Your session is about to expire
← Back to Search
Danicopan Add-On Therapy for Paroxysmal Nocturnal Hemoglobinuria
Phase 3
Waitlist Available
Research Sponsored by Alexion
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 3 years
Awards & highlights
Study Summary
This trial will study the long-term safety and effectiveness of a medicine to treat PNH. It follows earlier trials with the medicine.
Who is the study for?
This trial is for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have finished a previous Alexion study using Danicopan as an extra treatment. They must be able to consent, follow the study rules, and be vaccinated against Neisseria meningitidis. Those with conditions that could affect the study or pose risks can't join, nor can women who are pregnant, breastfeeding, or planning pregnancy.Check my eligibility
What is being tested?
The trial is testing the long-term safety and effectiveness of Danicopan when added to another drug called a C5 inhibitor in patients with PNH. It's a single-arm extension study meaning everyone gets the same treatment without comparison to a placebo or other medication.See study design
What are the potential side effects?
While specific side effects for Danicopan aren't listed here, common issues may include reactions at injection sites, potential risk of infections due to immune system impact, and possibly others based on how it affects complement proteins in blood.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been vaccinated against Neisseria meningitidis as required.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Secondary outcome measures
Change In Absolute Reticulocyte Count
Mental disorders
Change In Hemoglobin Values
+6 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
1Treatment groups
Experimental Treatment
Group I: DanicopanExperimental Treatment1 Intervention
Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Danicopan
2020
Completed Phase 3
~680
Find a Location
Who is running the clinical trial?
AlexionLead Sponsor
246 Previous Clinical Trials
39,182 Total Patients Enrolled
Alexion Pharmaceuticals, Inc.Lead Sponsor
252 Previous Clinical Trials
41,057 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,328 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You took part in a previous study sponsored by Alexion that involved using danicopan along with a C5 inhibitor treatment.I have been vaccinated against Neisseria meningitidis as required.I am not pregnant, breastfeeding, nor planning to become pregnant during the study.
Research Study Groups:
This trial has the following groups:- Group 1: Danicopan
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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.
What is the Danicopan FDA status?
"Danicopan is considered safe based on its status as a Phase 3 trial drug and the rounds of safety data that have been collected."
Answered by AI
Is there a way that I can sign up for this research project?
"Unfortunately, this particular clinical trial is not currently enrolling patients. The trial was first posted on November 1st, 2022 but was last updated on October 21st, 2022. There are, however, 33 other trials that are actively recruiting patients."
Answered by AI
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger