WithdrawnPhase 1

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

Stopped: Study stopped prior to subject administered first dose, due to COVID-19

Bulgaria, United Kingdom0Started 2019-08-20

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

# 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

Timeframe: Up to two months

12 lead electrocardiogram parameters included PR, QTcF and QTcB will be measured from baseline to post dose follow up

Timeframe: 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

Timeframe: Up to two months

height, weight, blood pressure, heart rate, respiration rate and temperature will be measured from baseline to post dose follow up

Timeframe: Up to two months

Trial details

NCT IDNCT04176653

SponsorSilence Therapeutics plc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-09-30

View on ClinicalTrials.gov More Non-transfusion-dependent Thalassemia trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

# 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

Timeframe: Up to two months

12 lead electrocardiogram parameters included PR, QTcF and QTcB will be measured from baseline to post dose follow up

Timeframe: 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

Timeframe: Up to two months

height, weight, blood pressure, heart rate, respiration rate and temperature will be measured from baseline to post dose follow up

Timeframe: Up to two months