Paroxysmal Nocturnal Hemoglobinuria study: Pegcetacoplan
Official title A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
ClinicalTrials.gov ID: NCT04901936
What this study is testing
What is Pegcetacoplan?
An investigational subcutaneous injectable medicine being studied as a potential treatment for paroxysmal nocturnal hemoglobinuria (PNH) in adolescents. It is designed to inhibit the complement protein C3, which may reduce the destruction of red blood cells.
Also referred to as Empaveli.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to check if the medicine is safe, works well, and how the body handles it.
- Who it's for
- The study is looking for teenagers aged 12 to 17 who have paroxysmal nocturnal hemoglobinuria (PNH).
- What's involved
- Participants will receive the study medicine and undergo tests to monitor their health and how their body processes the drug.
- Time commitment
- The source text does not specify the total duration or the number of visits required.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 17
You may be able to join if
- 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...
- Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia.
You likely can't join if
- 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
See the full eligibility 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%)
- Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia.
- Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia. Ongoing anemia is defined as Hb \ ULN
- Have a platelet count \>75,000/mm3 and an absolute neutrophil count \>1000/mm3
- 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
The study team makes the final eligibility decision.
Where it is enrolling
- Atlanta, Georgia, United States
- Ampang, Malaysia
- Utrecht, Netherlands
- Belgrade, Serbia
- Barcelona, Spain
- Madrid, Spain
- Chiang Mai, Thailand
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Atlanta, Georgia, United States; Ampang, Malaysia; Utrecht, Netherlands; Belgrade, Serbia; Barcelona, Spain; Madrid, Spain and 1 more location. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.