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COMPLETEDPHASE1

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

An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).

NCT02264639•Apellis Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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 \< 10 g/dL at screening OR have received at least one transfusion within 12 months prior to screening
•Platelet count of \>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
+1 more criteria

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.

Locations (7)

University of Southern California Norris Comprehensive Cancer Center

Los Angeles, California, United States

Lakes Research

Miami Lakes, Florida, United States

University of Lousiville

Louisville, Kentucky, United States

John Hopkins Hospital

Baltimore, Maryland, United States

Cure 4 The Kids Foundation

Las Vegas, Nevada, United States

Duke University Medical Center

Durham, North Carolina, United States

Cleveland Clinic

Cleveland, Ohio, United States

Key Dates

Start Date

February 23, 2015

Primary Completion Date

October 22, 2018

Completion Date

October 22, 2018

Last Updated

January 8, 2021

Enrollment

ACTUAL participants

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

Pegcetacoplan

DRUG

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)+1 more

View study

RECRUITINGPHASE1

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment

NCT07152288

Paroxysmal Nocturnal Hemoglobinuria (PNH)

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 8, 2021Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment

Hematological Disorders in EHPVO Patients

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy