A Phase 2 interventional study of Eculizumab and Ravulizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by BioCryst Pharmaceuticals. Terminated at 7 sites in 5 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-11-10.
Sponsored by BioCryst Pharmaceuticals · Phase 2, Interventional, and Treatment
The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH participants with residual anemia despite treatment with a C5 inhibitor.
This was a randomized, active comparator-controlled, open-label, parallel-group, 2-part study. Parts 1 and 2 were conducted in the same participants.
Part 1 of the study was designed to evaluate the efficacy, safety, and tolerability of oral BCX9930 monotherapy for 24 weeks versus continuing C5 inhibitor therapy in participants with PNH with inadequate response to their current C5 inhibitor therapy. Participants were randomized to either discontinue C5 inhibitor therapy and start BCX9930 monotherapy or to continue C5 inhibitor therapy for the 24-week randomized treatment period. The primary efficacy and safety analyses were based on Part 1.
Part 2 of the study was designed to evaluate the long-term safety, tolerability, and effectiveness of BCX9930 monotherapy when administered through Week 52. All participants in Part 2 received BCX9930. Participants who were randomized to BCX9930 monotherapy in Part 1 continued to receive BCX9930 in Part 2. Participants who were randomized to C5 inhibitor therapy in Part 1 discontinued that therapy at the Week 24 visit and received BCX9930 in Part 2.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's enrollment of 12 is below the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →BioCryst Pharmaceuticals is the lead sponsor of 54 studies on the registry; 1 is open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 8 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.
Drug: BCX9930
Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
Drug: Eculizumab · Drug: Ravulizumab · Drug: BCX9930
Administered by intravenous infusion per current dose regimen
Also known as: Soliris
Administered by intravenous infusion per current dose regimen
Also known as: Ultomiris, ALXN1210, ravulizumab-cwvz
Administered orally twice daily
Part 1: Change From Baseline in Hemoglobin at Week 24
Time frame: Baseline, Week 24
Part 1: Number of Participants Who Were Transfusion-free
The number of participants who did not receive any transfusions (packed red blood cells \[pRBCs\] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.
Time frame: From Week 4 to Week 24
Part 1: Number of Units of pRBCs Transfused
Time frame: From Week 4 to Week 24
Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale 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.
Time frame: Baseline, Week 24
Part 1: Percent Change From Baseline in Lactate Dehydrogenase
Time frame: Baseline, Week 24
Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused
The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate \* 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions.
Time frame: Prestudy (12 months prior to screening) and from Week 4 to Week 24
Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL)
Time frame: Week 24
Part 1: Number of Participants Who Achieved Hemoglobin Stabilization
Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24.
Time frame: From Week 4 to Week 24
Part 1: Change From Baseline in Complement Component 3 (C3)-Opsonized Red Blood Cells (RBCs)
Red blood cells opsonized by C3 were to be assessed by flow cytometry .
Time frame: Baseline, Week 24
Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size
The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population.
Time frame: Baseline, Week 24
Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size
The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs.
Time frame: Baseline, Week 24
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)
Time frame: Baseline, Week 24
Part 1: Number of Participants With ARC in the Normal Range
Number of participants with ARC in the normal range (0.03 - 0.12 10\^6 cells/uL) were reported.
Time frame: Week 24
Part 1: Change From Baseline in Haptoglobin
Time frame: Baseline, Week 24
Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range
Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.
Time frame: Week 24
Part 1: Change From Baseline in Total Bilirubin
Time frame: Baseline, Week 24
A total 12 participants were randomized and treated.
| Milestone | BCX9930/BCX9930 | C5-Inhibitor (C5-INH)/BCX9930 |
|---|---|---|
| Started | 8 | 4 |
| Completed | 7 | 3 |
| Not completed | 1 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
| Withdrew: Adverse event | 1 | 0 |
| Milestone | BCX9930/BCX9930 | C5-Inhibitor (C5-INH)/BCX9930 |
|---|---|---|
| Started | 7 | 3 |
| Completed | 3 | 2 |
| Not completed | 4 | 1 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Miscellaneous | 3 | 1 |
| grams per deciliter (g/dL) | BCX9930 | C5-INH |
|---|---|---|
| Baseline | 9.12 ± 1.096 | 9.13 ± 0.847 |
| Change at Week 24 | 3.48 ± 0.674 | 0.72 ± 0.884 |
The number of participants who did not receive any transfusions (packed red blood cells \[pRBCs\] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.
| Participants | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Number of Participants Who Were Transfusion-free | 7 | 4 |
| units of pRBCs | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Number of Units of pRBCs Transfused | 0 | 0 |
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.
| Scores on a scale | BCX9930 | C5-INH |
|---|---|---|
| Baseline | 36.3 ± 9.69 | 40.0 ± 8.49 |
| Change at Week 24 | -0.3 ± 12.86 | 1.0 |
| percent change | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Percent Change From Baseline in Lactate Dehydrogenase | 24.3 ± 53.07 | -21.0 ± 4.03 |
The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate \* 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions.
| percent reduction | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | 100 | NA |
| Participants | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL) | 6 | 0 |
Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24.
| Participants | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Number of Participants Who Achieved Hemoglobin Stabilization | 5 | 4 |
Red blood cells opsonized by C3 were to be assessed by flow cytometry .
