Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
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 have completed treatment in another BCX9930 clinical study and, in the opinion of the investigator, would benefit from continued treatment with BCX9930. 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 180 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
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 assess the safety and tolerability of long-term monotherapy with BCX9930 in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who previously received BCX9930 in a BioCryst-sponsored study;Secondary Objective: •To assess the continued effectiveness of BCX9930 in treatment of PNH during long-term administration •To evaluate the effects of long-term monotherapy with BCX9930 on the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale and Quality of Life Questionnaire for Patients with Aplastic Anemia/PNH (QLQ-AA/PNH) •To characterize plasma concentrations of BCX9930 and its metabolite BCX13559 (M1a) throughout the treatment period •To characterize the effects of long-term BCX9930 monotherapy in subjects with PNH on pharmacodynamic (PD) and complement biomarkers ;Primary end point(s): •Subject incidence of graded treatment-emergent adverse events (TEAEs), laboratory abnormalities, changes to vital signs, electrocardiogram (ECG) results, and physical examination findings;Timepoint(s) of evaluation of this end point: week 48 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change from baseline (CFB) in hemoglobin (Hb) • Proportion of subjects with Hb = 12 g/dL • Proportion of subjects with Hb stabilization, defined as avoidance of a > 2 g/dL decrease in the absence of transfusion • Percent CFB in lactate dehydrogenase (LDH) • Number of units of packed red blood cells (pRBCs) transfused • Proportion of subjects who are transfusion-free • CFB in FACIT-Fatigue scale score through 48 weeks of treatment in Study 205 • CFB in QLQ-AA/PNH scores through 48 weeks of treatment in Study 205 • Plasma concentrations of BCX9930 and BCX13559 at steady state in subjects with PNH ;Timepoint(s) of evaluation of this end point: week 48 | — |
Countries
Argentina, Austria, Azerbaijan, Brazil, Canada, China, Colombia, Czechia, France, Hong Kong, Hungary, Italy, Japan, Korea, Republic of, Lithuania, Malaysia, Netherlands, Philippines, Romania, Serbia, Slovakia, South Africa, Spain, Taiwan, Turkey, United Kingdom, United States
Contacts
Public ContactProject Management
AMS Advanced Medical Services
Outcome results
None listed