A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Recruiting
Phase 2
Interventional Study
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Hemoglobinuria
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 12 – 17
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 12 (estimated)
- Sponsor
- Apellis Pharmaceuticals, Inc. · Industry
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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).
Trial Locations
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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 (eculizum…
Contacts
Apellis Clinical Trial Information Line
1-833-284-6361 (833-CT Info-1)
CONTACT