Don't get left behind! The modernized is coming. Check it out now.
Say goodbye to!
The new site is coming soon - go to the modernized
Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04059406
Recruitment Status : Terminated (Favorable safety and tolerability were seen, but efficacy results in the mid-stage study did not meet Ionis' minimum target product profile to justify further development.)
First Posted : August 16, 2019
Last Update Posted : May 1, 2023
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Brief 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.

Condition or disease Intervention/treatment Phase
Beta Thalassemia Intermedia Drug: sapablursen Phase 2

Detailed 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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 24, 2020
Actual Primary Completion Date : March 28, 2023
Actual Study Completion Date : March 28, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arm Intervention/treatment
Experimental: sapablursen
A single injection of sapablursen at multiple dose levels, administered subcutaneously every 4 weeks
Drug: sapablursen
sapablursen administered subcutaneously
Other Names:
  • ISIS 702843

Primary Outcome Measures :
  1. Percentage of participants with a ≥ 1.0 grams per deciliter (g/dL) increase from Baseline in hemoglobin (Hb) at Week 27 [ Time Frame: Baseline and Week 27 ]

Secondary Outcome Measures :
  1. Percentage of participants with a ≥ 1.5 grams per deciliter (g/dL) increase from Baseline in hemoglobin (Hb) at Week 53 [ Time Frame: Baseline and Week 53 ]
  2. Percentage of participants with a ≥ 1.0 milligram of iron per gram of dry weight of liver (mg Fe/g) decrease from Baseline in liver iron concentration (LIC) at Week 53 [ Time Frame: Baseline and Week 53 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04059406

Show Show 19 study locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Layout table for additonal information
Responsible Party: Ionis Pharmaceuticals, Inc. Identifier: NCT04059406    
Other Study ID Numbers: ISIS 702843-CS2
2019-003505-96 ( EudraCT Number )
First Posted: August 16, 2019    Key Record Dates
Last Update Posted: May 1, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Ionis Pharmaceuticals, Inc.:
Beta Thalassemia
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn