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Complement Inhibitor
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab (PNH Trial)
Phase 2
Waitlist Available
Research Sponsored by Alexion
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1 (after dosing) through end of study (maximum exposure: 1631 days)
Awards & highlights
No Placebo-Only Group
Summary
This trial tests if danicopan, when used with eculizumab, can improve anemia in patients with PNH over several months. Danicopan aims to increase hemoglobin levels, while eculizumab stops red blood cell breakdown.
Eligible Conditions
- Paroxysmal Nocturnal Hemoglobinuria
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ day 1 (after dosing) through end of study (maximum exposure: 1631 days)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1 (after dosing) through end of study (maximum exposure: 1631 days)
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug
Side effects data
From 2024 Phase 3 trial • 88 Patients • NCT0446946513%
Pyrexia
11%
Diarrhoea
11%
Headache
5%
Anaemia
5%
Fatigue
5%
Neutropenia
4%
Dizziness
4%
Asthenia
4%
Dyspnoea
4%
Back pain
4%
Breakthrough haemolysis
4%
Gastroenteritis
2%
Staphylococcal sepsis
2%
Abdominal pain
2%
Pain in extremity
2%
Contusion
2%
Urinary tract infection
2%
Chromaturia
2%
Dieulafoy's vascular malformation
2%
Insomnia
2%
Vomiting
2%
COVID-19 pneumonia
2%
COVID-19
2%
Aspartate aminotransferase increased
2%
Cough
2%
Arthralgia
2%
Thrombocytopenia
2%
Nausea
2%
Chest discomfort
2%
Constipation
2%
Nasopharyngitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Danicopan-Danicopan (TP2)
Danicopan-Danicopan (LTE)
Placebo-Danicopan (TP2)
Placebo-Danicopan (LTE)
Danicopan (TP1)
Placebo (TP1)
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
4Treatment groups
Experimental Treatment
Group I: Group 4: Optimal Dose of Danicopan TID + EculizumabExperimental Treatment2 Interventions
Optimal dose (starting dose of either 100, 150, or 200 mg, as determined from Groups 1-3) of danicopan TID in combination with eculizumab.
Group II: Group 3: Initial dose of 100, 150, or 200 mg Danicopan TID + EculizumabExperimental Treatment2 Interventions
Starting dose of 100, 150, or 200 mg danicopan TID in combination with eculizumab.
Group III: Group 2: Initial dose 100 or 150 mg Danicopan TID + EculizumabExperimental Treatment2 Interventions
Starting dose of 100 or 150 mg danicopan TID in combination with eculizumab.
Group IV: Group 1: 100 mg Danicopan TID + EculizumabExperimental Treatment2 Interventions
Starting dose of 100 mg danicopan TID in combination with eculizumab.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Danicopan
2020
Completed Phase 3
~680
Eculizumab
2009
Completed Phase 4
~1200
Find a Location
Who is running the clinical trial?
AlexionLead Sponsor
246 Previous Clinical Trials
38,505 Total Patients Enrolled
Alexion Pharmaceuticals, Inc.Lead Sponsor
261 Previous Clinical Trials
140,472 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
36,591 Total Patients Enrolled
Achillion, a wholly owned subsidiary of AlexionIndustry Sponsor
27 Previous Clinical Trials
957 Total Patients Enrolled