).
Final results will be posted in the Part C registration record (NCT04958642).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||51 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||In Parts A/B (see other registration for Part C description)|
|Masking:||Double (Care Provider, Investigator)|
|Masking Description:||While it is a double-blind trial, the participant and outcomes assessor will be blinded, as well as the Care Provider and Investigator.|
|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|
|Actual Study Start Date :||October 2015|
|Estimated Primary Completion Date :||October 2021|
|Estimated Study Completion Date :||October 2021|
Experimental: Parts A/B: Sham Control
Participants receive no study drug
Other: Parts A/B: Sham Control
No experimental drug is administered to patients - intrathecal administrations are simulated by skin prick
Parts A/B: Adrabetadex
Participants receive adrabetadex
Drug: Parts A/B: Adrabetadex
900 - 1800 milligram (mg) of adrabetadex administered every 2 weeks via lumbar intrathecal infusion
- Parts A/B: Composite Niemann Pick Type C Severity Scale (NPC-SS) [ Time Frame: Baseline, Week 52 ]Each of four NPC-SS components (ambulation, cognition, fine motor, and swallowing) are rated on a scale from 0 (better) to 5 (worse). The highest (worst) possible score is 20.
- Parts A/ B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) [ Time Frame: Week 52 ]The study doctor rates his impression of the change in each participant's condition at week 52 on a scale from marked improvement (1) to marked worsening (7).
- Parts A/B: NPC-SS Total Score (Excluding Hearing and Auditory Brainstem Response) at Baseline and Week 52 [ Time Frame: Baseline, Week 52 ]
Each of four NPC-SS components (ambulation, cognition, fine motor, and swallowing) are rated on a scale from 0 (better) to 5 (worse). The highest (worst) possible score is 20.
The hearing domain and auditory brainstem response modifiers will be removed from the total NPC-SS total score for this measure.
- Parts A/ B: Number of Participants Classified as Responders by Their Caregiver at Week 52 [ Time Frame: Week 52 ]Caregiver rates the global impression of change at Week 52. Participants who receive the caregiver's rating of no change, minimally improved, moderately approved, or markedly improved will be classified as responders. The number of participants classified as responders will be recorded.
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 ClinicalTrials.gov identifier (NCT number): NCT02534844
|Study Director:||Clinical Study Lead||Mandos LLC|