Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
NCT ID: NCT04176653
Last Updated: 2020-04-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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WITHDRAWN
PHASE1
INTERVENTIONAL
2019-08-20
2021-10-14
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Placebo
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.
0.3 mg/kg
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.
1.0 mg/kg
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.
3.0 mg/kg
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.
10.0 mg/kg
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
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Silence Therapeutics plc
INDUSTRY
Responsible Party
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Locations
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MHAT Dr. Nikola Vasiliev AD
Kyustendil, , Bulgaria
UMHAT Dr. Georgi Stranski AD
Pleven, , Bulgaria
Medical Center COMAC MEDICAL
Sofia, , Bulgaria
UMHAT Sv. Ivan Rilski
Sofia, , Bulgaria
Hammersmith Hospital
London, , United Kingdom
Countries
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Other Identifiers
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SLN124-001
Identifier Type: -
Identifier Source: org_study_id
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