RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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).

Official Title

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

Quick Facts

Study Start:2021-02-04
Study Completion:2028-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04901936

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.

Ages Eligible for Study:12 Years to 17 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Are 12-17 years old at the time of screening
  2. * Weigh at least 20 kg (approx. 44 lbs)
  3. * Have the diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone \>10%)
  4. * EITHER:
  5. * 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
  6. * 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
  7. * Have a platelet count \>75,000/mm3 and an absolute neutrophil count \>1000/mm3
  1. * Are an adult, 18 years of age or older, with PNH
  2. * Known or suspected hereditary fructose intolerance (HFI)
  3. * History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
  4. * Females who are pregnant or breastfeeding

Contacts and Locations

Study Contact

Apellis Clinical Trial Information Line
CONTACT
1-833-284-6361 (833-CT Info-1)
clinicaltrials@apellis.com

Study Locations (Sites)

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

Collaborators and Investigators

Sponsor: Apellis Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-02-04
Study Completion Date2028-12

Study Record Updates

Study Start Date2021-02-04
Study Completion Date2028-12

Terms related to this study

Keywords Provided by Researchers

  • Paroxysmal Nocturnal Hemoglobinuria
  • PNH
  • Pediatric
  • Adolescent
  • Apellis
  • Anemia, Hemolytic
  • Pegcetacoplan
  • APL-2

Additional Relevant MeSH Terms

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Paroxysmal Hemoglobinuria