RECRUITING

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Description

The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria
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Related Conditions:

Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria

Study Overview

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Study Details

Study overview

The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).

An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Condition
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Intervention / Treatment

-

Contacts and Locations

Atlanta

Children's Hospital of Atlanta, Atlanta, Georgia, United States, 30329

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

12 Years to 17 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Apellis Pharmaceuticals, Inc.,

Study Record Dates

2028-12