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

Phase 1b Single Ascending and Multiple Dose First-in-man Study in Adult Patients With Non-transfusion-dependent Beta-thalassaemia or Low Risk Myelodysplastic Syndrome to Investigate the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Response of SLN124

Status

Withdrawn

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04176653

Enrollment

Registered

2019-11-25

Start date

2019-08-20

Completion date

2021-10-14

Last updated

2020-04-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Low Risk Myelodysplastic Syndrome, Non-transfusion-dependent Thalassemia

Brief 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.

Interventions

DRUGSLN124 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.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Intervention model description

Multiple Group Assignment

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
# of participants with all AEs as assessed by CTCAE V4.0 included injection site reaction, will be measured from baseline to post dose follow up	Up to two months
12 lead electrocardiogram parameters included PR, QTcF and QTcB will be measured from baseline to post dose follow up	Up to two months
clinical chemistry, haematology, urinalysis, liver function tests (included ALT, AST, GGT) will be measured from baseline to post dose follow up	Up to two months
height, weight, blood pressure, heart rate, respiration rate and temperature will be measured from baseline to post dose follow up	Up to two months

Secondary

Measure	Time frame	Description
Biomarkers will be measured from baseline to post dose follow up	Up to two months	serum hepcidin level (ng/ml), ferritin level (ug/L), Transferrin saturation (%), iron level (umol/L), haemoglobin and reticulocyte count will be analysed by central laboratory.
Pharmacokinetic of SLN124 in plasma from baseline to post dose follow up	Up to two months	Peak Plasma Concentration (Cmax) will be analysed by central laboratory.

Countries

Bulgaria, United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026