Purpose of this Study
This study starts with a screening visit to see if a person can join. After that, there are thirteen study visits over about twenty weeks. During this time, participants will receive three shots of the study medicine called DISC 3045 given under the skin. All study visits include blood tests, and one visit will include two blood draws. People may also choose to stay in the study longer for another twelve weeks. If they continue, they will receive three more shots of the study medicine and have seven more study visits with blood tests.
Who Can Participate?
Eligibility
People can join this study if they are 18 years old or older and have sickle cell disease. This includes two types called HbSS and HbSC. Their hemoglobin level must be at least 9. They must have had between one and ten sickle cell pain crises in the past year, or have had health problems linked to sickle cell disease such as eye damage, small strokes that do not cause symptoms, bone damage, or hearing loss. Their iron levels must also be within a certain range based on their type of sickle cell disease.
Age Range
18-110
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study is testing a new medicine called DISC 3405 to see if it is safe. The medicine lowers iron levels in the body and is being studied for people with sickle cell disease. Lower iron levels may help reduce sickle hemoglobin in red blood cells. This can help keep the cells from becoming sickle shaped. It may also help stop red blood cells from breaking apart, which can lead to pain and trouble breathing.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
A Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants with Sickle Cell Disease
Study Website
Principal Investigator
John
Strouse
Protocol Number
PRO00118958
NCT ID
NCT07187973
Phase
I
Enrollment Status
Pending Open to Enrollment