Phase 2 N=4 Treatment

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

PNH

Bottom Line

View on ClinicalTrials.gov: NCT03593200 ↗

Enrolled (actual)

Serious AEs

25.0%

Results posted

Dec 2020

Primary outcome: Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity — 3; 2; 1; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Pegcetacoplan (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Apellis Pharmaceuticals, Inc.
Primary completion: Oct 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity	3; 2; 1; 0; 2; 0	—
PRIMARY Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level	-2322.8	—
PRIMARY Mean Change From Baseline in Haptoglobin Level	0.08	—
PRIMARY Mean Change From Baseline in Hemoglobin (Hb) Level	5.27	—
SECONDARY Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score	6.5	—
SECONDARY Mean Change From Baseline in Absolute Reticulocyte Count (ARC) Level	-144.3	—
SECONDARY Mean Change From Baseline in Total Bilirubin Level	-21.53	—
SECONDARY Mean Number of Red Blood Cell (RBC) Transfusions Per Month	—	—
SECONDARY Mean Change From Baseline in Linear Analog Scale Assessment (LASA) Score for QoL	66.5	—
SECONDARY Mean Serum Concentrations of Pegcetacoplan	622.0	—
SECONDARY Mean Area Under the Serum Concentration Versus Time Curve From Time 0 to the Last Measurable Concentration at the End of the Study (AUCtotal)	5818803.2534	—
SECONDARY Mean Maximum Observed Predose Serum Concentration During the Study (Ctrough,Max,Total)	783.5	—

Summary

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.

Eligibility Criteria

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) 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03593200). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.