Study to Evaluate Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic Syndrome
Status:
Unknown status
Trial end date:
2019-10-23
Target enrollment:
Participant gender:
Summary
Background:
- Eosinophils are white blood cells that fight infections. In people with hypereosinophilic
syndrome (HES), eosinophil levels are too high and can damage their organs. HES is usually
treated with steroids, but steroids can cause side effects and stop working over time.
Researchers want to see if a drug called dexpramipexole, being developed by Knopp
Pharmaceuticals, can help people with HES to reduce their steroid dose.
Objective:
- To test whether dexpramipexole can reduce the steroid dose needed to control eosinophilia
and HES symptoms.
Eligibility:
- Adults 18 and older with HES who respond to steroids, but need more than 10 mg daily to
control eosinophilia and symptoms.
Design:
- The study will last 9 months with 6 visits to NIH.
- Participants will be screened with medical history, physical exam, and urine and blood
samples.
- Participants steroids will be tapered to the lowest effective dose. During this time,
blood will be drawn weekly. Participants will take this dose for 2 weeks before starting
the study drug.
- Participants will take the study drug twice daily by mouth for 12 weeks along with
steroids. The steroid dose will not be decreased during this time and participants will
be seen monthly for a medical history, physical examination and blood work.
- Just before and 12 weeks after starting the study drug, the following tests will be
performed:
- medical history and physical exam
- blood and urine tests
- lung function tests
- electrocardiogram (measures heart electrical activity)
- echocardiogram (takes pictures of the heart using sound waves)
- bone marrow biopsy (a needle inserted into the hip bone that removes bone marrow cells
for study)
- After 12 weeks, the participants steroid dose will be tapered again to the lowest
effective dose while on study drug.
- Two weeks after the lowest effective dose is reached, participants will return for a
medical history, physical examination, blood work, lung and heart tests.
- Participants who respond to the study drug may be able to continue to receive the drug
on a planned separate study.
- Four weeks after stopping the study drug, participants will have medical history,
physical exam, and blood tests.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)