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TerminatedNCT04702568Updated Apr 13, 2025Results posted

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

A Phase 2 interventional study of BCX9930 and Eculizumab in Paroxysmal Nocturnal Hemoglobinuria and PNH, sponsored by BioCryst Pharmaceuticals. Terminated at 5 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-04-13.

Sponsored by BioCryst Pharmaceuticals · Phase 2, Interventional, and Treatment

Why this study was terminated
Sponsor decision
Phase
Phase 2
Study type
Interventional
Enrollment
19
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

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.

02

Conditions studied

  • Paroxysmal Nocturnal Hemoglobinuria
  • PNH

Keywords

  • BCX9930
  • Factor D inhibitor
  • PNH
  • paroxysmal Nocturnal Hemoglobinuria
  • oral treatment
03

In context

Hemoglobinuria, Paroxysmal

191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.

This study's enrollment of 19 is below the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.

Browse Hemoglobinuria, Paroxysmal studies →

Lead sponsor

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.

Browse BioCryst Pharmaceuticals studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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.

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

  • Apart from a diagnosis of PNH, any 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

Terms in these criteria, in plain language(1)

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.

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.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
19 participants (actual)

Study arms

  • Experimental
    Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group

    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.

    Drug: BCX9930

  • Experimental
    C5 INH Inadequate Response Group

    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.

    Drug: BCX9930 · Drug: Eculizumab · Drug: Ravulizumab

Interventions

  • DrugBCX9930

    BCX9930 for oral administration

  • DrugEculizumab

    Administered at stable dose at the time of study entry

  • DrugRavulizumab

    Administered at stable dose at the time of study entry

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)

Secondary outcomes

  1. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  2. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  3. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  4. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  5. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  6. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  7. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  8. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  9. 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.

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  10. Change From Baseline in Lactate Dehydrogenase (LDH)

    Time frame: Baseline, Weeks, 24, 48, 72, 96, 120, and 144

  11. Change From Baseline in Hemoglobin

    Time frame: Baseline, Weeks 24, 48, 72, 96, 120, and 144

  12. Change From Baseline in Haptoglobin

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  13. Change From Baseline in Reticulocytes

    Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

  14. 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.

    Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)

  15. Number of Blood Transfusions

    Number of blood transfusions were reported.

    Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)

  16. 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.

    Time frame: Baseline, Weeks 24, 48, 72, and 96

07

Results

Posted Dec 13, 2024
Limitations and caveats
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\]).

Participant flow

Participants who had participated in previous BCX9930 trials (BCX9930-101 \[NCT04330534\], BCX9930-202 \[NCT05116774\], or BCX9930-203 \[NCT05116787\]) for Paroxysmal Nocturnal Hemoglobinuria (PNH) and showed a benefit of treatment as determined by the investigator were enrolled in this long-term safety trial.

Participant flow — Overall Study
MilestoneComplement Component 5 (C5) Inhibitor (C5-INH) Naïve GroupC5-INH Inadequate Response Group
Started127
Completed00
Not completed127
Withdrew: Transitioned to the bcx9930-205 roll-over study104
Withdrew: Pregnancy10
Withdrew: Bone marrow transplant10
Withdrew: Withdrawn due to investigator decision01
Withdrew: Withdrawal by subject02

Outcome measures

PrimaryNumber 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.

