Status:
TERMINATED
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
Lead Sponsor:
Ionis Pharmaceuticals, Inc.
Conditions:
Beta Thalassemia Intermedia
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
Brief Summary
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalas...
Detailed Description
This was a multi-center, randomized, open-label study in up to 29 participants. The duration of participation for each subject in the study was approximately 29 months and included an approximately 2-...
Eligibility Criteria
Inclusion
- 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 liver iron concentration (LIC) \> 5.0 mg iron (Fe) per gram of dry weight of liver (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
- 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, antisense oligonucleotide (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
Key Trial Info
Start Date :
September 24 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 28 2023
Estimated Enrollment :
29 Patients enrolled
Trial Details
Trial ID
NCT04059406
Start Date
September 24 2020
End Date
March 28 2023
Last Update
February 18 2025
Active Locations (19)
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1
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia, 2050
2
Monash Medical Centre
Clayton, Victoria, Australia, 3168
3
Royal Perth Hospital
Perth, Western Australia, Australia, 6000
4
Aghia Sophia General Children's Hospital
Athens, Attica, Greece, 115 27