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Recruiting NCT06449001

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

Phase III Interventional Paroxysmal Nocturnal Hemoglobinuria PNH Extravascular Hemolysis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Danicopan.
Who it may be relevant to
Registry conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis. Basic parameters: 12 years — 17 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Canada, France, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis

Overview

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).

Interventions

  • Drug Danicopan
    Participants will receive danicopan on a weight-based dosing regimen.

Primary outcome measures

  • Change From Baseline in Hemoglobin (Hgb) Concentration at Week 12 [Time frame: Baseline, Week 12]
Secondary outcome measures (9)
  • Maximum Plasma Concentration (Cmax) of Danicopan [Time frame: Day 1 up to Week 12]
  • Number of Participants With Transfusion Avoidance Through Weeks 12 and 24 [Time frame: Weeks 12 and 24]
  • Change From Baseline in Absolute Reticulocyte Count at Weeks 12 and 24 [Time frame: Baseline, Weeks 12 and 24]
  • Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales Score at Weeks 12 and 24 [Time frame: Baseline, Weeks 12 and 24]
  • Change from Baseline in Pediatric Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 12 and 24 [Time frame: Baseline, Weeks 12 and 24]
  • Acceptability and Palatability Questionnaire Score [Time frame: Week 2]
  • Change from Baseline in Hgb Concentration at Week 24 [Time frame: Baseline, Week 24]
  • Change from Baseline in Serum Alternative Pathway (AP) Activity [Time frame: Baseline up to Week 64]
  • Change from Baseline in Plasma Bb Concentrations [Time frame: Baseline up to Week 64]

Eligibility criteria

Inclusion 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 the first 12 weeks of this study.
  • 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

Exclusion criteria

  • 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 weeks before Day 1 and is expected to remain stable through Week 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 administration.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United Kingdom · 2 centers
  • Research Site — Leeds
  • Research Site — London
Canada · 1 center
  • Research Site — Saskatoon
France · 1 center
  • Research Site — Paris

Identifiers

NCT: NCT06449001 · D7332C00006 · ALXN2040-PNH-302

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