A Study of Pegcetacoplan in Pediatric Patients With Parox... | NCT04901936

Status Module

Record Verification Date

Jun 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Feb 4, 2021Actual

Primary Completion Date

Dec 2028Estimated

Completion Date

Dec 2028Estimated

First Submitted Date

Mar 8, 2021

First Submission Date that Met QC Criteria

May 20, 2021

First Posted Date

May 26, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 23, 2026

Last Update Posted Date

Jun 26, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

12Estimated

Label	Type	Description	Intervention Names
Pegcetacoplan	Experimental		Drug: Pegcetacoplan

Name	Type	Description	Arm Group Labels	Other Names
Pegcetacoplan	Drug	Complement (C3) inhibitor	Pegcetacoplan

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Change from baseline from week 16 to week 52 of C3 deposition on RBC cells		Week 16-52
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

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

12 Years

Maximum Age

17 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Apellis Clinical Trial Information Line	Contact	1-833-284-6361 (833-CT Info-1)		clinicaltrials@apellis.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital of Atlanta	Recruiting	Atlanta	Georgia	30329	United States

ID	Term
D006457	Hemoglobinuria, Paroxysmal
D000743	Anemia, Hemolytic

ID	Term
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D009190	Myelodysplastic Syndromes
D001855	Bone Marrow Diseases