Autopsy report · NCT04059406

Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)

“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.”

The sponsor’s own words in the registry.

Life of this trial

• Registered 1 year 1 month before it started.
• Ran for about 2 years 6 months before it stopped.
• 29 participants enrolled.
• Results were posted on the registry 1 year 11 months after it ended.

Vital statistics

Status

Terminated

Phase

Phase 2

Sponsor

Ionis Pharmaceuticals, Inc.

Sponsor type

Industry

Conditions

Beta Thalassemia Intermedia

Interventions

sapablursen

Participants

29

Sites

19

Countries

Australia, Greece, Lebanon, Thailand, Turkey (Türkiye)

Trials that died the same way

• 2-Year Extension Study of Azeliragon in Subjects With Alzheimer's Disease (STEADFAST Extension)
  “Not due to safety but due to a lack of efficacy at the 5 mg azeliragon dose.” (57% match in meaning)

Look deeper

• The official registry record (the source of truth)
• Record history: every change the sponsor made
• Open it in the database

Everything here comes from the current ClinicalTrials.gov record. The timeline uses the record’s dates; dates marked planned are the sponsor’s plans. The cause of death is an automatic, unverified label. This is research information, not medical advice.