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

  • End date
    Oct 28, 2022
  • participants needed
  • sponsor
    Ionis Pharmaceuticals, Inc.
Updated on 28 January 2021
Ionis Pharmaceuticals
Primary Contact
Chronic Care Center (3.3 mi away) Contact
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The purpose is to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of IONIS TMPRSS6-LRx administered subcutaneously to participants with non-transfusion dependent -Thalassemia Intermedia.


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.

Condition Beta Thalassemia Intermedia
Treatment IONIS TMPRSS6-Lrx
Clinical Study IdentifierNCT04059406
SponsorIonis Pharmaceuticals, Inc.
Last Modified on28 January 2021


Yes No Not Sure

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
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
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