Trials / Completed
CompletedNCT04718844
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome
A Randomised, Single-blind, Placebo-controlled, Phase 1, Single-ascending and Multiple-dose Study in Adult Subjects With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome to Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124.
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 44 (actual)
- Sponsor
- Silence Therapeutics plc · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c. doses and multiple doses in healthy male and female subjects. Up to 7 cohorts of 56 patients with Thalassaemia and up to 7 cohorts of 56 patients with MDS will be enrolled. Each subject will receive single or multiple doses of SLN124 or placebo given by subcutaneous (s.c) injection.
Conditions
- Non-transfusion-dependent Thalassemia
- Low Risk Myelodysplastic Syndrome
- Very-Low Risk Myelodysplastic Syndrome
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | SLN124 | SLN124 for subcutaneous (s.c.) injection |
| DRUG | Placebo | Sodium chloride for s.c. injection |
Timeline
- Start date
- 2021-04-14
- Primary completion
- 2023-05-23
- Completion
- 2023-05-23
- First posted
- 2021-01-22
- Last updated
- 2024-01-03
Locations
20 sites across 7 countries: Germany, Israel, Italy, Jordan, Malaysia, Thailand, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04718844. Inclusion in this directory is not an endorsement.