Phase 2
Completed N=4
A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
PNH
Source: ClinicalTrials.gov NCT03593200 ↗
Enrolled (actual)
4
Serious AEs
25.0%
Results posted
Dec 2020
Primary outcomePrimary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity — 3; 2; 1; 0 Participants
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.
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 | — |
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
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. Informational only — not medical advice.