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

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Pegcetacoplan
Sponsored by
Apellis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH) focused on measuring Paroxysmal Nocturnal Hemoglobinuria, PNH, Pediatric, Adolescent, Apellis, Anemia, Hemolytic, Pegcetacoplan, APL-2

Eligibility Criteria

12 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital of AtlantaRecruiting
  • Motol University HospitalRecruiting
  • Robert-Debré Hospital Paris
  • Hospital AmpangRecruiting
  • Radboud University Hospital Nijmegen
  • University Children's Hospital
  • University Hospital Vall d'Hebron
  • University Hospital 12 de Octubre
  • Phramongkutklao Hospital and College of MedicineRecruiting
  • St. Mary's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Pegcetacoplan

Arm Description

Outcomes

Primary Outcome Measures

Pegcetacoplan serum concentrations over the course of the 16-week treatment period
Change from baseline to Wk 16 in hemoglobin (Hb)
Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections
Change from baseline to wk 16 lactate dehydrogenase (LDH)
Change from baseline to wk 16 absolute reticulocyte count (ARC)

Secondary Outcome Measures

Change from baseline from week 16 to week 52 of C3 deposition on RBC cells
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
Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan
Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb
Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments
Number of packed red blood cell (PRBC) units
Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan
Change from baseline to Week 52, and from Week 16 to Week 52, in LDH
Change from baseline to Week 52, and from Week 16 to Week 52 ARC

Full Information

First Posted
March 8, 2021
Last Updated
July 18, 2022
Sponsor
Apellis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04901936
Brief Title
A Study of Pegcetacoplan in Pediatric Patients With 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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
February 4, 2021 (Actual)
Primary Completion Date
April 2024 (Anticipated)
Study Completion Date
October 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Apellis Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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).
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
Keywords
Paroxysmal Nocturnal Hemoglobinuria, PNH, Pediatric, Adolescent, Apellis, Anemia, Hemolytic, Pegcetacoplan, APL-2

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Pegcetacoplan
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Pegcetacoplan
Intervention Description
Complement (C3) inhibitor
Primary Outcome Measure Information:
Title
Pegcetacoplan serum concentrations over the course of the 16-week treatment period
Time Frame
16 weeks
Title
Change from baseline to Wk 16 in hemoglobin (Hb)
Time Frame
16 weeks
Title
Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections
Time Frame
16 weeks
Title
Change from baseline to wk 16 lactate dehydrogenase (LDH)
Time Frame
16 weeks
Title
Change from baseline to wk 16 absolute reticulocyte count (ARC)
Time Frame
16 weeks
Secondary Outcome Measure Information:
Title
Change from baseline from week 16 to week 52 of C3 deposition on RBC cells
Time Frame
Week 16-52
Title
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
Time Frame
52 weeks
Title
Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan
Time Frame
Week 16-52
Title
Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb
Time Frame
Week 16-52
Title
Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments
Time Frame
Week 16-52
Title
Number of packed red blood cell (PRBC) units
Time Frame
Week 16-52
Title
Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan
Time Frame
Week 16-52
Title
Change from baseline to Week 52, and from Week 16 to Week 52, in LDH
Time Frame
Week 16-52
Title
Change from baseline to Week 52, and from Week 16 to Week 52 ARC
Time Frame
Week 16-52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Apellis Clinical Trial Information Line
Phone
1-833-284-6361 (833-CT Info-1)
Email
clinicaltrials@apellis.com
Facility Information:
Facility Name
Children's Hospital of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Individual Site Status
Recruiting
Facility Name
Motol University Hospital
City
Prague
ZIP/Postal Code
150 06
Country
Czechia
Individual Site Status
Recruiting
Facility Name
Robert-Debré Hospital Paris
City
Paris
ZIP/Postal Code
75009
Country
France
Individual Site Status
Not yet recruiting
Facility Name
Hospital Ampang
City
Ampang
ZIP/Postal Code
68000
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Radboud University Hospital Nijmegen
City
Nijmegen
ZIP/Postal Code
6525 GA
Country
Netherlands
Individual Site Status
Not yet recruiting
Facility Name
University Children's Hospital
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Individual Site Status
Not yet recruiting
Facility Name
University Hospital Vall d'Hebron
City
Barcelona
ZIP/Postal Code
E-08035
Country
Spain
Individual Site Status
Not yet recruiting
Facility Name
University Hospital 12 de Octubre
City
Madrid
ZIP/Postal Code
E-28041
Country
Spain
Individual Site Status
Not yet recruiting
Facility Name
Phramongkutklao Hospital and College of Medicine
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Individual Site Status
Recruiting
Facility Name
St. Mary's Hospital
City
London
ZIP/Postal Code
W2 1NY
Country
United Kingdom
Individual Site Status
Not yet recruiting

12. IPD Sharing Statement

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

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