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

NCT04059406 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2025-02-18

Study results available
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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.

Conditions

  • Beta Thalassemia Intermedia

Interventions

DRUG

sapablursen

sapablursen administered subcutaneously

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-24
Primary Completion
2023-03-28
Completion
2023-03-28
FDA Drug
Yes

Countries

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

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04059406 on ClinicalTrials.gov