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Beta Thalassemia Intermedia| IONIS TMPRSS6-LRx

Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-LRx

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.

Purpose of Study

The purpose is to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of IONIS TMPRSS6-LRx administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.


18 - 65
Female Icon + Male Icon

Key Inclusion / Exclusion Criteria

Inclusion Criteria:

  • Willingness to comply with study procedures
  • Clinical diagnosis of Beta-Thalassemia Intermedia with genotypic confirmation
  • Non-transfusion dependent, as defined by: no more than 6 transfusions in the past 12-month period, and no transfusions in the 8-week period prior to Day 1
  • Mean Hb within the range of 6.0-10.0 g/dL, inclusive at Screening
  • LIC within the range of 3.0-20.0 mg Fe/g dry weight, inclusive
  • If using chelators, must be on a stable dose for at least 3 months with LIC > 5.0 mg Fe/g dry weight and serum ferritin > 300 nanograms per milliliter (ng/mL)
  • Females must be non-pregnant and non-lactating, and either surgically sterile or postmenopausal
  • Males must be surgically sterile, abstinent or using an acceptable contraceptive method

Exclusion Criteria:

  • Clinically significant abnormalities in lab values, medical history, or physical examination
  • α-globin gene triplication
  • Symptomatic splenomegaly
  • Platelet count < lower limit of normal (LLN) or > 1,000 x 10^9/L
  • Significant concurrent/recent coagulopathy, history of non-traumatic significant bleeding; history of immune thrombocytopenic purpura (ITP); current use of SC anti-coagulants; history of thrombotic events, including stroke or DVT
  • Clinically significant renal, liver or cardiac dysfunction
  • Uncontrolled hypertension (> 140 mm Hg systolic or > 90 mm Hg diastolic)
  • Fasting blood glucose > 2.0 × upper limit of normal (ULN)
  • Inability to have a magnetic resonance imaging (MRI) scan
  • Known history or positive test for human immunodeficiency virus (HIV), hepatitis C (HCV), or hepatitis B (HBV)
  • Active infection requiring systemic antiviral or antimicrobial therapy
  • Regular excessive use of alcohol
  • Recent start of hydroxyurea (6 months prior to Day 1)
  • Treatment with or recent exposure to another investigational drug, biological agent, ASO, small interfering ribonucleic acid (siRNA), or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer; or treatment with or exposure to:
  • sotatercept (ACE-011), luspatercept (ACE-536), or ruxolitinib within 4 months of Screening
  • hematopoietic stimulating agents or any hypoxia-inducible factor prolyl hydroxylase inhibitors within 8 weeks of Day 1
  • prior bone marrow transplant, stem cell transplant, or gene therapy
  • Surgery associated with significant blood loss within 4 months of Screening, splenectomy within 12 months of Screening, or splenectomy scheduled during treatment


Beta Thalassemia Intermedia




Phase 2

Clinical Trials ID


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Start Date / End Date:

09 2020 - 10 2022





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