Paroxysmal Nocturnal HemoglobinuriaPhase 1/2NCT04330534
First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH
A Phase 1/2 study testing BCX9930 in people with Paroxysmal Nocturnal Hemoglobinuria, run by BioCryst Pharmaceuticals at 4 sites in 3 countries.
Trial 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 subjects and in subjects with paroxysmal nocturnal hemoglobinuria (PNH; Part 3). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- BCX9930
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 168 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 3 Mar 2020to 25 Jan 2021
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
- 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
Show all 10Show fewer
- 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
Who cannot join
- 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
Show all 20Show fewer
- 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
- 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
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
About this study
Official title: A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria
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 subjects and in subjects with paroxysmal nocturnal hemoglobinuria (PNH; Part 3). Pharmacokinetics is an analysis of how the body handles the study drug BCX9930 and pharmacodynamics is an analysis of the activity that the study drug BCX9930 may have in the body.
Read the 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 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo). Escalation to the next higher dose level will occur only after completion of a review of clinical safety and pharmacokinetics by the Sponsor and PI.
Up to 7 ascending, multiple dose cohorts will be enrolled in a sequential manner in Part 2 of the study. In Cohorts 1 through 3, twelve subjects will be treated with either a 7-day or 14-day course of study drug (10 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo) administered orally. In Cohorts 4 through 7, twelve subjects will be treated with a 3-day course of study drug (10 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo) administered orally. The daily dose may be split into 2 times daily (BID) or 3 times daily (TID) dosing for the multiple ascending dose part as needed. Escalation to the next higher dose level in Part 2 will occur only after completion of a review of clinical safety and pharmacokinetics by the Sponsor and PI.
Part 3 of the study consists of up to 2 sequential ascending multiple dose cohorts of up to 8 subjects; each cohort may enroll up to 4 subjects with PNH who are naïve to both eculizumab and ravulizumab and up to 4 subjects with PNH who are currently being treated with either eculizumab or ravulizumab. In each cohort, subjects will receive one daily dose of BCX9930 on Days 1 to 14 and a higher daily dose on Days 15 to 28. Cohort 2 will start after independent data monitoring committee (DMC) review of Cohort 1 data and communication of their evaluation to Part 3 investigators. In South Africa, subjects that have clinical benefit from BCX9930 will be allowed to continue dosing for up to 48 weeks.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- BCX9930, Placebo
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, first-in-human, Factor D, complement inhibitor, alternative pathway inhibitor, BioCryst, proof-of-concept
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCX9930Drug
BCX9930 capsules for oral administration
PlaceboDrug
placebo to match BCX9930 capsules for oral administration
| Study group | Role | What they receive |
|---|---|---|
| BCX9930 | Experimental | Parts 1, 2 and 3 Receives: Drug: BCX9930 |
| Placebo | Placebo comparator | Parts 1 and 2 only Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of graded treatment-emergent adverse events
Measured: Part 1: Day 16
Incidence of graded treatment-emergent adverse events
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded treatment-emergent adverse events
Measured: Part 3:Day 44 or Week 50 (South Africa only)
Incidence of graded laboratory chemistry abnormalities
Measured: Part 1: Day 16
Incidence of graded laboratory chemistry abnormalities
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded laboratory chemistry abnormalities
Measured: Part 3:Day 44 or Week 50 (South Africa only)
Incidence of graded urinalysis abnormalities
Measured: Part 1: Day 16
Incidence of graded urinalysis abnormalities
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded urinalysis abnormalities
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Incidence of graded coagulation abnormalities
Measured: Part 1: Day 16
Incidence of graded coagulation abnormalities
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded coagulation abnormalities
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Incidence of graded hematology abnormalities
Measured: Part 1: Day 16
Incidence of graded hematology abnormalities
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded hematology abnormalities
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in blood pressure
Measured: Part 1: Day 16
Change from baseline in blood pressure
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change from baseline in blood pressure
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in temperature
Measured: Part 1: Day 16
Change from baseline in temperature
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change from baseline in temperature
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in heart rate
Measured: Part 1: Day 16
Change from baseline in heart rate
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change from baseline in heart rate
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in respiratory rate
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (PR interval)
