At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Pegcetacoplan (drug)
- How long the study runs
- Study runs about 94 months (dates as stated)
- About the drug or intervention
- Pegcetacoplan — drug: Complement (C3) inhibitor
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a medicine called pegcetacoplan in children aged 12 to 17 who have paroxysmal nocturnal haemoglobinuria (PNH), a rare blood condition. PNH causes red blood cells to break down, leading to a type of anaemia. The study is run by Apellis Pharmaceuticals, Inc.
Who can take part
- Aged 12 to 17 at the time of screening
- Weigh at least 20 kg (about 44 lbs)
- Have a confirmed diagnosis of PNH, based on a specific blood test showing that more than 10% of certain white blood cells are affected
- Either not currently taking an approved complement inhibitor medicine (eculizumab or ravulizumab) and have haemolytic anaemia (low haemoglobin and high LDH, a marker of red blood cell breakdown), or currently taking one of these medicines and still have ongoing anaemia (low haemoglobin and high reticulocyte count, a marker of the body making new red blood cells)
- Have platelet counts above 75,000/mm3 and neutrophil (a type of white blood cell) counts above 1,000/mm3
Who may not be able to
- Adults aged 18 or older with PNH
- Known or suspected hereditary fructose intolerance (a rare inherited problem processing a type of sugar)
- A history of hereditary complement deficiency (an inherited immune system problem), bone marrow transplant, or meningococcal disease (serious infections such as meningitis or blood poisoning)
- Females who are pregnant or breastfeeding
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 12 Years to 17 Years
- Who
- All
- Number of participants
- 12
- Started
- 2021-02-04
- Last checked
- 2026-06
Plain English Summary
What is this study?
- • Testing a new treatment for paroxysmal nocturnal hemoglobinuria (pnh)
- • Phase2 - 12 participants
- • 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)
Who can take part?
- • Ages 12 Years to 17 Years
- • Diagnosed with paroxysmal nocturnal hemoglobinuria (pnh)
Where?
- • London - St. Mary's Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
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).
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 12 Years - 17 Years
- Who can join: All genders
What the study is looking for
- ✓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:
- ✓Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of...
Who cannot take part
- ✗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,...
- ✗Females who are pregnant or breastfeeding
See the full criteria
Where Is This Study? (1 UK site)
St. Mary's Hospital
London W2 1NY, United Kingdom
How to Get in Touch
Apellis Clinical Trial Information Line
Sponsor contactCONTACT