Phase 1 Thalassaemia Clinical Trials

2 recruitingPhase 1

What is a Phase 1 trial?

Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.

Showing 1–2 of 2 trials

Recruiting

Phase 1

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Beta-ThalassemiaSickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)+1 more

Daniel Bauer10 enrolled1 locationNCT06647979

Completed

Phase 1

An interventional study to evaluate the effects of dosages on the Pharmacokinetics (PK, the measure of how the human body processes a substance), Pharmacodynamics (PD, the measure of what a substance does to the human body), Tolerability (how well a substance is tolerated by participants), and Safety of different dosages of IONIS-TMPRSS6-Lrx when given to healthy participants as either a single subcutaneous (SC, an injection just under the skin) dose, or as multiple SC doses.

Thalassaemia

INCResearch Australia Pty Ltd44 enrolled1 locationACTRN12617000458336