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

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04901936
• Other study ID #: APL2-PNH-209
• Status: Recruiting
• Phase: Phase 2
• Start date: February 4, 2021
• Est. completion date: October 2024

Study information

• Verified date: July 2022
• Source: Apellis Pharmaceuticals, Inc.
• Contact: Apellis Clinical Trial Information Line
• Phone: 1-833-284-6361 (833-CT Info-1)
• Email: clinicaltrials[@]apellis.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

Description:

This is an open-label study to evaluate pegcetacoplan in people with PNH who are 12-17 years old. The study will consist of a 4-week screening period followed by a 16-week treatment period. Participants switching from a C5 inhibitor will have an additional 4 week run-in period between the screening and treatment periods. At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-month follow-up period. 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: October 2024
• Est. primary completion date: April 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Hemoglobinuria
• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention

Drug:
• Pegcetacoplan
Complement (C3) inhibitor

Locations

Country	Name	City	State
Czechia	Motol University Hospital	Prague
France	Robert-Debré Hospital Paris	Paris
Malaysia	Hospital Ampang	Ampang
Netherlands	Radboud University Hospital Nijmegen	Nijmegen
Serbia	University Children's Hospital	Belgrade
Spain	University Hospital Vall d'Hebron	Barcelona
Spain	University Hospital 12 de Octubre	Madrid
Thailand	Phramongkutklao Hospital and College of Medicine	Bangkok
United Kingdom	St. Mary's Hospital	London
United States	Children's Hospital of Atlanta	Atlanta	Georgia

Sponsors (1)

Lead Sponsor	Collaborator
Apellis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Czechia,  France,  Malaysia,  Netherlands,  Serbia,  Spain,  Thailand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pegcetacoplan serum concentrations over the course of the 16-week treatment period		16 weeks
Primary	Change from baseline to Wk 16 in hemoglobin (Hb)		16 weeks
Primary	Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections		16 weeks
Primary	Change from baseline to wk 16 lactate dehydrogenase (LDH)		16 weeks
Primary	Change from baseline to wk 16 absolute reticulocyte count (ARC)		16 weeks
Secondary	Change from baseline from week 16 to week 52 of C3 deposition on RBC cells		Week 16-52
Secondary	Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 52 weeks of treatment with pegcetacoplan		52 weeks
Secondary	Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan		Week 16-52
Secondary	Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb		Week 16-52
Secondary	Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments		Week 16-52
Secondary	Number of packed red blood cell (PRBC) units		Week 16-52
Secondary	Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan		Week 16-52
Secondary	Change from baseline to Week 52, and from Week 16 to Week 52, in LDH		Week 16-52
Secondary	Change from baseline to Week 52, and from Week 16 to Week 52 ARC		Week 16-52