A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
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ClinicalTrials.gov Identifier: NCT04085601 |
Recruitment Status :
Active, not recruiting
First Posted : September 11, 2019
Last Update Posted : December 19, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2 | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 53 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
Actual Study Start Date : | August 15, 2019 |
Estimated Primary Completion Date : | January 8, 2021 |
Estimated Study Completion Date : | January 30, 2021 |

Arm | Intervention/treatment |
---|---|
No Intervention: Standard of Care (SOC) excluding complement inhibitors | |
Experimental: 1,080mg APL-2 administered subcutaneously twice weekly |
Drug: APL-2
Complement (C3) Inhibitor |
- Hemoglobin stabilization defined as avoidance of a > 1 g/dl decrease in hemoglobin levels in the absence of transfusion. [ Time Frame: Baseline through Week 26 ]
- Reduction in lactate dehydrogenase (LDH) level [ Time Frame: Baseline to Week 26 ]

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.
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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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04085601

Responsible Party: | Apellis Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT04085601 |
Other Study ID Numbers: |
APL2-308 |
First Posted: | September 11, 2019 Key Record Dates |
Last Update Posted: | December 19, 2020 |
Last Verified: | December 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Hemoglobinuria Hemoglobinuria, Paroxysmal Proteinuria Urination Disorders Urologic Diseases Urological Manifestations |
Anemia, Hemolytic Anemia Hematologic Diseases Myelodysplastic Syndromes Bone Marrow Diseases |