Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Sponsors
BioCryst Pharmaceuticals Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Able to provide written informed consent. 2. Non-pregnant, non-lactating female subjects. 3. Subjects with PNH who are participating in another BCX9930 clinical study, have completed at lest 12 weeks treatment with BCX9930, and, in the opinion of the investigator, have benefitted from treatment with BCX9930 and would benefit from continued treatment with BCX9930, wish to continue treatment, and do not have access to other effective treatment options. 4. Female participants will continue to meet at least one of the following requirements per the prior study: a. Be a woman of nonchildbearing potential. b. Be a woman of childbearing potential who agrees to use a highly effective contraceptive method throughout the study and for a duration of 30 days after the last dose of BCX9930. c. Alternatively, true abstinence is acceptable for women of childbearing potential when it is in line with the subject’s preferred and usual lifestyle. 5. Male participants will continue to meet at least one of the following requirements per the prior study: a. Males with a female partner of childbearing potential (including a pregnant partner) must use condoms throughout the study and for a duration of 90 days after the dose of BCX9930 unless their partner is using a highly effective contraceptive method independent of the study. b. Alternatively, true abstinence is acceptable when it is in line with the subject’s preferred and usual lifestyle. 6. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures and restrictions for the study Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 29 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject. 2. An ongoing adverse event (AE), including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the subject to continue study-specific procedures or it is considered not to be in the subject’s best interest to continue or benefit-risk assessment is no longer in favor of the subject’s continued treatment. 3. Daily use of medications listed in the currently applicable prohibited medications list. 4. Known or suspected hypersensitivity to BCX9930 or any of its formulation excipients (Note: prior drug rash is not exclusionary).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To provide continued assess to BCX9930 for subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have benefitted from treatment with BCX9930 in another BioCryst-sponsored study and who, in the opinion of the Investigator, would benefit from continued treatment with BCX9930, and who who do not have access to other effective treatment options To monitor the safety of BCX9930 in subjects continuing to receive BCX9930 for the treatment of PNH ;Secondary Objective: Not Applicable;Primary end point(s): Subject incidence of graded treatment-emergent adverse events (TEAEs), laboratory abnormalities, changes to vital signs and physical examination findings;Timepoint(s) of evaluation of this end point: week 96 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: not applicable | — |
Countries
France, Hungary, Korea, Republic of, Malaysia, South Africa, Spain, United Kingdom
Contacts
Public ContactProject Management
AMS Advanced Medical Services
Outcome results
None listed