Time frame:
From first dose of study drug up to 3 weeks after last dose (Week 147)
Reported as:
Count of participants · Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Number of Participants With Treatment Emergent Adverse Events (TEAEs)127
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none84
Baseline-mild32
Baseline-moderate01
Baseline-severe10
Week 24-none103
Week 24-mild23
Week 24-moderate01
Week 24-severe00
Week 48-none62
Week 48-mild20
Week 48-moderate01
Week 48-severe00
Week 72-none71
Week 72-mild12
Week 72-moderate00
Week 72-severe00
Week 96-none62
Week 96-mild20
Week 96-moderate00
Week 96-severe01
Week 120-none82
Week 120-mild00
Week 120-moderate01
Week 120-severe00
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none116
Baseline-mild11
Baseline-moderate00
Baseline-severe00
Week 24-none117
Week 24-mild10
Week 24-moderate00
Week 24-severe00
Week 48-none62
Week 48-mild21
Week 48-moderate00
Week 48-severe00
Week 72-none72
Week 72-mild11
Week 72-moderate00
Week 72-severe00
Week 96-none72
Week 96-mild00
Week 96-moderate11
Week 96-severe00
Week 120-none82
Week 120-mild01
Week 120-moderate00
Week 120-severe00
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none127
Baseline-mild00
Baseline-moderate00
Baseline-severe00
Week 24-none127
Week 24-mild00
Week 24-moderate00
Week 24-severe00
Week 48-none72
Week 48-mild11
Week 48-moderate00
Week 48-severe00
Week 72-none72
Week 72-mild10
Week 72-moderate01
Week 72-severe00
Week 96-none73
Week 96-mild10
Week 96-moderate00
Week 96-severe00
Week 120-none83
Week 120-mild00
Week 120-moderate00
Week 120-severe00
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none116
Baseline-mild11
Baseline-moderate00
Baseline-severe00
Week 24-none127
Week 24-mild00
Week 24-moderate00
Week 24-severe00
Week 48-none62
Week 48-mild21
Week 48-moderate00
Week 48-severe00
Week 72-none72
Week 72-mild11
Week 72-moderate00
Week 72-severe00
Week 96-none72
Week 96-mild11
Week 96-moderate00
Week 96-severe00
Week 120-none83
Week 120-mild00
Week 120-moderate00
Week 120-severe00
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none107
Baseline-mild10
Baseline-moderate10
Baseline-severe00
Week 24-none96
Week 24-mild20
Week 24-moderate11
Week 24-severe00
Week 48-none62
Week 48-mild11
Week 48-moderate00
Week 48-severe10
Week 72-none72
Week 72-mild11
Week 72-moderate00
Week 72-severe00
Week 96-none72
Week 96-mild11
Week 96-moderate00
Week 96-severe00
Week 120-none83
Week 120-mild00
Week 120-moderate00
Week 120-severe00
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Headache
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none107
Baseline-mild20
Baseline-moderate00
Baseline-severe00
Week 24-none116
Week 24-mild11
Week 24-moderate00
Week 24-severe00
Week 48-none72
Week 48-mild00
Week 48-moderate11
Week 48-severe00
Week 72-none83
Week 72-mild00
Week 72-moderate00
Week 72-severe00
Week 96-none73
Week 96-mild10
Week 96-moderate00
Week 96-severe00
Week 120-none82
Week 120-mild01
Week 120-moderate00
Week 120-severe00
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none92
Baseline-mild00
Baseline-moderate00
Baseline-severe00
Week 24-none92
Week 24-mild00
Week 24-moderate00
Week 24-severe00
Week 48-none71
Week 48-mild10
Week 48-moderate00
Week 48-severe00
Week 72-none71
Week 72-mild10
Week 72-moderate00
Week 72-severe00
Week 96-none72
Week 96-mild10
Week 96-moderate00
Week 96-severe00
Week 120-none71
Week 120-mild10
Week 120-moderate00
Week 120-severe00
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none107
Baseline-mild00
Baseline-moderate00
Baseline-severe00
Baseline-missing severity20
Week 24-none115
Week 24-mild00
Week 24-moderate00
Week 24-severe00
Week 24-missing severity12
Week 48-none63
Week 48-mild00
Week 48-moderate10
Week 48-severe00
Week 48-missing severity10
Week 72-none72
Week 72-mild01
Week 72-moderate00
Week 72-severe00
Week 72-missing severity10
Week 96-none63
Week 96-mild00
Week 96-moderate00
Week 96-severe00
Week 96-missing severity20
Week 120-none73
Week 120-mild00
Week 120-moderate00
Week 120-severe00
Week 120-missing severity10
SecondaryNumber 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.

Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Count of participants · Participants
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline-none117
Baseline-mild00
Baseline-moderate00
Baseline-severe00
Baseline-missing severity10
Week 24-none127
Week 24-mild00
Week 24-moderate00
Week 24-severe00
Week 48-none83
Week 48-mild00
Week 48-moderate00
Week 48-severe00
Week 72-none83
Week 72-mild00
Week 72-moderate00
Week 72-severe00
Week 96-none83
Week 96-mild00
Week 96-moderate00
Week 96-severe00
Week 120-none83
Week 120-mild00
Week 120-moderate00
Week 120-severe00
SecondaryChange From Baseline in Lactate Dehydrogenase (LDH)
Time frame:
Baseline, Weeks, 24, 48, 72, 96, 120, and 144
Reported as:
Mean · Units per liter (U/L)
Change From Baseline in Lactate Dehydrogenase (LDH)
Units per liter (U/L)C5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline475.6 ± 210.98248.3 ± 125.71
Change at Week 24-46.7 ± 154.5538.6 ± 45.51
Change at Week 48-62.5 ± 80.9738.5 ± 65.76
Change at Week 72-6.1 ± 238.41163.5 ± 118.09
Change at Week 96237.4 ± 347.65-4.0 ± 134.35
Change at Week 120195.4 ± 355.0331.0 ± 125.87
Change at Week 144-59.0—
SecondaryChange From Baseline in Hemoglobin
Time frame:
Baseline, Weeks 24, 48, 72, 96, 120, and 144
Reported as:
Mean · grams per deciliter (g/dl)
Change From Baseline in Hemoglobin
grams per deciliter (g/dl)C5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline11.88 ± 1.68811.53 ± 1.803
Change at Week 240.75 ± 1.159-1.32 ± 2.578
Change at Week 48-0.61 ± 1.974-0.95 ± 2.333
Change at Week 72-0.64 ± 2.327-0.93 ± 1.484
Change at Week 96-0.33 ± 1.914-0.70 ± 2.687
Change at Week 1200.38 ± 1.555-0.47 ± 2.098
Change at Week 144-2.70—
SecondaryChange From Baseline in Haptoglobin
Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Mean · grams per liter (g/L)
Change From Baseline in Haptoglobin
grams per liter (g/L)C5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline0.325 ± 0.28320.216 ± 0.1350
Change at Week 240.000 ± 0.0447-0.082 ± 0.0844
Change at Week 480.213 ± 0.1126—
Change at Week 720.025 ± 0.1165-0.210 ± 0.1414
Change at Week 96-0.013 ± 0.03540.195 ± 0.4313
Change at Week 120-0.013 ± 0.0354-0.100
SecondaryChange From Baseline in Reticulocytes
Time frame:
Baseline, Weeks 24, 48, 72, 96, and 120
Reported as:
Mean · 10^6 cells per microliter (μL)
Change From Baseline in Reticulocytes
10^6 cells per microliter (μL)C5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline0.1066 ± 0.023630.0889 ± 0.03939
Change at Week 240.0164 ± 0.023390.0913 ± 0.09144
Change at Week 480.0048 ± 0.035180.0507 ± 0.03161
Change at Week 720.0044 ± 0.036430.0463 ± 0.03008
Change at Week 960.0409 ± 0.038050.0607 ± 0.03585
Change at Week 1200.0173 ± 0.033030.0653 ± 0.02380
SecondaryNumber 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.

Time frame:
From first dose of study drug up to 3 weeks after last dose (Week 147)
Reported as:
Count of participants · Participants
Number of Participants With Blood Transfusions or Thromboses
ParticipantsC5-INH Naïve GroupC5-INH Inadequate Response Group
Blood transfusions34
Thromboses00
SecondaryNumber of Blood Transfusions

Number of blood transfusions were reported.

Time frame:
From first dose of study drug up to 3 weeks after last dose (Week 147)
Reported as:
Number · Number of blood transfusions
Number of Blood Transfusions
Number of blood transfusionsC5-INH Naïve GroupC5-INH Inadequate Response Group
Number of Blood Transfusions1325
SecondaryChange 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.

