Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
NCT04176653 · Status: WITHDRAWN · Phase: PHASE1 · Type: INTERVENTIONAL
Last updated 2020-04-27
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.
Conditions
- Non-transfusion-dependent Thalassemia
- Low Risk Myelodysplastic Syndrome
Interventions
- DRUG
-
SLN124 is a GalNAc conjugated double stranded fully modified siRNA. Sodium chloride 0.9% w/v is used as Placebo
IMP will be provided as a solution to be injected by qualified clinical staff in patient's abdomen, upper arms or front of the thighs.
Sponsors & Collaborators
-
Silence Therapeutics plc
lead INDUSTRY
Study Design
- Allocation
- RANDOMIZED
- Purpose
- TREATMENT
- Masking
- DOUBLE
- Model
- PARALLEL
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2019-08-20
- Primary Completion
- 2021-09-30
- Completion
- 2021-10-14
Countries
- Bulgaria
- United Kingdom
Study Locations
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