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Complement Inhibitor
Iptacopan for Atypical Hemolytic Uremic Syndrome (APPELHUS Trial)
Phase 3
Recruiting
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Adult patients with evidence of thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks of study treatment
Awards & highlights
APPELHUS Trial Summary
This trial is testing a new drug to see if it is safe and effective for treating a rare kidney disease.
Who is the study for?
Adults with atypical Hemolytic Uremic Syndrome (aHUS) who haven't been treated with complement inhibitors can join. They must have vaccinations against certain infections or take antibiotics if the vaccine was recent. People can't join if they've had certain treatments for aHUS, other kidney diseases, severe infections including COVID-19, or immune system disorders like lupus.Check my eligibility
What is being tested?
The trial is testing Iptacopan's effectiveness and safety in treating aHUS in adults new to complement inhibitor therapy. It's a Phase 3 study which means it’s closer to potentially being approved for general use if successful.See study design
What are the potential side effects?
While specific side effects of Iptacopan are not listed here, similar medications may cause reactions at the injection site, headaches, nausea, or increase the risk of infections due to immune system suppression.
APPELHUS Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have TMA, including low platelet count, blood cell destruction, and kidney issues.
APPELHUS Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 52 weeks of study treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks of study treatment
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Long term safety and efficacy evaluations
Percentage of participants with complete TMA response without the use of PE/PI and anti-C5 antibody
Secondary outcome measures
Change from baseline in chronic kidney disease (CKD) stage
Change from baseline in patient-reported outcomes score as measured by the EuroQol 5-level EQ-5D version (EQ-5D-5L) Questionnaire
Change from baseline in patient-reported outcomes score as measured by the Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Questionnaire
+7 moreAPPELHUS Trial Design
1Treatment groups
Experimental Treatment
Group I: Iptacopan 200 mg b.i.dExperimental Treatment1 Intervention
Single arm open-label with 50 adult patients receiving 200mg oral twice daily doses of iptacopan
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Iptacopan
2021
Completed Phase 1
~40
Find a Location
Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
2,859 Previous Clinical Trials
4,198,194 Total Patients Enrolled
2 Trials studying Atypical Hemolytic Uremic Syndrome
200 Patients Enrolled for Atypical Hemolytic Uremic Syndrome
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have liver disease or injury.I have had a transplant involving bone marrow, heart, lung, small bowel, pancreas, or liver.I have HUS due to drug exposure or specific genetic conditions.I have been on PE/PI treatment for at least 28 days before screening for TMA.My kidney issues are not due to aHUS or chronic failure, nor do I have a family history of genetic kidney diseases not related to the immune system.I do not have severe infections, COVID-19, or a history of serious bacterial infections.I have a very low ADAMTS13 activity, or STx-HUS, or a positive direct Coombs test.I am currently taking medication that affects my immune system.I am on long-term dialysis.I have scleroderma, lupus, or antiphospholipid syndrome.I have received all required vaccinations or will start antibiotics if my treatment begins sooner.I have TMA, including low platelet count, blood cell destruction, and kidney issues.
Research Study Groups:
This trial has the following groups:- Group 1: Iptacopan 200 mg b.i.d
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.
Has Iptacopan been cleared by the FDA?
"Iptacopan is estimated to be a safe medication by our team at Power. This is due to it being in Phase 3 of clinical trials, meaning that not only is there data supporting its efficacy, but also multiple rounds of data affirming its safety."
Answered by AI
What is the past research on Iptacopan?
"Currently, there are 10 different clinical trials studying Iptacopan. Of those active studies, 7 have reached Phase 3. The majority of research is conducted in Praha and Antwerpen; however, 469 locations worldwide are running Iptacopan-based trials."
Answered by AI
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