Instructions
If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.
Status:
Recruiting
Associated Drug(s):
Phase:
Phase 2
Gender:
Female
Male
Age Group:
12 years to 17 years
Accepts Healthy Volunteers:
No
Inclusion Criteria:
- 12-17 years old at the time of screening
- Weigh at least 20 kg (approximately 44 lbs)
- Have been diagnosed with PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%)
- EITHER:
- Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia. Hemolytic anemia is defined as hemoglobin (Hb) less than the lower limit of normal (Hb < LLN) and LDH >1.5 times the upper limit of normal (ULN);
OR - Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia. Ongoing anemia is defined as Hb < LLN and ARC > ULN
- Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia. Hemolytic anemia is defined as hemoglobin (Hb) less than the lower limit of normal (Hb < LLN) and LDH >1.5 times the upper limit of normal (ULN);
- Have a platelet count >75,000/mm3 and an absolute neutrophil count >1000/mm3
Exclusion Criteria:
- Are an adult, 18 years of age or older, with PNH
- Known or suspected hereditary fructose intolerance (HFI)
- History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
- Females who are pregnant or breastfeeding