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

ConditionsParoxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12-17
SponsorApellis Pharmaceuticals, Inc.

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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%)

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Pegcetacoplan

    Drug

    Complement (C3) inhibitor

Treatment groups

12 Participants
are divided into 1 treatment group
Group A: PegcetacoplanExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Pegcetacoplan serum concentrations over the course of the 16-week treatment period

Time frame
16 weeks
2

Change from baseline to Wk 16 in hemoglobin (Hb)

Time frame
16 weeks
3

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
4

Change from baseline to wk 16 lactate dehydrogenase (LDH)

Time frame
16 weeks

Secondary outcomes

1

Change from baseline from week 16 to week 52 of C3 deposition on RBC cells

Time frame
Week 16-52
2

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
3

Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan

Time frame
Week 16-52
4

Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb

Time frame
Week 16-52

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Apellis Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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