Status:
COMPLETED
First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH
Lead Sponsor:
BioCryst Pharmaceuticals
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX9930 in healthy s...
Detailed Description
Up to 6 sequential ascending dose cohorts are planned to be dosed in a sequential manner in Part 1 of the study. Eight subjects will be treated with a single dose of the study drug per dose cohort (6 ...
Eligibility Criteria
Inclusion
- Key Inclusion Criteria (Parts 1, 2, and 3):
- Able to provide written informed consent
- Acceptable birth control measures for male subjects and women of childbearing potential
- Is expected to adequately comply with required study procedures and restrictions
- Key Inclusion Criteria (Parts 1 and 2):
- Body mass index (BMI) of 18.0 to 32.0 kg/m2.
- Males and non-pregnant, non-lactating females age 18 to 55 years.
- Part 2: Must have recent vaccination against Neisseria meningitidis and must be negative for colonisation by Neisseria meningitidis
- Key Inclusion Criteria (Part 3 only):
- Male or non-pregnant, non-lactating female subjects ≥ 18 years old
- Have been diagnosed with PNH and have laboratory values indicative of active PNH
- Subjects naïve to both eculizumab and ravulizumab treatment, who have no access to, or are considered unsuitable for proven effective alternative options as per the local standard of care OR subjects currently receiving treatment with eculizumab or ravulizumab have been on a stable dose of eculizumab or ravulizumab for 6 months
- Must have recent vaccination against Neisseria meningitidis
- Key Exclusion Criteria (Parts 1 and 2):
- Clinically significant medical history, current medical or psychiatric condition that, in the opinion of the Investigator or Sponsor, would interfere with the subject's ability to participate in the study or increase the risk of participation for that subject.
- Clinically significant ECG finding or laboratory/urinalysis abnormality
- Use of prescription or over the counter medication within 14 days of dosing
- Participation in any other investigational drug study within 90 days of screening
- Recent or current history of alcohol or drug abuse within the last 12 months
- Current smokers and those who have smoked within the last 12 months
- Positive serology for HIV or active infection with HBV or HCV
- Pregnant or nursing
- Donation or loss of greater than 400 mL of blood within 3 months
- History of severe hypersensitivity to any drug or Neisseria meningitidis vaccines (Part 2)
- Subject has recently received a live attenuated vaccine within 30 days of dosing or another type of vaccine within 14 days of Day 1
- Key Exclusion Criteria (Part 3):
- Apart from a diagnosis of PNH, any clinically significant medical or psychiatric condition or medical history, other than those associated with PNH disease, that, in the opinion of the Investigator or Sponsor, would interfere with the subject's ability to participate in the study or participation would increase the risk for that subject
- Active bacterial infection
- Hereditary complement deficiency
- History of hematopoietic stem cell /marrow transplantation
- Current participation in any other investigational drug study or participation in an investigational drug study within 30 days of the screening visit
- History of meningococcal disease
- Positive drugs of abuse screen at screening visit
- Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating
- History of severe hypersensitivity to any drug
Exclusion
Key Trial Info
Start Date :
March 3 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 25 2021
Estimated Enrollment :
168 Patients enrolled
Trial Details
Trial ID
NCT04330534
Start Date
March 3 2020
End Date
January 25 2021
Last Update
February 17 2022
Active Locations (4)
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1
Study Site
Vienna, Austria
2
Study Site
Bloemfontein, South Africa
3
Study Site
Pretoria, South Africa
4
Study Site
London, United Kingdom