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BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy
Phase 2 trial testing Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH) in 12 participants. Terminated before completion.
| Lead sponsor | BioCryst Pharmaceuticals |
|---|---|
| Phase | Phase 2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 12 |
| Start date | 6 December 2021 |
| Primary completion | 14 September 2023 |
| Estimated completion | 14 September 2023 |
| Sites | 7 locations across France, Italy, United Kingdom, Hungary, Spain |
BioCryst Pharmaceuticals — full company profile →
18 and older, any sex, with Paroxysmal Nocturnal Hemoglobinuria (PNH). Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 9.12 | ± 1.096 |
| C5-INH | 9.13 | ± 0.847 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 3.48 | ± 0.674 |
| C5-INH | 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
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 7 | |
| C5-INH | 4 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 0 | |
| C5-INH | 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.
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 36.3 | ± 9.69 |
| C5-INH | 40.0 | ± 8.49 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | -0.3 | ± 12.86 |
| C5-INH | 1.0 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 24.3 | ± 53.07 |
| C5-INH | -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.
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 100 | |
| C5-INH | NA |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 6 | |
| C5-INH | 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.
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 5 | |
| C5-INH | 4 |
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.
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 80.51 | ± 16.94 |
| C5-INH | 83.40 | ± 14.34 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 23.18 | ± 11.81 |
| C5-INH | -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.
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 0.8128 | ± 0.16528 |
| C5-INH | 0.9059 | ± 0.12113 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 0.2336 | ± 0.11595 |
| C5-INH | -0.0668 | ± 0.09462 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 0.2933 | ± 0.10453 |
| C5-INH | 0.3129 | ± 0.16201 |
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | -0.2142 | ± 0.05384 |
| C5-INH | -0.0462 | ± 0.01823 |
Number of participants with ARC in the normal range (0.03 - 0.12 10\^6 cells/uL) were reported.
| Group | Value | 95% CI |
|---|---|---|
| BCX9930 | 4 | |
| C5-INH | 1 |
Time frame: Baseline up to Day 407. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | BCX9930/BCX9930 | C5-INH | BCX9930 After C5-INH |
|---|---|---|---|---|
| Blood creatinine increased | Investigations | — | — | — |
| Acute kidney injury | Renal and urinary disorders | — | — | — |
| Rash erythematous | Skin and subcutaneous tissue disorders | — | — | — |
| Reaction | System | BCX9930/BCX9930 | C5-INH | BCX9930 After C5-INH |
|---|---|---|---|---|
| Headache | Nervous system disorders | — | — | — |
| Dysphagia | Gastrointestinal disorders | — | — | — |
| Nausea | Gastrointestinal disorders | — | — | — |
| Asthenia | General disorders | — | — | — |
| Fatigue | General disorders | — | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — | — |
| Nasopharyngitis | Infections and infestations | — | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — | — | — |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — | — |
| Abdominal pain upper | Gastrointestinal disorders | — | — | — |
| Inguinal hernia | Gastrointestinal disorders | — | — | — |
| Vomiting | Gastrointestinal disorders | — | — | — |
| Chest pain | General disorders | — | — | — |
| Face oedema | General disorders | — | — | — |
| Pain | General disorders | — | — | — |
| Pyrexia | General disorders | — | — | — |
| Urinary tract infection | Infections and infestations | — | — | — |
| Bronchitis | Infections and infestations | — | — | — |
| COVID-19 | Infections and infestations | — | — | — |
| Influenza | Infections and infestations | — | — | — |
| Localised infection | Infections and infestations | — | — | — |
| Respiratory tract infection | Infections and infestations | — | — | — |
| Subcutaneous abscess | Infections and infestations | — | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — | — |
| Viral infection | Infections and infestations | — | — | — |
| Rash macular | Skin and subcutaneous tissue disorders | — | — | — |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | — | — | — |
| Rash erythematous | Skin and subcutaneous tissue disorders | — | — | — |
| Axillary mass | Musculoskeletal and connective tissue disorders | — | — | — |
| Bone pain | Musculoskeletal and connective tissue disorders | — | — | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — | — | — |
| Lethargy | Nervous system disorders | — | — | — |
| Loss of consciousness | Nervous system disorders | — | — | — |
| Nervous system disorder | Nervous system disorders | — | — | — |
| Sciatica | Nervous system disorders | — | — | — |
| Acute kidney injury | Renal and urinary disorders | — | — | — |
| Chromaturia | Renal and urinary disorders | — | — | — |
Most-reported serious reactions: Blood creatinine increased, Acute kidney injury, Rash erythematous.
Data from ClinicalTrials.gov NCT05116774 adverse events section.
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.
7 peer-reviewed publications reference this trial (live from Europe PMC):
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