BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy (REDEEM-1 Trial) | Aplastic Anemia & MDS International Foundation Return to top.

ClinicalTrials.gov Identifier:


Contact Info

BioCryst Pharmaceuticals, Inc.


+1 (844) 273-2328 (toll free)


Start: November 2021
End: October 2023

Official Title

BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy (REDEEM-1 Trial)


The purpose of this study is to evaluate the efficacy and safety of BCX9930 as a treatment for PNH in adult patients with ongoing anemia despite treatment with a C5 inhibitor (either eculizumab or ravulizumab). BCX9930 is a Factor D inhibitor administered orally (by mouth). The study will recruit approximately 81 patients worldwide and each individual’s participation will last approximately 1 year. The study will be run in two parts. In part 1, participants will be randomly assigned in a 2:1 ratio to either (a) stop their C5 inhibitor and start oral BCX9930 or (b) remain on the C5 inhibitor for 24 weeks. In part 2, all participants will receive BCX9930. Participants assigned to continue their C5 inhibitor initially will stop treatment after Week 24 and switch to BCX9930. Patients assigned to BCX9930 initially will continue BCX9930 in part 2. At the end of the study (Week 52), participants who are experiencing benefit from BCX9930 treatment may be allowed to continue BCX9930 treatment in a long-term follow-up study.

BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy (REDEEM-1 Trial)

Clinical Trial: NCT05116774


If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.

Active, not recruiting
Associated Drug(s): 
Phase 2
Age Group: 
18 years and older
Accepts Healthy Volunteers: 
Inclusion Criteria: 
  • Body weight >= 40 kg (88 lb).
  • Documented diagnosis of PNH confirmed by flow cytometry (white blood cell clone size >= 10%).
  • Treatment with eculizumab at a stable dose for >= 3 months or ravulizumab at a stable dose for >=6 months.
  • Hemoglobin <= 10.5 g/dL at screening.
  • Current with recommended vaccinations against Neisseria meningitidis and other bacterial infections as needed.
  • If a woman of childbearing potential or a male with a female partner of childbearing potential, willing to practice appropriate contraception to avoid pregnancy throughout the study and at least 30 (women) or 90 (men) days after the study.
  • Willing and able to comply with all study visits and procedures, including twice daily dosing with BCX9930.
  • Able to provide written informed consent to participate.
Exclusion Criteria: 
  • Prior diagnosis of hereditary complement deficiency.
  • History of or a current candidate for bone marrow or other transplant.
  • Ongoing or recent history (<= 30 days) of clinically relevant cardio or cerebrovascular disease or a clinically significant abnormal electrocardiogram (ECG) tracing at screening.
  • Ongoing or recent history (<= 5 years) of malignancy (cancer) (some exceptions apply).
  • Ongoing or recent history of (<= 14 days) serious bacterial, fungal, or viral infection.
  • Ongoing or recent (<= 30 days) participation in a clinical trial with an investigational (experimental) therapy.
  • Treatment with ATG (anti-thymocyte globulin) in past 6 months.
  • Recently started treatment (<= 28 days) with a red blood cell or platelet growth factor, danazol, or iron supplementation regimen at an unstable dose.
  • Use of any other medication prohibited by the study protocol.
  • Elevated liver function test or bilirubin levels at screening that is not explained by hemolysis or Gilbert’s syndrome.
  • HIV or hepatitis B or C infection (unless infection is well-controlled on antiviral therapy).
  • Positive drugs of abuse screen (unless supported by prescription).
  • Known severe sensitivity to drugs (eg, prior anaphylactic reaction).
  • Pregnant or breastfeeding, or intending to get pregnant during the study.
  • Any other clinically significant medical or psychiatric condition that could interfere with study participation or increase the risk of study participation.
  • The above list is not intended to be exhaustive; each individual’s participation must be confirmed by the responsible physician conducting the study.


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