Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
Sponsored by Silence Therapeutics plc
About this trial
Last updated 5 years ago
Study ID
Status
Type
Phase
Placebo
Accepting
Not accepting
Trial Timing
Ended 4 years ago
What is this trial about?
What are the participation requirements?
Inclusion Criteria
- Age ≥ 18yrs; BMI 18-35 kg/m2
- β-thalassaemia intermedia or compound heterozygous HbE/β-thalassaemia
- Non-transfusion dependent: ≤ 5 units red cells in last 6 months and transfusion-free for ≥8 weeks
- Hb between 5 & 11 g/dL
- Ferritin > 250 µg/L and /or liver iron ≥ 3mg Fe/g dry weight and TSAT >40%
Exclusion Criteria
- Haemoglobin S/β-thalassaemia, homozygous β-0 thalassaemia or α thalassaemia
- ALT/AST > 1.5 x upper limit normal or cirrhosis
- eGFR < 60 mL/min/1.73m2
- Platelets <100 or > 1000 x 109/L
- Untreated B12/folate deficiency
- Iron chelation therapy unless stable for ≥8 weeks
- Daily NSAID, therapeutic dose anticoagulant, ESA ≤12 weeks or stable dosing of hydroxyurea ≤ 6 months
- Significant cardiac disease (MI in 6 months, NYHA class III-IV heart failure, long QT)
- HIV or active hepatitis B/C or malignancy within 5 year