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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

SLN124-001

Status

Withdrawn

Type

Interventional

Phase

Phase 1

Placebo

No

Accepting

18-75 Years
18+ Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Ended 4 years ago

What is this trial about?

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.

What are the participation requirements?

Yes

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%

No

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

Locations

Location

Status