A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT ID: NCT04901936
Last Updated: 2025-06-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
12 participants
INTERVENTIONAL
2021-02-04
2028-12-31
Brief Summary
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Detailed Description
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All eligible study participants will receive pegcetacoplan, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or caregivers will be trained on home administration of pegcetacoplan.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Pegcetacoplan
Pegcetacoplan
Complement (C3) inhibitor
Interventions
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Pegcetacoplan
Complement (C3) inhibitor
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
12 Years
17 Years
ALL
No
Sponsors
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Apellis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Children's Hospital of Atlanta
Atlanta, Georgia, United States
Motol University Hospital
Prague, , Czechia
Robert-Debré Hospital Paris
Paris, , France
Hospital Ampang
Ampang, , Malaysia
Radboud University Hospital Nijmegen
Nijmegen, , Netherlands
University Medical Center Utrecht
Utrecht, , Netherlands
University Children's Hospital
Belgrade, , Serbia
University Hospital Vall d'Hebron
Barcelona, , Spain
University Hospital 12 de Octubre
Madrid, , Spain
Phramongkutklao Hospital and College of Medicine
Bangkok, , Thailand
Maharaj Nakorn Chiang Mai hospital
Chiang Mai, , Thailand
St. Mary's Hospital
London, , United Kingdom
Countries
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Central Contacts
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Apellis Clinical Trial Information Line
Role: CONTACT
Other Identifiers
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APL2-PNH-209
Identifier Type: -
Identifier Source: org_study_id
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