Measured: Part 1: Day 16
Change in Electrocardiogram (PR interval)
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (PR interval)
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (QRS interval)
Measured: Part 1: Day 16
Change in Electrocardiogram (QRS interval)
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (QRS interval)
Measured: Part 3: Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (RR interval)
Measured: Part 1: Day 16
Change in Electrocardiogram (RR interval)
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (RR interval)
Measured: Part 3:Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (QT interval)
Measured: Part 1: Day 16
Change in Electrocardiogram (QT interval)
Measured: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (QT interval)
Measured: Part 3:Day 44 or Week 50 (South Africa only)
Secondary outcomes (12)
Plasma BCX9930 Cmax
Measured: plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 Tmax
Measured: plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 AUCinf
Measured: plasma PK parameters are based on blood sampling through Day 4 for Part 1
Plasma BCX9930 t1/2
Measured: plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 AUCtau
Measured: plasma PK parameters are based on blood sampling through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Serum AP complement activity
Measured: Part 1:through Study Day 4; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and Part 3 through Day 28 or Week 48 (South Africa only)
Plasma Factor Bb
Measured: Part 1:through Study Day 4; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and Part 3 through Day 28 or Week 48 (South Africa only)
Number of blood transfusions
Measured: Part 3:baseline through Day 28 or Week 50 (South Africa only)
Lactate dehydrogenase
Measured: Part 3: absolute and change from baseline through Day 28 or Week 50 (South Africa only)
Hemoglobin
Measured: Part 3: absolute and change from baseline through Day 28 or Week 50 (South Africa only)
Absolute reticulocyte count
Measured: Part 3: values and change from baseline through Day 28 or Week 50 (South Africa only)
Haptoglobin
Measured: Part 3: absolute and change from baseline through Day 28 or Week 50 (South Africa only)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2020
- Study start3 Mar 2020
- Primary completion11 Nov 2020
- Study completion25 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Each cohort in Part 3 is enrolled as a single group; Parts 1 and 2 follow a parallel study model.
- Masking
- None (open label)
- Enrolment
- 168 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Antionio Risitano, Principal investigator, University of Naples
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04330534
- Sponsor's protocol ID
- BCX9930-101
- Status last verified
- Feb 2022
- First submitted
- 23 Mar 2020
- Last updated
- 17 Feb 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT04330534
- Acronym / sponsor ID
- BCX9930-101
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, first-in-human, Factor D, complement inhibitor, alternative pathway inhibitor, BioCryst, proof-of-concept
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 168 participants (actual)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 3 Mar 2020 (actual)
- Primary completion
- 11 Nov 2020 (actual)
- Study completion
- 25 Jan 2021 (actual)
- First posted
- 1 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 subjects and in subjects with paroxysmal nocturnal hemoglobinuria (PNH; Part 3). Pharmacokinetics is an analysis of how the body handles the study drug BCX9930 and pharmacodynamics is an analysis of the activity that the study drug BCX9930 may have in the body.
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 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo). Escalation to the next higher dose level will occur only after completion of a review of clinical safety and pharmacokinetics by the Sponsor and PI.
Up to 7 ascending, multiple dose cohorts will be enrolled in a sequential manner in Part 2 of the study. In Cohorts 1 through 3, twelve subjects will be treated with either a 7-day or 14-day course of study drug (10 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo) administered orally. In Cohorts 4 through 7, twelve subjects will be treated with a 3-day course of study drug (10 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo) administered orally. The daily dose may be split into 2 times daily (BID) or 3 times daily (TID) dosing for the multiple ascending dose part as needed. Escalation to the next higher dose level in Part 2 will occur only after completion of a review of clinical safety and pharmacokinetics by the Sponsor and PI.
Part 3 of the study consists of up to 2 sequential ascending multiple dose cohorts of up to 8 subjects; each cohort may enroll up to 4 subjects with PNH who are naïve to both eculizumab and ravulizumab and up to 4 subjects with PNH who are currently being treated with either eculizumab or ravulizumab. In each cohort, subjects will receive one daily dose of BCX9930 on Days 1 to 14 and a higher daily dose on Days 15 to 28. Cohort 2 will start after independent data monitoring committee (DMC) review of Cohort 1 data and communication of their evaluation to Part 3 investigators. In South Africa, subjects that have clinical benefit from BCX9930 will be allowed to continue dosing for up to 48 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Each cohort in Part 3 is enrolled as a single group; Parts 1 and 2 follow a parallel study model.
| Arm | Type | Receives |
|---|---|---|
| BCX9930 | Experimental | Drug: BCX9930Parts 1, 2 and 3 |
| Placebo | Placebo comparator | Drug: PlaceboParts 1 and 2 only |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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
- Body mass index (BMI) of 18.0 to 32.0 kg/m2.
