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

Beta Thalassemia Intermedia Clinical Trial

Official title:
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

Clinical Trial Summary

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

Clinical Trial Description

This is a multi-center, randomized, open-label study in up to 45 participants. The duration of each participant in the study will be approximately 29 months and will include an approximately 2-month screening period, a 24-month treatment period, and a 3-month post-treatment period. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04059406
Study type	Interventional
Source	Ionis Pharmaceuticals, Inc.
Contact
Status	Terminated
Phase	Phase 2
Start date	September 24, 2020
Completion date	March 28, 2023

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See also
Status	Clinical Trial	Phase
Recruiting	`NCT04432623` - The BENeFiTS Trial in Beta Thalassemia Intermedia	Phase 1/Phase 2
Terminated	`NCT01571635` - Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.	Phase 2
Completed	`NCT01642758` - Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon	Phase 2
Completed	`NCT01609595` - Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand	Phase 2