Pegcetacoplan treatment for adolescents (12–17) with PNH

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label Phase 2 trial enrolls adolescents aged 12–17 with confirmed PNH and includes a 4-week screening period followed by a 16-week treatment period, with a 4-week run-in for those switching from a C5 inhibitor. All participants receive pegcetacoplan administered by subcutaneous infusion twice weekly at home after training for self- or caregiver-administration. The study measures safety, biological activity (how the drug is processed), and effectiveness on hemolysis and blood counts, with participants either entering a long-term extension or a 2-month follow-up after treatment. Regular clinic visits at participating sites are required for monitoring and laboratory testing.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Are 12-17 years old at the time of screening
* Weigh at least 20 kg (approx. 44 lbs)
* Have the diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone \>10%)
* EITHER:

  * Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia. Hemolytic anemia is defined as hemoglobin (Hb) less than the lower limit of normal (Hb \< LLN) and LDH \>1.5 times the upper limit of normal (ULN); OR
  * Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia. Ongoing anemia is defined as Hb \< LLN and ARC \> ULN
* Have a platelet count \>75,000/mm3 and an absolute neutrophil count \>1000/mm3

Exclusion Criteria:

* Are an adult, 18 years of age or older, with PNH
* Known or suspected hereditary fructose intolerance (HFI)
* History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
* Females who are pregnant or breastfeeding

Where it's running

• Children's Hospital of Atlanta
  Atlanta, Georgia, United States Recruiting
• Hospital Ampang
  Ampang, Malaysia Recruiting
• University Medical Center Utrecht
  Utrecht, Netherlands Recruiting
• University Children's Hospital
  Belgrade, Serbia Recruiting
• University Hospital Vall d'Hebron
  Barcelona, Spain Recruiting
• University Hospital 12 de Octubre
  Madrid, Spain Recruiting

Study contacts

• Study coordinator: Apellis Clinical Trial Information Line
• Email: clinicaltrials@apellis.com
• Phone: 1-833-284-6361 (833-CT Info-1)

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.