Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of S… (NCT04059406) | Clinical Trial Compass
TerminatedPhase 2
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
Stopped: 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.
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.
Who can participate
Age range18 Years – 65 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 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 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…
What they're measuring
1
Percentage of Participants With a ≥1.0 Grams Per Deciliter (g/dL) Increase From Baseline in Hemoglobin (Hb) at Week 27