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New drug pegcetacoplan tested in teens with rare blood disorder

NCT ID NCT04901936

Disease control Sponsor: Apellis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called pegcetacoplan in 12 to 17-year-olds with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug aims to control the disease by blocking part of the immune system. The trial will check safety, how the drug works in the body, and its effects on blood counts over 16 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pegcetacoplan
What this could lead to
If successful, this could provide a new treatment option for teenagers with PNH, helping control red blood cell destruction and reduce symptoms.
What could go wrong
This is a small, early-phase study with only 12 participants, so results may not apply to all patients. Risks include infections and other side effects from the drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2021

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal hemolytic anemia paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Paroxysmal hemoglobinuria Paroxysmal nocturnal hemoglobinuria, PNH

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    8 sites in 7 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's Hospital of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Hospital Ampang

    RECRUITING

    Ampang, 68000, Malaysia

  • Maharaj Nakorn Chiang Mai hospital

    RECRUITING

    Chiang Mai, 50200, Thailand

  • Motol University Hospital

    COMPLETED

    Prague, 150 06, Czechia

  • Phramongkutklao Hospital and College of Medicine

    COMPLETED

    Bangkok, 10400, Thailand

  • Radboud University Hospital Nijmegen

    COMPLETED

    Nijmegen, 6525 GA, Netherlands

  • Robert-Debré Hospital Paris

    NOT_YET_RECRUITING

    Paris, 75009, France

  • St. Mary's Hospital

    COMPLETED

    London, W2 1NY, United Kingdom

  • University Children's Hospital

    RECRUITING

    Belgrade, 11000, Serbia

  • University Hospital 12 de Octubre

    RECRUITING

    Madrid, E-28041, Spain

  • University Hospital Vall d'Hebron

    RECRUITING

    Barcelona, E-08035, Spain

  • University Medical Center Utrecht

    RECRUITING

    Utrecht, 3508 GA, Netherlands

More trials for these conditions

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