About the PIONEER Study

About Clinical Trials

Visit the "Understanding Clinical Research" page for definitions and explanations

This study is evaluating the safety and effectiveness of an investigational medication called pegcetacoplan to reduce anemia in 12 to 17 year-olds (adolescents) who have PNH.

Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare blood disease where blood cells are vulnerable to attack and destruction by a particular part of the body’s immune system called the complement system. The red blood cells are missing a protein that protects them from the complement system and so they are more likely to be destroyed.

Hemolysis is a process by which red blood cells are destroyed, resulting in the release of hemoglobin. Hemoglobin is the part of red blood cells that carries oxygen around the body. The hemolysis, or destruction, of red blood cells, and release of hemoglobin, is responsible for many of the symptoms of PNH, including anemia.

Symptoms of PNH can include:

  • Dark or tea-colored urine
  • Anemia (low red blood cell count). This can lead to feeling tired, having headaches, having trouble breathing during exercise, or having an irregular heartbeat
  • Muscle spasms, especially in the abdomen / belly, and in the esophagus, which can impact swallowing

More information about PNH can be found at the following websites*

*These links are being provided as a convenience, for informational purposes only and do not replace medical advice; they do not constitute an endorsement or an approval by Apellis Pharmaceuticals, Inc. for services or opinions of the organizations. Apellis Pharmaceuticals, Inc. bears no responsibility for the accuracy, legality or content of the external sites or for that of subsequent links. Contact the external site for answers to questions regarding its content.

The PIONEER study is for people who:

  • Are 12 - 17 years old
  • Weigh at least 20 kg (44 lbs)
  • Have been diagnosed with PNH (primary nocturnal hemoglobinuria) and have anemia, confirmed by blood tests

Adolescents currently taking an approved complement inhibitor such as eculizumab or ravulizumab, or other treatments for PNH, may be eligible to participate. The study doctor can provide more detail.

Additional criteria will be assessed at the research site to confirm eligibility.

1Eculizumab and ravulizumab are manufactured by Alexion Pharmaceuticals, Inc.

The PIONEER study is not for:

  • Adults (people 18 years of age or older) with PNH
  • Anyone with a history of hereditary fructose intolerance (HFI), hereditary complement deficiency, bone marrow transplant, or meningococcal disease
  • Females who are pregnant or breastfeeding

Additional criteria will be assessed at the research site to confirm eligibility.

The investigational medication in this study is called pegcetacoplan (peg-set-a-koé-plan). It is an investigational, targeted C3 inhibitor that is administered via a needle under the skin (subcutaneously) twice a week, at home. Study participants and/or their caregiver(s) will be taught how to administer the study drug at home.

Investigational means that it is not approved by health authorities for adolescents with PNH.

Pegcetacoplan, the investigational medication, targets the complement system, the part of the immune system that is responsible for the destruction of red blood cells in PNH. It has been developed to regulate over-activation of the complement system. It is thought that this has the potential to prevent the destruction of red blood cells and that this may reduce the symptoms of PNH.

All study participants who complete the screening period and are confirmed eligible for the study will receive pegcetacoplan during the study.

The total length of participation may vary but will be at least 6 months:

  • Screening Period: To confirm study eligibility before receiving the investigational medication. This can take up to 4 weeks.
  • Run-in Period: Only for participants who are currently taking an approved complement inhibitor, to allow for the transition from their current complement inhibitor medication to the investigational medication. The duration of the run-in period may be 4 or 8 weeks.
  • Study Treatment Period: To evaluate the safety and effectiveness of pegcetacoplan in adolescents and understand how it is processed by the body. This will last for up to 4 months.
  • Long-Term Extension Period: This is an optional part of the study, to evaluate the long-term safety and effectiveness of pegcetacoplan in adolescents. It could continue for many months.
  • Follow-up Period: To evaluate safety and how study participants are doing for up to 2 months after the last dose of pegcetacoplan.

During the 4-month Study Treatment Period, participants will have approximately 7 clinic visits. During the Long-Term Extension Period, clinic visits will occur every 3 months. The Follow-up Period includes 2 clinic visits.

Reimbursement or pre-paid travel support may be available to study participants and their caregiver(s).

Study participants or caregivers will administer pegcetacoplan twice a week at home and keep a dosing diary. There are no other at-home assessments for this study.

All study-related tests and evaluations, and investigational medication will be provided at no cost. Reimbursement or pre-paid travel support for study-related visits may be available to participants and their caregiver(s). The study team can discuss this with you in more detail.

No, connecting with a site does not obligate you to participate, nor does it guarantee your eligibility to participate in the study. Participating in this or any clinical trial is completely voluntary. If you decide to participate in a research study, you are always free to withdraw at any time without any penalty or effect on your future medical care.

Should COVID-19, or restrictions related to the pandemic, affect the ability of participants to go to the clinic for study visits, the study doctor will discuss the available options with participants.