Clinical Trial: Study to Evaluate Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic Syndrome

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional



Official Title: An Open-Label, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic Syndrome

Brief 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 histor