Purpose of this Study
In this study, people will take a medicine called R289 by mouth once or twice a day. The study has two parts: one part is to find the best dose, and the other part is to test two different doses.
You may take the medicine at home or at the clinic. You will visit the clinic regularly for tests like blood work, physical check-ups, and heart exams. Some visits may take up to 8 hours.
You can stay in the study as long as the medicine is helping and the side effects are not too strong.
Who Can Participate?
Eligibility
People in this study must:
- Be 18 years old or older.
- Have a blood problem called lower-risk myelodysplastic syndromes (LR-MDS).
- Have tried other treatments that did not work or the disease came back.
- May need regular red blood cell transfusions to feel better.
- Cannot be pregnant or breastfeeding.
Age Range
18-100
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 R289 to see if it can help people with a blood problem called lower-risk myelodysplastic syndromes (LR-MDS). Doctors want to learn how safe the medicine is and how well it works. They also want to find the best dose and see if it can help people need fewer blood transfusions.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
No
Spanish Materials Available
No
Study Details
Full Title
An Open-Label, Phase 1b Study of R289, an IRAK1/4 Inhibitor, in Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) who are Relapsed/Refractory/Resistant to Prior Therapies
Principal Investigator
Harry
Erba
Protocol Number
PRO00118897
NCT ID
NCT05308264
Phase
I
Enrollment Status
Pending Open to Enrollment