NCT04059406

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

A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Patients With Non-Transfusion Dependent β-Thalassemia Intermedia

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.

Detailed description

This was a multi-center, randomized, open-label study in up to 29 participants. The duration of participation for each subject in the study was approximately 29 months and included an approximately 2-month screening period, a 24-month treatment period, and a 3-month post-treatment period.

Conditions

• Beta Thalassemia Intermedia

Interventions

Type	Name	Description
DRUG	sapablursen	sapablursen administered subcutaneously

Timeline

• Start date: 2020-09-24
• Primary completion: 2023-03-28
• Completion: 2023-03-28
• First posted: 2019-08-16
• Last updated: 2025-02-18
• Results posted: 2025-02-18

Locations

19 sites across 5 countries: Australia, Greece, Lebanon, Thailand, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study