Phase 1 N=9 Treatment

A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Bottom Line

View on ClinicalTrials.gov: NCT02264639 ↗

Enrolled (actual)

Serious AEs

13.3%

Results posted

Jan 2021

Primary outcome: Primary: Number of Subjects With Treatment-Emergent Adverse Events (TEAEs), Including by Severity, During Single-dose Phase — 1; 2; 0; 1 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: Pegcetacoplan (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Apellis Pharmaceuticals, Inc.
Primary completion: Oct 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Subjects With Treatment-Emergent Adverse Events (TEAEs), Including by Severity, During Single-dose Phase	1; 2; 0; 1; 0; 0	—
PRIMARY Number of Subjects With TEAEs, Including by Severity, During Multiple-dose Phase	1; 2; 1; 6; 0; 1	—
PRIMARY Area Under the Curve (AUC) From Time 0 to the Last Measurable Concentration (AUC0-t) Over the Multiple Dosing Phase for Cohort 4	6,500,000	—
PRIMARY Maximum Pre-dose Serum Concentration (Ctrough,Max) Over the Multiple Dosing Phase for Cohort 4	627; 543; 624	—

Summary

This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide decisions to further develop the drug.

Eligibility Criteria

Inclusion Criteria

Male or Female
At least 18 years of age
Weigh >55 kg
Diagnosed with PNH
On treatment with eculizumab (Soliris®) for at least 3 months
Hb 30,000/mm3
Absolute neutrophil count > 500/mm3
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 (see below)
Males with female partners of child bearing potential must agree to use protocol defined methods of contraception (see below) and agree to refrain from donating sperm for the duration of the study
Willing and able to give informed consent

Exclusion Criteria

Active bacterial infection
Known infection with hepatitis B, C or HIV
Hereditary complement deficiency
History of bone marrow transplantation
Participation in any other investigational drug trial or exposure to other 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
Creatinine clearance (CrCl) < 50 mL/min (Cockcroft-Gault formula) at screening
Breast-feeding women
History of meningococcal disease
No vaccination against N. meningitidis types A, C, W, Y and B (administered as two separate vaccinations), Pneumococcal conjugate vaccine or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23, respectively) and Haemophilus influenzae Type B (Hib) vaccination within 2 years prior to Day 1 (Visit 2) dosing.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02264639). 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.