Phase 3
Completed N=53
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Source: ClinicalTrials.gov NCT04085601 ↗Enrolled (actual)
53
Serious AEs
14.1%
Results posted
Oct 2022
Primary outcomePrimary: Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization — 30; 0 Participants — p=<0.0001
◆ Published Evidence
Emerging
6citations · ~2 / year
The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA.
Summary
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
Linked Publications (5)
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The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA.
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Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France.
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Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan.
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Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin ≥10 g/dL) who had received eculizumab or were naive to complement inhibitors.
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Normalization of Hemoglobin, Lactate Dehydrogenase, and Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan.
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 |
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
Data sourced from ClinicalTrials.gov (NCT04085601) and the linked publication. 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.