Your session is about to expire
← Back to Search
Complement Inhibitor
Danicopan + C5 Inhibitor for Paroxysmal Nocturnal Hemoglobinuria
Phase 3
Waitlist Available
Research Sponsored by Alexion Pharmaceuticals, Inc.
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 24 weeks prior to initiation of treatment to post 24 weeks of treatment
Awards & highlights
Study Summary
This trial will study whether danicopan, when used in addition to a C5 inhibitor, is effective in treating participants with PNH who have clinically evident EVH.
Who is the study for?
This trial is for people with PNH who have low red blood cell counts and high reticulocyte counts despite being on a C5 inhibitor like eculizumab or ravulizumab for at least 6 months. They must not have had major organ or stem cell transplants, other bone marrow issues needing transplant, complement deficiencies, HIV, hepatitis B/C infections, certain liver problems or severe kidney disease.Check my eligibility
What is being tested?
The study tests if adding danicopan to standard treatment with a C5 inhibitor can better control red blood cell destruction outside of blood vessels in PNH patients. Participants will either receive danicopan or a placebo alongside their current C5 inhibitor therapy.See study design
What are the potential side effects?
Potential side effects of danicopan may include reactions where the drug is given, increased risk of infections due to immune system suppression, possible liver function changes and potential impact on kidney function.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 24 weeks prior to initiation of treatment to post 24 weeks of treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 weeks prior to initiation of treatment to post 24 weeks of treatment
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change From Baseline in Hgb at Week 12
Secondary outcome measures
Change From Baseline FACIT Fatigue Scores at Week 24
Change From Baseline in Absolute Reticulocyte Count at Week 12
Change From Baseline in C3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
+15 moreSide effects data
From 2024 Phase 3 trial • 86 Patients • NCT0446946511%
Headache
9%
Nausea
7%
Diarrhoea
7%
Arthralgia
5%
Pain in extremity
5%
Alanine aminotransferase increased
5%
Hypertension
5%
Vomiting
5%
Pyrexia
4%
White blood cell count decreased
4%
Myalgia
4%
Aspartate aminotransferase increased
4%
Constipation
4%
Urinary tract infection
4%
Viral infection
4%
Oropharyngeal pain
4%
Rhinorrhoea
4%
Haemolysis
2%
Noninfective gingivitis
2%
Post procedural diarrhoea
2%
Splenomegaly
2%
Neutropenia
2%
Upper respiratory tract infection
2%
Flatulence
2%
Hypercholesterolaemia
2%
Chromaturia
2%
Pancreatitis
2%
Neutrophil count decreased
2%
Oedema peripheral
2%
Hepatic function abnormal
2%
Sinusitis
2%
Flushing
2%
Platelet count decreased
2%
Decreased appetite
2%
Liver disorder
2%
Hot flush
2%
Cholecystitis
2%
Leukopenia
2%
Anaemia
2%
Dry eye
2%
Abdominal pain upper
2%
Dyspepsia
2%
Stomatitis
2%
Jaundice
2%
Localised infection
2%
Cellulitis
2%
Contusion
2%
Blood bilirubin increased
2%
Blood pressure increased
2%
Bone pain
2%
Osteoarthritis
2%
Acne
2%
Rash maculo-papular
2%
Discoloured vomit
2%
Blood lactate dehydrogenase increased
2%
Lymphocyte count decreased
2%
SARS-CoV-2 test positive
2%
Chest discomfort
2%
Hepatic enzyme increased
2%
Fatigue
2%
COVID-19
2%
Proteinuria
2%
Cough
2%
Abdominal discomfort
2%
Fall
2%
Febrile nonhaemolytic transfusion reaction
2%
Post procedural contusion
2%
Post procedural haemorrhage
2%
Skin abrasion
2%
Thoracic vertebral fracture
2%
Dizziness
2%
Lethargy
2%
Hallucination
2%
Insomnia
2%
Epistaxis
2%
Sinus pain
2%
Salivary gland pain
2%
Peripheral swelling
100%
80%
60%
40%
20%
0%
Study treatment Arm
Danicopan
Placebo
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Danicopan + C5 InhibitorExperimental Treatment2 Interventions
Participants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2).
Group II: Placebo + C5 InhibitorPlacebo Group3 Interventions
Participants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Danicopan
2020
Completed Phase 3
~680
C5 Inhibitor
2020
Completed Phase 3
~90
Find a Location
Who is running the clinical trial?
Alexion Pharmaceuticals, Inc.Lead Sponsor
252 Previous Clinical Trials
41,051 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,322 Total Patients Enrolled
AlexionLead Sponsor
246 Previous Clinical Trials
39,176 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have a condition called aplastic anemia or other bone marrow failure that needs treatment like a bone marrow transplant or certain medications.You have a known or suspected problem with your immune system called complement deficiency.You have low red blood cell count and high reticulocyte count.You have a low number of a type of white blood cells called neutrophils.Your alanine aminotransferase levels are higher than the normal range, especially if you have high levels of iron in your liver.You have a current infection of HIV, hepatitis B, or active hepatitis C.You have been diagnosed with paroxysmal nocturnal hemoglobinuria (PNH).You have been taking a certain type of medication called a C5 inhibitor for at least 6 months before the start of the study.You need to have at least 30,000 platelets per microliter of blood.You currently have a condition causing bile flow problems.Your kidney function is very low, and you may need dialysis.Your direct bilirubin levels are higher than two times the upper limit of normal, unless it is due to a specific condition or syndrome documented by a doctor.
Research Study Groups:
This trial has the following groups:- Group 1: Danicopan + C5 Inhibitor
- Group 2: Placebo + C5 Inhibitor
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Can you give me an estimate of how many different hospitals are participating in this research project?
"Currently, this clinical trial is enrolling at 10 sites. These locations include Kansas City, Toronto, and Durham. To minimize the inconvenience of travel for enrolled patients, it may be best to select a site nearest you."
Answered by AI
Are participants still being recruited for this research project?
"No, this study is not looking for participants at the moment. According to information on clinicaltrials.gov, the last update was on September 30th, 2022 and the study was first posted December 16th, 2020. There are other medical trials actively recruiting patients right now though."
Answered by AI
Could you please list any other C5 Inhibitor medical trials that have been completed?
"There are currently 3 different clinical trials underway for C5 Inhibitors. One is in Phase 3 while the others are still in earlier stages. Out of the 165 total locations, several Naples, Ohio facilities are running these tests."
Answered by AI
What is the FDA's stance on C5 Inhibitor?
"C5 Inhibitor is at a Phase 3 in terms of testing, which indicates that while there is some data supporting efficacy, multiple rounds of data have been collected to show its safety."
Answered by AI
Have patients undergone this treatment before?
"3 clinical trials testing C5 Inhibitors are currently underway in 96 cities and 21 countries. The first study began in 2018, 12 patients were enrolled, and the trial completed Phase 2 drug approval in that same year. Since then, a total of 18303 studies have been completed."
Answered by AI
How many test subjects are a part of this research?
"Unfortunately, this study is not currently seeking candidates. Although, it's important to note that the trial was first posted on December 16th 2020 and was edited most recently on September 30th 2022. There are other ongoing studies which may be of interest; at present, there are 68 clinical trials actively recruiting participants with hemoglobinuria and 3 C5 Inhibitor studies also seeking patients."
Answered by AI
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger