Other Clinical Trial - Study in Beta-thalassaemia or Myelodysplastic

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

beta-Thalassemia
Low Risk Myelodysplastic Syndrome
Myelodysplastic Syndromes
Non-transfusion-dependent Thalassemia
Preleukemia
Syndrome
Thalassemia

See also
Status	Clinical Trial	Phase
Completed	`NCT04364269` - Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia	Phase 2
Completed	`NCT02993224` - Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet	Phase 2
Completed	`NCT04718844` - 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	Phase 1

See also

Status

Clinical Trial

Phase

Completed

NCT04364269 - Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia

Phase 2

Completed

NCT02993224 - Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet

Phase 2

Completed

NCT04718844 - 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

Phase 1

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT04176653
Study type	Interventional
Source	Silence Therapeutics plc
Contact
Status	Withdrawn
Phase	Phase 1
Start date	August 20, 2019
Completion date	October 14, 2021

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms