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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

PNH Trial Summary

This trial will test if the drug danicopan can help improve anemia in people with PNH when given with eculizumab. Danicopan doses will be increased up to 200 mg three times daily based on how well the participant tolerates it and how effective it is.

Eligible Conditions

Paroxysmal Nocturnal Hemoglobinuria

PNH Trial 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)

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change From Baseline In Hemoglobin At Week 24

Secondary outcome measures

Change From Baseline In Lactate Dehydrogenase At Week 24

Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Adverse Events (AEs), And Events Leading To Discontinuation Of Study Drug

Number Of Participants Without RBC Transfusions During 24 Weeks Of Treatment

+1 more

Side effects data

From 2024 Phase 3 trial • 86 Patients • NCT04469465

11%

Headache

Nausea

Diarrhoea

Arthralgia

Pain in extremity

Alanine aminotransferase increased

Hypertension

Vomiting

Pyrexia

White blood cell count decreased

Myalgia

Aspartate aminotransferase increased

Constipation

Urinary tract infection

Viral infection

Oropharyngeal pain

Rhinorrhoea

Haemolysis

Noninfective gingivitis

Post procedural diarrhoea

Splenomegaly

Upper respiratory tract infection

Flatulence

Hypercholesterolaemia

Chromaturia

Neutropenia

Pancreatitis

Neutrophil count decreased

Oedema peripheral

Hepatic function abnormal

Sinusitis

Flushing

Platelet count decreased

Decreased appetite

Liver disorder

Hot flush

Cholecystitis

Leukopenia

Anaemia

Dry eye

Abdominal pain upper

Dyspepsia

Stomatitis

Jaundice

Localised infection

Cellulitis

Contusion

Blood bilirubin increased

Blood pressure increased

Bone pain

Osteoarthritis

Acne

Rash maculo-papular

Discoloured vomit

Blood lactate dehydrogenase increased

Lymphocyte count decreased

SARS-CoV-2 test positive

Chest discomfort

Hepatic enzyme increased

Fatigue

COVID-19

Proteinuria

Cough

Abdominal discomfort

Fall

Febrile nonhaemolytic transfusion reaction

Post procedural contusion

Post procedural haemorrhage

Skin abrasion

Thoracic vertebral fracture

Dizziness

Lethargy

Hallucination

Insomnia

Epistaxis

Sinus pain

Salivary gland pain

Peripheral swelling

100%

80%

60%

40%

20%

Study treatment Arm

Danicopan

Placebo

PNH 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

2021

Completed Phase 4

~1190

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

AlexionLead Sponsor

246 Previous Clinical Trials

39,265 Total Patients Enrolled

Alexion Pharmaceuticals, Inc.Lead Sponsor

252 Previous Clinical Trials

41,140 Total Patients Enrolled

Alexion PharmaceuticalsLead Sponsor

230 Previous Clinical Trials

37,411 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the most recent findings from research on Danicopan?

"There are a total of 16 clinical trials testing the efficacy of Danicopan. Of these, 9 have reached Phase 3. The majority of trial locations for this treatment are in Haifa and Malaga; however, there are 756 centres running trials globally."

Answered by AI

How many people are included in the experimental group for this research project?

"This study wrapped up recruitment as of August 15th, 2022. It was first posted on May 8th, 2018. For those looking for other opportunities, there are 33 trials actively recruiting patients with hemoglobinuria and 16 studies still enrolling participants for Danicopan treatment."

Answered by AI

Is this a clinical trial that is testing something completely new?

"The development of Danicopan began in 2016 with a clinical trial sponsored by Alexion Pharmaceuticals. This first study included 270 patients and, after successful completion, led to the drug's Phase 3 approval. Currently, there are 16 active studies involving Danicopan being conducted across 198 different cities in 44 countries worldwide."

Answered by AI

When was Danicopan cleared by the FDA?

"Danicopan is a medication that, while Phase 2 trial data only supports its safety and not efficacy, our team at Power rates as a 2."

Answered by AI

Are people currently signing up to participate in this trial?

"The clinicaltrials.gov website indicates that this study is not currently looking for new participants. Although the last update was on August 15th, 2022, the trial has not been recruiting since May 8th, 2018. There are other 49 ongoing trials that are actively seeking candidates right now."

Answered by AI

Does this experiment allow elderly volunteers?

"The age criteria for participants in this study is that they must be over 18 and under 65."

Answered by AI

Are there any restrictions for volunteers in this experiment?

"This study, which is looking for 12 participants in total, requires candidates to have hemoglobinuria and be of legal age (18 years or older)."

Answered by AI

What are the primary medical conditions that Danicopan is used to improve?

"Danicopan is a medication that is used to treat myasthenia gravis, neuromyelitis optica, paroxysmal nocturnal haemoglobinuria (pnh), and thyroid stimulating immunoglobulins."

Answered by AI

Recent research and studies

BloodJournal

Phase 2 Study of Danicopan in Patients with Paroxysmal Nocturnal Hemoglobinuria with an Inadequate Response to Eculizumab

Kulasekararaj, Austin G., Antonio M. Risitano, Jaroslaw P. Maciejewski, Rosario Notaro, Peter Browett, Jong Wook Lee, Mingjun Huang, Michael Geffner, and Robert A. Brodsky. 2021. “Phase 2 Study of Danicopan in Patients with Paroxysmal Nocturnal Hemoglobinuria with an Inadequate Response to Eculizumab”. Blood. American Society of Hematology. doi:10.1182/blood.2021011388.

BloodJournal

Phase 2 Study of Danicopan in Patients with Paroxysmal Nocturnal Hemoglobinuria with an Inadequate Response to Eculizumab

clinicaltrials.govStudy

Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

2018. "Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03472885.