CLINICAL_TRIALS ClinicalTrials.gov record update

A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway

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Record identity and dates

Source: ClinicalTrials.gov
Source record ID: NCT04221451
Title field or adapter rule: protocolSection.identificationModule.officialTitle
Ingested At: 2026-04-25T22:28:33.250874+00:00
Published At: 2026-01-28T00:00:00+00:00
Record Updated At: 2026-01-28
Results Posted At: 2026-01-28
Timestamp Basis: source retrieval time was not retained by the feed adapter
Date limitation: source retrieval time was not retained by the feed adapter

protocolSection.conditionsModule.conditions: ["Tay-Sachs Disease", "Sandhoff Disease"]
protocolSection.statusModule.lastUpdatePostDateStruct.date: "2026-01-28"
protocolSection.identificationModule.nctId: "NCT04221451"
protocolSection.identificationModule.officialTitle: "A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway"
protocolSection.statusModule.overallStatus: "TERMINATED"
protocolSection.designModule.phases: ["PHASE3"]
protocolSection.statusModule.resultsFirstPostDateStruct.date: "2026-01-28"

Intervention details are not present in the imported fields for this record. No drug-condition relationship is inferred from co-listing.

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