Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT04702568

A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 2 study testing BCX9930, Eculizumab and Ravulizumab in people with Paroxysmal Nocturnal Hemoglobinuria and PNH, run by BioCryst Pharmaceuticals at 5 sites in 3 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Paroxysmal Nocturnal Hemoglobinuria, PNH
What is being tested?: BCX9930, Eculizumab and Ravulizumab
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 19 participants
Where: 5 sitesin 3 countries
Study dates: 18 Dec 2020to 4 Oct 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 participants
Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH

Who cannot join

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 participant's ability to participate in the study or participation would increase the risk for that participant
Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating
Note: Other protocol-defined Inclusion/Exclusion criteria may apply

Trial locations and contacts

5 sites in 3 countries.

Country

City, postcode or hospital

5 sites in 3 countries

South Africa3 sites

Study Center
Bloemfontein
Study Center
Cape Town
Study Center
Pretoria

Other trials in South Africa

Austria1 site

Study Center
Vienna

Other trials in Austria

United Kingdom1 site

Study Center
London

Other trials in United Kingdom

About this study

Official title: A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This study was designed to evaluate the long-term safety of daily oral treatment with BCX9930 in participants who had participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allowed continued access to BCX9930 for enrolled participants. The study also evaluated the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the participant's satisfaction with the medication.

Conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH
Being tested: BCX9930, Eculizumab, Ravulizumab
Keywords: BCX9930, Factor D inhibitor, PNH, paroxysmal Nocturnal Hemoglobinuria, oral treatment
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 for oral administration
EculizumabDrug
Administered at stable dose at the time of study entry
RavulizumabDrug
Administered at stable dose at the time of study entry

Study groups in NCT04702568
Study group	Role	What they receive
Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group	Experimental	This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Receives: Drug: BCX9930
C5 INH Inadequate Response Group	Experimental	This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Receives: Drug: BCX9930; Drug: Eculizumab; Drug: Ravulizumab

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)
An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.
Measured: From first dose of study drug up to 3 weeks after last dose (Week 147)

Secondary outcomes (16)

Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue
Data was reported for number of participants with clinical PNH symptom of fatigue. The severity of clinical PNH symptom of fatigue was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea
Data was reported for number of participants with clinical PNH symptom of Dyspnea. The severity of clinical PNH symptom of Dyspnea was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort
Data was reported for number of participants with clinical PNH symptom of Chest pain/discomfort. The severity of clinical PNH symptom of chest pain/discomfort was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing
Data was reported for number of participants with clinical PNH symptom of difficulty swallowing. The severity of clinical PNH symptom of difficulty swallowing was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain
Data was reported for number of participants with clinical PNH symptom of abdominal pain. The severity of clinical PNH symptom of abdominal pain was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Headache
Data was reported for number of participants with clinical PNH symptom of headache. The severity of clinical PNH symptom of headache was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction
Data was reported for number of participants with clinical PNH symptom of erectile dysfunction. The severity of clinical PNH symptom of erectile dysfunction was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria
Data was reported for number of participants with clinical PNH symptom of Hemoglobinuria.The severity of clinical PNH symptom of hemoglobinuria was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice
Data was reported for number of participants with clinical PNH symptom of jaundice. The severity of clinical PNH symptom of jaundice was graded as none, mild, moderate and severe based solely on investigator's discretion.
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Change From Baseline in Lactate Dehydrogenase (LDH)
Measured: Baseline, Weeks, 24, 48, 72, 96, 120, and 144
Change From Baseline in Hemoglobin
Measured: Baseline, Weeks 24, 48, 72, 96, 120, and 144
Change From Baseline in Haptoglobin
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Change From Baseline in Reticulocytes
Measured: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Blood Transfusions or Thromboses
Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events.
Measured: From first dose of study drug up to 3 weeks after last dose (Week 147)
Number of Blood Transfusions
Number of blood transfusions were reported.
Measured: From first dose of study drug up to 3 weeks after last dose (Week 147)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score
The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.
Measured: Baseline, Weeks 24, 48, 72, and 96