No measurements were reported for this outcome.
The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population.
| % of PNH-RBC within total RBC population | BCX9930 | C5-INH |
|---|---|---|
| Baseline | 80.51 ± 16.94 | 83.40 ± 14.34 |
| Change at Week 24 | 23.18 ± 11.81 | -6.34 ± 8.986 |
The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs.
| ratio | BCX9930 | C5-INH |
|---|---|---|
| Baseline | 0.8128 ± 0.16528 | 0.9059 ± 0.12113 |
| Change at Week 24 | 0.2336 ± 0.11595 | -0.0668 ± 0.09462 |
| 10^6 cells/microliter (μL) | BCX9930 | C5-INH |
|---|---|---|
| Baseline | 0.2933 ± 0.10453 | 0.3129 ± 0.16201 |
| Change at Week 24 | -0.2142 ± 0.05384 | -0.0462 ± 0.01823 |
Number of participants with ARC in the normal range (0.03 - 0.12 10\^6 cells/uL) were reported.
| Participants | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Number of Participants With ARC in the Normal Range | 4 | 1 |
| grams per liter (g/L) | BCX9930 | C5-INH |
|---|---|---|
| Baseline | 0.126 ± 0.0732 | 0.105 ± 0.0268 |
| Change at Week 24 | -0.024 ± 0.0409 | -0.015 ± 0.0268 |
Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.
| Participants | BCX9930 | C5-INH |
|---|---|---|
| Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | 0 | 0 |
| milligram per deciliter (mg/dL) | BCX9930 | C5-INH |
|---|---|---|
| Baseline | 3.44 ± 3.679 | 1.10 ± 0.424 |
| Change at Week 24 | -2.66 ± 3.672 | -0.02 ± 0.250 |
Collected over Baseline up to Day 407. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BCX9930/BCX9930 | 0/8 (0%) | 2/8 (25%) | 7/8 (87.5%) |
| C5-INH | 0/4 (0%) | 0/4 (0%) | 3/4 (75%) |
| BCX9930 After C5-INH | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Event | BCX9930/BCX9930 | C5-INH | BCX9930 After C5-INH |
|---|---|---|---|
| Rash erythematousSkin and subcutaneous tissue disorders | 0/8 | 0/4 | 1/3 |
| Blood creatinine increasedInvestigations | 1/8 | 0/4 | 0/3 |
| Acute kidney injuryRenal and urinary disorders | 1/8 | 0/4 | 0/3 |
| Event | BCX9930/BCX9930 | C5-INH | BCX9930 After C5-INH |
|---|---|---|---|
| AstheniaGeneral disorders | 1/8 | 3/4 | 0/3 |
| Back painMusculoskeletal and connective tissue disorders | 2/8 | 2/4 | 0/3 |
| HeadacheNervous system disorders | 4/8 | 0/4 | 0/3 |
| Dyspnoea exertionalRespiratory, thoracic and mediastinal disorders | 0/8 | 2/4 | 0/3 |
| DysphagiaGastrointestinal disorders | 3/8 | 0/4 | 0/3 |
| NauseaGastrointestinal disorders | 3/8 | 0/4 | 0/3 |
| FatigueGeneral disorders | 3/8 | 0/4 | 0/3 |
| RashSkin and subcutaneous tissue disorders | 3/8 | 0/4 | 0/3 |
| Urinary tract infectionInfections and infestations | 1/8 | 0/4 | 1/3 |
| Localised infectionInfections and infestations | 0/8 | 0/4 | 1/3 |
The safety population included all participants who received at least 1 dose of study drug, whether C5 inhibitor or BCX9930.
| Age, Continuous(years) | BCX9930/BCX9930 | C5-INH/BCX9930 | Total |
|---|---|---|---|
| Mean | 58.8 ± 17.66 | 48.5 ± 14.25 | 55.3 ± 16.71 |
| Sex: Female, Male(Participants) | BCX9930/BCX9930 | C5-INH/BCX9930 | Total |
|---|---|---|---|
| Female | 4 | 3 | 7 |
| Male | 4 | 1 | 5 |
| Ethnicity (NIH/OMB)(Participants) | BCX9930/BCX9930 | C5-INH/BCX9930 | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 5 | 2 | 7 |
| Unknown or Not Reported | 3 | 2 | 5 |
| Race (NIH/OMB)(Participants) | BCX9930/BCX9930 | C5-INH/BCX9930 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 1 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 2 | 0 | 2 |
| White | 5 | 2 | 7 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 1 | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Pelecopan