- Males and non-pregnant, non-lactating females age 18 to 55 years.
Key exclusion criteria (20 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BCX9930BCX9930 capsules for oral administration | Drug | BCX9930 |
| Placeboplacebo to match BCX9930 capsules for oral administration | Drug | Placebo |
Primary outcome
Incidence of graded treatment-emergent adverse events
Time frame: Part 1: Day 16
Incidence of graded treatment-emergent adverse events
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded treatment-emergent adverse events
Time frame: Part 3:Day 44 or Week 50 (South Africa only)
Incidence of graded laboratory chemistry abnormalities
Time frame: Part 1: Day 16
Incidence of graded laboratory chemistry abnormalities
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded laboratory chemistry abnormalities
Time frame: Part 3:Day 44 or Week 50 (South Africa only)
Incidence of graded urinalysis abnormalities
Time frame: Part 1: Day 16
Incidence of graded urinalysis abnormalities
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded urinalysis abnormalities
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Incidence of graded coagulation abnormalities
Time frame: Part 1: Day 16
Incidence of graded coagulation abnormalities
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded coagulation abnormalities
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Incidence of graded hematology abnormalities
Time frame: Part 1: Day 16
Incidence of graded hematology abnormalities
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Incidence of graded hematology abnormalities
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in blood pressure
Time frame: Part 1: Day 16
Change from baseline in blood pressure
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change from baseline in blood pressure
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in temperature
Time frame: Part 1: Day 16
Change from baseline in temperature
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change from baseline in temperature
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in heart rate
Time frame: Part 1: Day 16
Change from baseline in heart rate
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change from baseline in heart rate
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Change from baseline in respiratory rate
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (PR interval)
Time frame: Part 1: Day 16
Change in Electrocardiogram (PR interval)
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (PR interval)
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (QRS interval)
Time frame: Part 1: Day 16
Change in Electrocardiogram (QRS interval)
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (QRS interval)
Time frame: Part 3: Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (RR interval)
Time frame: Part 1: Day 16
Change in Electrocardiogram (RR interval)
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (RR interval)
Time frame: Part 3:Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (QT interval)
Time frame: Part 1: Day 16
Change in Electrocardiogram (QT interval)
Time frame: Part 2: Day 23 (cohorts with 3 and 7 day dosing duration) or Day 31 (cohorts with 14 day dosing duration)
Change in Electrocardiogram (QT interval)
Time frame: Part 3:Day 44 or Week 50 (South Africa only)
12 secondary outcomes
Plasma BCX9930 Cmax
Time frame: plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 Tmax
Time frame: plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 AUCinf
Time frame: plasma PK parameters are based on blood sampling through Day 4 for Part 1
Plasma BCX9930 t1/2
Time frame: plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 AUCtau
Time frame: plasma PK parameters are based on blood sampling through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Serum AP complement activity
Time frame: Part 1:through Study Day 4; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and Part 3 through Day 28 or Week 48 (South Africa only)
Plasma Factor Bb
Time frame: Part 1:through Study Day 4; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and Part 3 through Day 28 or Week 48 (South Africa only)
Number of blood transfusions
Time frame: Part 3:baseline through Day 28 or Week 50 (South Africa only)
Lactate dehydrogenase
Time frame: Part 3: absolute and change from baseline through Day 28 or Week 50 (South Africa only)
Hemoglobin
Time frame: Part 3: absolute and change from baseline through Day 28 or Week 50 (South Africa only)
Absolute reticulocyte count
Time frame: Part 3: values and change from baseline through Day 28 or Week 50 (South Africa only)
Haptoglobin
Time frame: Part 3: absolute and change from baseline through Day 28 or Week 50 (South Africa only)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Antionio Risitano principal investigator
University of Naples
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BCX9930 in people with Paroxysmal Nocturnal Hemoglobinuria, run by BioCryst Pharmaceuticals at 4 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in South Africa, Austria, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of graded treatment-emergent adverse events (time frame: Part 1: Day 16). The study lists 37 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2022.