Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-003829-18-GB

Enrollment

Registered

2020-02-05

Start date

2020-09-18

Completion date

Unknown

Last updated

2020-10-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders

Interventions

Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 Pharmaceutical Form: Tablet INN or Proposed INN: DANICOPAN CAS Number: 1903768-17-1 Current Sponsor code: ACH-0144471 Other descriptive nam

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Diagnosis of PNH • Clinically Evident EVH defined by: - Anemia (Hgb =9.5 gram/deciliter) with absolute reticulocyte count =120 x 10^9/liter. - At least 1 packed red blood cell or whole blood transfusion within 6 months prior to the start of the study. • Receiving a C5 inhibitor for at least 6 months prior to Day 1 • Platelet count =30,000/microliters (µL) • Absolute neutrophil counts =750/µL. • Documentation of/or willingness to receive vaccinations and prophylactic antibiotics as required. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 82 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2

Exclusion criteria

Exclusion criteria: • History of a major organ transplant or hematopoietic stem cell transplantation (HSCT). • Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants. • Known or suspected complement deficiency. • Laboratory abnormalities at screening, including: - Alkaline phosphatase >2 x upper limit of normal (ULN). - Alanine aminotransferase >2 x ULN. - Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert’s Syndrome. • Current evidence of biliary cholestasis. • Estimated glomerular filtration rate <30 milliliters/minute/1.73 meter squared and/or are on dialysis.

Design outcomes

Secondary

Measure	Time frame
Secondary end point(s): • Percentage Of Participants With Transfusion Avoidance • Change From Baseline In Functional Assessment Of Chronic Illness Therapy (FACIT) Fatigue Scores • Change From Baseline In Absolute Reticulocyte Count;Timepoint(s) of evaluation of this end point: At Week 12	—

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy of danicopan as compared to placebo as add-on therapy to a C5 inhibitor at 12 weeks;Secondary Objective: • Percentage Of Participants With Transfusion Avoidance • Change From Baseline In Functional Assessment Of Chronic Illness Therapy (FACIT) Fatigue Scores • Change From Baseline In Absolute Reticulocyte Count;Primary end point(s): • Change From Baseline In Hemoglobin (Hgb);Timepoint(s) of evaluation of this end point: At Week 12	—

Measure

Time frame

Main Objective: To evaluate the efficacy of danicopan as compared to placebo as add-on therapy to a C5 inhibitor at 12 weeks;Secondary Objective: • Percentage Of Participants With Transfusion Avoidance • Change From Baseline In Functional Assessment Of Chronic Illness Therapy (FACIT) Fatigue Scores • Change From Baseline In Absolute Reticulocyte Count;Primary end point(s): • Change From Baseline In Hemoglobin (Hgb);Timepoint(s) of evaluation of this end point: At Week 12

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Countries

Australia, Austria, Brazil, Canada, Denmark, Finland, France, Germany, Italy, Japan, Korea, Republic of, Malaysia, Netherlands, Poland, Spain, Sweden, Switzerland, Taiwan, Thailand, United Kingdom, United States

Contacts

Public ContactEuropean Clinical Trial Information

Alexion Europe SAS

clinicaltrials.eu@alexion.com0033 147100615

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026