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New treatment option for Paroxysmal Nocturnal Hemoglobinuria
Official title Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
ClinicalTrials.gov ID: NCT06449001
What this study is testing
What is Danicopan?
Danicopan is an investigational medicine, being studied as a potential treatment for paroxysmal nocturnal hemoglobinuria.
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
- The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).
- Phase 3: a large, late-stage study
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
- Confirmed diagnosis of PNH.
- CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
- Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and...
- all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at...
- vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae
You likely can't join if
- Platelet count \< 30000/μL or there is a need for platelet transfusions.
- ANC \< 500/μL.
- Clinically significant laboratory abnormalities related to liver function, including:
- ALT \> 2 × ULN or ALT \> 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
- Direct bilirubin \> 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.
- Current evidence of biliary cholestasis.
See the full eligibility criteria
- Confirmed diagnosis of PNH.
- CS-EVH defined by: Anemia: Hgb ≤ 11.0 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
- Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during...
- all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
- vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae
- Platelet count \< 30000/μL or there is a need for platelet transfusions.
- ANC \< 500/μL.
- Clinically significant laboratory abnormalities related to liver function, including:
- ALT \> 2 × ULN or ALT \> 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
- Direct bilirubin \> 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.
- Current evidence of biliary cholestasis.
- Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12...
- History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.
- Known or suspected complement deficiency.
- Active bacterial or viral infection, a body temperature \> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention...
The study team makes the final eligibility decision.
Where it's taking place
- Saskatoon, Saskatchewan, Canada
- Paris, France
- Leeds, United Kingdom
- London, United Kingdom
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 - 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?
Study sites include Saskatoon, Saskatchewan, Canada; Paris, France; Leeds, United Kingdom; London, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.