Phase 3 N=53 Randomized Treatment

A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH

Paroxysmal Nocturnal Hemoglobinuria

Bottom Line

View on ClinicalTrials.gov: NCT04085601 ↗

Enrolled (actual)

Serious AEs

14.1%

Results posted

Oct 2022

Primary outcome: Primary: Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization — 30; 0 Participants — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: APL-2 (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Apellis Pharmaceuticals, Inc.
Primary completion: Jun 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization	30; 0	<0.0001 sig
PRIMARY Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26	-1870.47; -400.09	<0.0001 sig
SECONDARY Number of Subjects With an Hb Response in the Absence of Transfusions	25; 1	<0.0001 sig
SECONDARY Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26	-123.26; -19.44	0.0002 sig
SECONDARY Change From Baseline in Hb Concentration at Week 26	2.94; 0.27	0.0019 sig
SECONDARY Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline	11.4; 100	<0.0001 sig
SECONDARY Percentage of Subjects With Transfusion Avoidance	91.4; 5.6	<0.0001 sig
SECONDARY Number of PRBC Units Transfused From Baseline Through Week 26	0.0; 3.0	<0.0001 sig
SECONDARY Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26	7.78; 3.26	0.061
SECONDARY Percentage of Subjects With Hb Normalization Levels at Week 26	45.7; 0	0.001 sig
SECONDARY Percentage of Subjects With LDH Normalization at Week 26	65.7; 0	<0.0001 sig
SECONDARY Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26	18.90; -2.85	0.0006 sig
SECONDARY Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26	50.39; -5.39	0.005 sig
SECONDARY Percentage of Subjects With ARC Normalization	60.0; 5.6	0.0002 sig
SECONDARY Number of Subjects With Failure of Hb Stabilization	4; 18	<0.0001 sig
SECONDARY Time to First PRBC Transfusion	NA; 7.000	<0.0001 sig

Summary

Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .

Eligibility Criteria

Inclusion Criteria

Be at least 18 years old (inclusive).
Have LDH ≥1.5 x ULN at the screening visit.
Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
Have Hb less than the lower limit of normal (LLN) at the screening visit.
Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
Have a platelet count of >50, 000/mm3 at the screening visit.
Have an absolute neutrophil count >500/mm3 at the screening visit.

Exclusion Criteria

Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
Hereditary complement deficiency.
History of bone marrow transplantation.
Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:
Erythropoietin or immunosuppressants for at least 8 weeks
Systemic corticosteroids for at least 4 weeks
Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
Iron supplements, vitamin B12, or folic acid for at least 4 weeks
Low-molecular-weight heparin for at least 4 weeks

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04085601). 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.