Paroxysmal Nocturnal HemoglobinuriaPhase 1NCT06100900
Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 1 study testing BCX10013 in people with Paroxysmal Nocturnal Hemoglobinuria, run by BioCryst Pharmaceuticals at 4 sites in 2 countries.
Trial summary
This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH. Approximately 8 participants will be enrolled in this study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- BCX10013
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 24 Oct 2023to 11 Dec 2024
- Phase
- Phase 1
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or non-pregnant, non-lactating female adults ≥ 18 years old.
- Documented diagnosis of PNH confirmed by flow cytometry.
- Body mass index (BMI) ≤ 40 kg/m^2.
- Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with ravulizumab for at least 12 months prior to the screening visit and have received no treatment with eculizumab or pegcetacoplan for 6 months prior to the screening visit.
- Documentation of current vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae type B [Hib] or willingness to start vaccination series at least 14 days prior to Day 1.
Who cannot join
- Known history of or existing diagnosis of hereditary complement deficiency.
- History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
- Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis.
- History of malignancy within 5 years prior to the screening visit.
- Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
- Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit.
- Receiving iron with an unstable dose (ie, increasing or decreasing) in the 28 days prior to the screening visit.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
South Africa3 sites
BioCryst Investigative Site
Bloemfontein
BioCryst Investigative Site
Cape Town
BioCryst Investigative Site
Pretoria
About this study
Official title: An Open-Label, Multicenter, Intra-Subject Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Therapeutic Potential of BCX10013 in Subjects with Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH. Approximately 8 participants will be enrolled in this study. Participants may receive treatment for up to 52 weeks.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- BCX10013
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, Factor D, complement inhibitor, alternative pathway inhibitor, BioCryst
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCX10013Drug
Multiple dose levels may be tested in this study.
| Study group | Role | What they receive |
|---|---|---|
| BCX10013 | Experimental | Participants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur. Receives: Drug: BCX10013 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings.
Measured: up to 52 weeks
Secondary outcomes (9)
CFB in Lactate Dehydrogenase
Measured: Baseline, Week 52
CFB in the Ratio of Total PNH Red Blood Cell Clone Size to PNH White Blood Cell Clone Size
Measured: Baseline, Week 52
CFB in Hemoglobin
Measured: Baseline, Week 52
Percentage of Participants who are Transfusion-free
Measured: 52 weeks
Percentage of Participants Achieving a Within-subject Clinically Meaningful CFB in the FACIT-Fatigue scale
Measured: 52 weeks
CFB in Other Clinical Biomarkers of PNH Disease Activity including absolute reticulocyte count, total PNH red blood cell clone size, haptoglobin levels, total bilirubin, and aspartate transaminase
Measured: Baseline, Week 52
Number of Participants with Clinical PNH Symptoms
Measured: up to 52 weeks
Concentration of BCX10013 and its Metabolite(s) in Plasma
Measured: Pre-dose, 0.5, 1, 2, 4, and 6 hours post dose on Day 1, Week 2, and Week 4
Concentration of BCX10013 and its Metabolite(s) in Urine (if applicable)
Measured: Pre-dose and all urine from 0 to 6 hours post dose on Day 1, Week 2, and Week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2023
- Study start24 Oct 2023
- Primary completion11 Dec 2024
- Study completion11 Dec 2024
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Multiple dose levels of BCX10013 are planned for escalation.
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06100900
- Sponsor's protocol ID
- BCX10013-105
- Status last verified
- Jan 2025
- First submitted
- 20 Oct 2023
- Last updated
- 8 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multicenter, Intra-Subject Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Therapeutic Potential of BCX10013 in Subjects with Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT06100900
- Acronym / sponsor ID
- BCX10013-105
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, Factor D, complement inhibitor, alternative pathway inhibitor, BioCryst
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Oct 2023 (actual)
- Primary completion
- 11 Dec 2024 (actual)
- Study completion
- 11 Dec 2024 (actual)
- First posted
- 25 Oct 2023 (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 multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH. Approximately 8 participants will be enrolled in this study. Participants may receive treatment for up to 52 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Multiple dose levels of BCX10013 are planned for escalation.
| Arm | Type | Receives |
|---|---|---|
| BCX10013 | Experimental | Drug: BCX10013Participants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or non-pregnant, non-lactating female adults ≥ 18 years old.
- Documented diagnosis of PNH confirmed by flow cytometry.
- Body mass index (BMI) ≤ 40 kg/m^2.
- Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with ravulizumab for at least 12 months prior to the screening visit and have received no treatment with eculizumab or pegcetacoplan for 6 months prior to the screening visit.
- Documentation of current vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae type B [Hib] or willingness to start vaccination series at least 14 days prior to Day 1.
Key exclusion criteria (7 listed)
- Known history of or existing diagnosis of hereditary complement deficiency.
- History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
- Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis.
- History of malignancy within 5 years prior to the screening visit.
- Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
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 |
|---|---|---|
| BCX10013Multiple dose levels may be tested in this study. | Drug | BCX10013 |
Primary outcome
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings.
Time frame: up to 52 weeks
9 secondary outcomes
CFB in Lactate Dehydrogenase
Time frame: Baseline, Week 52
CFB in the Ratio of Total PNH Red Blood Cell Clone Size to PNH White Blood Cell Clone Size
Time frame: Baseline, Week 52
CFB in Hemoglobin
Time frame: Baseline, Week 52
Percentage of Participants who are Transfusion-free
Time frame: 52 weeks
Percentage of Participants Achieving a Within-subject Clinically Meaningful CFB in the FACIT-Fatigue scale
Time frame: 52 weeks
CFB in Other Clinical Biomarkers of PNH Disease Activity including absolute reticulocyte count, total PNH red blood cell clone size, haptoglobin levels, total bilirubin, and aspartate transaminase
Time frame: Baseline, Week 52
Number of Participants with Clinical PNH Symptoms
Time frame: up to 52 weeks
Concentration of BCX10013 and its Metabolite(s) in Plasma
Time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post dose on Day 1, Week 2, and Week 4
Concentration of BCX10013 and its Metabolite(s) in Urine (if applicable)
Time frame: Pre-dose and all urine from 0 to 6 hours post dose on Day 1, Week 2, and Week 4
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 8 Jan 2025.
- Individual participant dataSharing planned: No
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
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A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
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Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
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Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
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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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BCX10013 in people with Paroxysmal Nocturnal Hemoglobinuria, run by BioCryst Pharmaceuticals at 4 sites in 2 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 5 inclusion and 7 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 8 Jan 2025. 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, Malaysia. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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?
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings. (time frame: up to 52 weeks). 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 8 Jan 2025.