Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124

• Status: Withdrawn
• Trial end date: 2021-10-14
• Target enrollment: 0
• Participant gender: All

Summary

The primary purpose of this study is to determine the safety and tolerability of SLN124 for the treatment of non-transfusion-dependent (NTD) β-thalassaemia and low risk myelodysplastic syndrome.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Silence Therapeutics plc

Criteria

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