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 Dec 20204 Oct 2023

First posted11 Jan 2021
Study start18 Dec 2020
Primary completion4 Oct 2023
Study completion4 Oct 2023

Why it stopped: Sponsor decision

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 19 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04702568
Sponsor's protocol ID: BCX9930-201
Other IDs: 2020-000501-93
Status last verified: Apr 2025
First submitted: 7 Jan 2021
Last updated: 13 Apr 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
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
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)

All Paroxysmal Nocturnal Hemoglobinuria trials

Study at a glance

From the registry record, last updated by the sponsor on 13 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
ClinicalTrials.gov ID: NCT04702568
Acronym / sponsor ID: BCX9930-201
Conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH
Keywords: BCX9930, Factor D inhibitor, PNH, paroxysmal Nocturnal Hemoglobinuria, oral treatment
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Sponsor decision
Enrolment: 19 participants (actual)
Lead sponsor: BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 18 Dec 2020 (actual)
Primary completion: 4 Oct 2023 (actual)
Study completion: 4 Oct 2023 (actual)
First posted: 11 Jan 2021 (actual)
Results: Posted 13 Dec 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group	Experimental	Drug: BCX9930This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis.
C5 INH Inadequate Response Group	Experimental	Drug: BCX9930, Drug: Eculizumab, Drug: RavulizumabThis group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (2 listed)

Male or non-pregnant, non-lactating female participants
Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH

Key exclusion criteria (3 listed)

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 participant's ability to participate in the study or participation would increase the risk for that participant
Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating
Note: Other protocol-defined Inclusion/Exclusion criteria may apply

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 for oral administration	Drug	C5 INH Inadequate Response Group, Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group
EculizumabAdministered at stable dose at the time of study entry	Drug	C5 INH Inadequate Response Group
RavulizumabAdministered at stable dose at the time of study entry	Drug	C5 INH Inadequate Response Group

Primary outcome

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)
An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.

16 secondary outcomes

Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Headache
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Change From Baseline in Lactate Dehydrogenase (LDH)
Time frame: Baseline, Weeks, 24, 48, 72, 96, 120, and 144
Change From Baseline in Hemoglobin
Time frame: Baseline, Weeks 24, 48, 72, 96, 120, and 144
Change From Baseline in Haptoglobin
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Change From Baseline in Reticulocytes
Time frame: Baseline, Weeks 24, 48, 72, 96, and 120
Number of Participants With Blood Transfusions or Thromboses
Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)
Number of Blood Transfusions
Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score
Time frame: Baseline, Weeks 24, 48, 72, and 96

Results and safety

Results posted to the registry on 13 Dec 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group	12	0
C5-INH Inadequate Response Group	7	0

Primary outcome: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147). Count of participants. Unit: Participants

Measure	C5-INH Naïve Group n = 12	C5-INH Inadequate Response Group n = 7
Value	12	7

Population analysed: The safety population included all participants who received at least 1 capsule or tablet of study drug.

16 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group	6 of 12 (50%)	12 of 12 (100%)	0 of 12
C5-INH Inadequate Response Group	3 of 7 (42.9%)	7 of 7 (100%)	0 of 7

Limitations and caveats (from the sponsor): The sponsor decided to prematurely terminate the study due to business reasons. Per change in planned analysis, data were analyzed and reported for safety and selected efficacy parameters (assessment of various clinical PNH symptoms severity, incidence of thromboembolic events, change from baseline in clinical measurements of PNH \[LDH, hemoglobin, haptoglobin, reticulocytes, transfusion requirements\], and change from baseline in FACIT\]).

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Paroxysmal Nocturnal Hemoglobinuria

Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
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
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)

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.

Morag Griffin, MBChB principal investigator

Leeds Teaching Hospitals NHS Trust, Leeds, UK

5 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BCX9930, Eculizumab and Ravulizumab in people with Paroxysmal Nocturnal Hemoglobinuria and PNH, run by BioCryst Pharmaceuticals at 5 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 2 inclusion and 3 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: stopped early, as of the sponsor's last update on 13 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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. 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 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?

Yes. Results are posted in the registry (first posted 13 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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) (time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04702568.

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 13 Apr 2025.