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Complement Inhibitor

Danicopan for Paroxysmal Nocturnal Hemoglobinuria (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

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 weeks
Treatment ~ Varies
Follow Up ~day 1 (after dosing) through end of study (maximum exposure: 1631 days)
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 (after dosing) through end of study (maximum exposure: 1631 days) for reporting.

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 • 86 Patients • NCT04469465
11%
Headache
9%
Nausea
7%
Diarrhoea
7%
Arthralgia
5%
Hypertension
5%
Pyrexia
5%
Pain in extremity
5%
Vomiting
5%
Alanine aminotransferase increased
4%
Rhinorrhoea
4%
Constipation
4%
White blood cell count decreased
4%
Aspartate aminotransferase increased
4%
Myalgia
4%
Haemolysis
4%
Viral infection
4%
Urinary tract infection
4%
Oropharyngeal pain
2%
Decreased appetite
2%
Anaemia
2%
Dyspepsia
2%
Discoloured vomit
2%
Peripheral swelling
2%
Febrile nonhaemolytic transfusion reaction
2%
Bone pain
2%
Neutropenia
2%
Fatigue
2%
COVID-19
2%
SARS-CoV-2 test positive
2%
Hepatic function abnormal
2%
Proteinuria
2%
Abdominal discomfort
2%
Chest discomfort
2%
Upper respiratory tract infection
2%
Hot flush
2%
Sinusitis
2%
Leukopenia
2%
Cellulitis
2%
Post procedural diarrhoea
2%
Post procedural haemorrhage
2%
Thoracic vertebral fracture
2%
Flushing
2%
Chromaturia
2%
Epistaxis
2%
Cough
2%
Pancreatitis
2%
Blood pressure increased
2%
Dizziness
2%
Splenomegaly
2%
Post procedural contusion
2%
Noninfective gingivitis
2%
Salivary gland pain
2%
Stomatitis
2%
Jaundice
2%
Liver disorder
2%
Lethargy
2%
Dry eye
2%
Abdominal pain upper
2%
Hypercholesterolaemia
2%
Rash maculo-papular
2%
Lymphocyte count decreased
2%
Localised infection
2%
Contusion
2%
Fall
2%
Skin abrasion
2%
Blood bilirubin increased
2%
Hepatic enzyme increased
2%
Neutrophil count decreased
2%
Platelet count decreased
2%
Osteoarthritis
2%
Sinus pain
2%
Acne
2%
Blood lactate dehydrogenase increased
2%
Flatulence
2%
Oedema peripheral
2%
Hallucination
2%
Insomnia
2%
Cholecystitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Danicopan
Placebo

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
39,258 Total Patients Enrolled
Alexion Pharmaceuticals, Inc.Lead Sponsor
257 Previous Clinical Trials
41,053 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,344 Total Patients Enrolled
~2 spots leftby Sep 2025
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