Trial
NCT04059406
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
Phase IITERMINATEDEFFICACY/FUTILITYConfidence: MEDIUM
Sponsor
Ionis Pharmaceuticals, Inc.
Collaborators
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Condition
Beta Thalassemia Intermedia
Intervention
sapablursen
Why stopped
Favorable safety and tolerability were seen, but efficacy results in the mid-stage study did not meet Ionis' minimum target product profile to justify further development.
Provenance
Dataset: 2026-07-02 • Source: ClinicalTrials.gov API v2 • Last update: 2025-02-18