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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
Must not have
Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 3 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial

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.
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.
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 below
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I have been vaccinated against Neisseria meningitidis as required.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I am not pregnant, breastfeeding, nor planning to become pregnant during the study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Mental disorders
Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores

Side effects data

From 2024 Phase 3 trial • 88 Patients • NCT04469465
13%
Pyrexia
11%
Headache
11%
Diarrhoea
5%
Fatigue
5%
Anaemia
5%
Neutropenia
4%
Dizziness
4%
Asthenia
4%
Dyspnoea
4%
Back pain
4%
Breakthrough haemolysis
4%
Gastroenteritis
2%
Urinary tract infection
2%
Abdominal pain
2%
Contusion
2%
Chromaturia
2%
Dieulafoy's vascular malformation
2%
Insomnia
2%
Vomiting
2%
COVID-19 pneumonia
2%
Staphylococcal sepsis
2%
COVID-19
2%
Aspartate aminotransferase increased
2%
Cough
2%
Arthralgia
2%
Pain in extremity
2%
Thrombocytopenia
2%
Nausea
2%
Chest discomfort
2%
Constipation
2%
Nasopharyngitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Danicopan-Danicopan (TP2)
Placebo-Danicopan (TP2)
Placebo-Danicopan (LTE)
Danicopan-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.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
38,437 Total Patients Enrolled
Alexion Pharmaceuticals, Inc.Lead Sponsor
261 Previous Clinical Trials
140,404 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
36,523 Total Patients Enrolled

Media Library

Danicopan Clinical Trial Eligibility Overview. Trial Name: NCT05389449 — Phase 3
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: Danicopan
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: Danicopan Highlights & Side Effects. Trial Name: NCT05389449 — Phase 3
Danicopan 2023 Treatment Timeline for Medical Study. Trial Name: NCT05389449 — Phase 3
~39 spots leftby Jan 2027