TerminatedPhase 2/3

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Stopped: Terminated by previous Sponsor decision

United States66 participantsStarted 2015-12-23

Plain-language summary

Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.

Who can participate

Age range4 Years – 21 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria: One of the following is required for inclusion into VTS301 Part C: * Has agreed to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 weeks dosing regimen * The investigator has received prior written authorization from the sponsor for the participant to enter VTS301 Part C on an amended dose and/or regimen * Has received prior written authorization from Vtesse to enroll directly into Part C Exclusion criteria: * None of the inclusion criteria are applicable

What they're measuring

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Timeframe: Baseline up to 5 years

Trial details

NCT IDNCT04958642

SponsorMandos LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-04-11

Results submitted2023-08-04

View on ClinicalTrials.gov More Niemann-Pick Disease, Type C trials