A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH

Apellis Pharmaceuticals

Status and phase

Completed

Phase 2

Conditions

PNH

Treatments

Drug: Pegcetacoplan

Study type

Interventional

Funder types

Industry

Identifiers

NCT03593200

APL2-202

Details and patient eligibility

About

This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.

Enrollment

4 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

At least 18 years old (inclusive)
Diagnosed with PNH (white blood cell (WBC) clone >10%)
Lactose dehydrogenase (LD) ≥2 times the upper limit of normal
Screening Ferritin ≥ normal and Total Iron Binding Capacity (TIBC) < LLN based on central lab reference ranges. If a subject is receiving iron supplements at screening, the investigator must ensure that his/her dose has been stable for 8 weeks prior to enrolment and must be maintained throughout the study
Last transfusion within 12 months prior to screening
Platelet count of >30,000/mm3 at the screening visit
Absolute neutrophil count >500/ mm3 at the screening visit
Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study
Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study
Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with pegcetacoplan. Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits
Willing and able to give informed consent

Exclusion criteria

Prior eculizumab (Soliris®) treatment
Active bacterial infection
Hereditary complement deficiency
History of bone marrow transplantation
Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin
Participation in any other investigational drug trial or exposure to another investigational agent, device or procedure within 30 days
Evidence of QTcF prolongation defined as >450 ms for males and >470 ms for females at screening
Breast-feeding women
History of meningococcal disease