Safety and tolerability of CSL889 in Sickle Cell Disease
Research type
Research Study
Full title
A 2-Part, Phase 1, Multicenter, Single-Dose, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CSL889 in Adult Patients with Sickle Cell Disease
IRAS ID
273136
Contact name
Rachel Kesse-Adu
Contact email
Sponsor organisation
CSL Behring LLC
Eudract number
2019-001870-27
Duration of Study in the UK
years, 22 months, days
Research summary
The study drug CSL889 is being developed for the treatment of vaso-occlusive crisis (also known as pain crisis) that occurs in patients with sickle cell disease.
All humans have hemopexin. However, patients with sickle cell disease may have very little hemopexin. The main job of hemopexin is to combine with a substance in blood called haem and to clear haem from the blood. The haem comes from red blood cells when red blood cells die. Research suggests that haem may play a role in pain crisis. CSL889 is a human protein called hemopexin. If CSL889 combines with haem, similar to native hemopexin, it may help clear haem from the blood.
This is the first study of CSL889 in humans. This study will measure levels of CSL889 in the blood and see how well it is tolerated (how safe it is). The study will also look for changes in several blood tests related to sickle cell disease to see how the drug might affect these measures. We will study how genes (pieces of DNA) affects the way the body responds to or handles the medicine.
The study duration for a subject is expected to last up to 8 weeks, but the overall study duration will be 12 to 18 months.
Approximately 24 subjects with sickle cell disease will take part, at 6 to 10 study sites. This includes up to 6 groups with 4 subjects in each group.
REC name
Wales REC 1
REC reference
19/WA/0341
Date of REC Opinion
20 Jan 2020
REC opinion
Further Information Favourable Opinion