Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
BCX9930, Factor D inhibitor, Proximal complement inhibitor, Oral therapy, Paroxysmal nocturnal hemoglobinuria
Brief summary
This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the opinion of the investigator, would benefit from continued treatment with BCX9930; who did not have access to other effective treatment options; and to monitor the safety of BCX9930 in participants continuing to receive BCX9930 for the treatment of PNH.
Detailed description
This was an open-label, non-randomized study to evaluate the long-term safety of BCX9930 in participants with PNH. PNH is an acquired, rare, serious, and potentially life-threatening disorder characterized by destruction of red blood cells (RBCs) resulting from uncontrolled activity of complement. The participants in this study had previously benefited from BCX9930 in BioCryst studies BCX9930-201, BCX9930-202, and BCX9930-203. As the development program was not being discontinued for safety reasons or due to a lack of efficacy, and the preliminary data available from the ongoing clinical studies in PNH did not change the current safety or efficacy profile for BCX9930, BioCryst remained committed to providing BCX9930 to those participants who had participated in the ongoing studies who were currently receiving benefit from BCX9930 and wished to continue treatment. This study was used to meet that commitment by allowing for continued access to treatment with BCX9930 for any clinical study participant with PNH currently receiving treatment with BCX9930. As the development of BCX9930 was terminated, treatment was provided for a maximum of 96 weeks, as long as the investigator believed it was in the participant's best interest to continue treatment, or until the participant had access to alternative therapy for PNH, whichever came first.
Interventions
DRUGBCX9930
Taken orally at 400 mg BID
Sponsors
BioCryst Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or non-pregnant, non-lactating female participants * Were receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, had benefited from treatment with BCX9930 and would have benefited from continued treatment with BCX9930, and who did not have access to other treatment options
Exclusion criteria
* Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would have interfered with the participant's ability to participate in the study or increased the risk of participation for that participant * An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromised the ability of the participant to continue study-specific procedures or it was considered not to be in the participant's best interest to continue, or benefit-risk assessment was no longer in favor of the participant's continued treatment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Events (TEAEs) | From Day 1 up to 30 days after last dose (up to approximately 100 weeks) | An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study intervention or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important event. An AE is considered treatment emergent if its start date was on or after the date of first dose of study treatment in Study 205 or if the AE was ongoing from the prior study. TEAEs included both serious TEAEs and non-serious TEAEs. |
Countries
France, Hungary, Malaysia, South Africa, South Korea, Spain, United Kingdom
Participant flow
Recruitment details
The study was conducted in France, Hungary, Malaysia, South Africa, South Korea, Spain, and the United Kingdom.
Pre-assignment details
A total of 28 participants were enrolled. The participants were enrolled from previous studies BCX9930-201 (2020-000501-93), BCX9930-202 (2020-004438-39), or BCX9930-203 (2020-004403-14).
Participants by arm
| Arm | Count |
|---|---|
| BCX9930 Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg BID for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement. | 28 |
| Total | 28 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawn due to termination of study | 28 |
Baseline characteristics
| Characteristic | BCX9930 |
|---|---|
| Age, Continuous | 41.1 years STANDARD_DEVIATION 14.51 |
| Race/Ethnicity, Customized Ethnicity: Hispanic or Latino | 0 Participants |
| Race/Ethnicity, Customized Ethnicity: Not Hispanic or Latino | 25 Participants |
| Race/Ethnicity, Customized Ethnicity: Not Reported | 1 Participants |
| Race/Ethnicity, Customized Ethnicity: Unknown | 2 Participants |
| Race/Ethnicity, Customized Race: American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Race: Asian | 5 Participants |
| Race/Ethnicity, Customized Race: Black or African American | 13 Participants |
| Race/Ethnicity, Customized Race: Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized Race: Other | 1 Participants |
| Race/Ethnicity, Customized Race: White | 9 Participants |
| Sex: Female, Male Female | 13 Participants |
| Sex: Female, Male Male | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 28 |
| other Total, other adverse events | 27 / 28 |
| serious Total, serious adverse events | 16 / 28 |
Outcome results
Primary
Number of Participants With Treatment-emergent Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study intervention or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important event. An AE is considered treatment emergent if its start date was on or after the date of first dose of study treatment in Study 205 or if the AE was ongoing from the prior study. TEAEs included both serious TEAEs and non-serious TEAEs.
Time frame: From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Population: Safety Population was defined as all participants who received at least 1 dose.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Number of Participants With Treatment-emergent Events (TEAEs) | 27 Participants |