Time frame:
Baseline, Weeks 24, 48, 72, and 96
Reported as:
Mean · Scores on a scale
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score
Scores on a scaleC5-INH Naïve GroupC5-INH Inadequate Response Group
Baseline43.82 ± 9.40040.14 ± 13.533
Change at Week 240.09 ± 4.110-3.36 ± 8.066
Change at Week 48-0.63 ± 2.3872.33 ± 3.215
Change at Week 72-0.38 ± 2.875-4.64 ± 12.016
Change at Week 96-1.13 ± 4.357-18.00 ± 27.185

Adverse events

Collected over From first dose of study drug up to 3 weeks after last dose (Week 147). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group0/12 (0%)6/12 (50%)12/12 (100%)
C5-INH Inadequate Response Group0/7 (0%)3/7 (42.9%)7/7 (100%)
Most frequent serious events
Showing 10 of 12
Most frequent serious events
EventComplement Component 5 (C5) Inhibitor (C5-INH) Naïve GroupC5-INH Inadequate Response Group
Breakthrough haemolysisBlood and lymphatic system disorders3/120/7
HaemolysisBlood and lymphatic system disorders1/121/7
PyelonephritisInfections and infestations0/121/7
SepsisInfections and infestations0/121/7
Crohn's diseaseGastrointestinal disorders0/121/7
PyrexiaGeneral disorders0/121/7
Epstein-Barr virus associated lymphomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/121/7
Febrile neutropeniaBlood and lymphatic system disorders1/120/7
Boutonneuse feverInfections and infestations1/120/7
Soft tissue infectionInfections and infestations1/120/7
Most frequent other events
Showing 10 of 123
Most frequent other events
EventComplement Component 5 (C5) Inhibitor (C5-INH) Naïve GroupC5-INH Inadequate Response Group
Breakthrough haemolysisBlood and lymphatic system disorders4/124/7
HeadacheNervous system disorders4/124/7
HaemoglobinuriaRenal and urinary disorders1/124/7
NasopharyngitisInfections and infestations2/123/7
Back painMusculoskeletal and connective tissue disorders3/123/7
DyspnoeaRespiratory, thoracic and mediastinal disorders1/123/7
Oropharyngeal painRespiratory, thoracic and mediastinal disorders0/123/7
FatigueGeneral disorders0/123/7
Blood creatinine increasedInvestigations5/120/7
NauseaGastrointestinal disorders4/121/7

Baseline characteristics

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

Age, Continuous
Age, Continuous(years)C5-INH Naïve GroupC5-INH Inadequate Response GroupTotal
Mean29.3 ± 7.8244.6 ± 18.5934.9 ± 14.50
Sex: Female, Male
Sex: Female, Male(Participants)C5-INH Naïve GroupC5-INH Inadequate Response GroupTotal
Female358
Male9211
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)C5-INH Naïve GroupC5-INH Inadequate Response GroupTotal
Hispanic or Latino000
Not Hispanic or Latino12719
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)C5-INH Naïve GroupC5-INH Inadequate Response GroupTotal
American Indian or Alaska Native000
Asian112
Native Hawaiian or Other Pacific Islander000
Black or African American729
White347
More than one race000
Unknown or Not Reported101
08

Study locations

5 sites
  • Study Center
    Vienna, Austria
  • Study Center
    Bloemfontein, South Africa
  • Study Center
    Cape Town, South Africa
  • Study Center
    Pretoria, South Africa
  • Study Center
    London, United Kingdom
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Dec 2020 to primary completion in Oct 2023, 33 months.

Results were first posted in Dec 2024, 1 year 2 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 7, 2021
First posted
Jan 11, 2021
StartActual
Dec 18, 2020
Primary completionActual
Oct 4, 2023
CompletionActual
Oct 4, 2023
Results first posted
Dec 13, 2024
Last update
Apr 13, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Apr 13, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04702568
Lead sponsor
BioCryst Pharmaceuticals
Responsible party
Sponsor
First posted
Jan 11, 2021
Start date
Dec 18, 2020
Primary completion
Oct 4, 2023
Completion
Oct 4, 2023
Results posted
Dec 13, 2024
Last update
Apr 13, 2025

Study contacts

Morag Griffin, MBChB
principal investigator · Leeds Teaching Hospitals NHS Trust, Leeds, UK

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.

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Discussion

Questions 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.

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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

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