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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
Must not have
Liver disease or liver injury at screening
Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks of study treatment
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing iptacopan, a new medication, in adults with a rare disease called aHUS. The drug aims to calm an overactive part of their immune system to improve their health.

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.
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.
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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have TMA, including low platelet count, blood cell destruction, and kidney issues.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have liver disease or injury.
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I have had a transplant involving bone marrow, heart, lung, small bowel, pancreas, or liver.
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I have HUS due to drug exposure or specific genetic conditions.
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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.
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I do not have severe infections, COVID-19, or a history of serious bacterial infections.
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I have a very low ADAMTS13 activity, or STx-HUS, or a positive direct Coombs test.
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I am currently taking medication that affects my immune system.
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I am on long-term dialysis.
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I have scleroderma, lupus, or antiphospholipid syndrome.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks of study treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks of study treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Long term safety and efficacy evaluations
Percentage of participants with complete TMA response without the use of PE/PI and anti-C5 antibody
Secondary study objectives
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 more

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: 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
2023
Completed Phase 3
~90

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Complement inhibitors, such as those being studied like iptacopan, target specific components of the complement system to prevent its uncontrolled activation, which is a key factor in the pathogenesis of Atypical Hemolytic Uremic Syndrome (aHUS). By inhibiting parts of the complement pathway, these treatments prevent endothelial damage, hemolysis, and thrombosis, which are critical in reducing kidney injury and improving outcomes for aHUS patients.

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,921 Previous Clinical Trials
4,254,262 Total Patients Enrolled
2 Trials studying Atypical Hemolytic Uremic Syndrome
200 Patients Enrolled for Atypical Hemolytic Uremic Syndrome

Media Library

Iptacopan (Complement Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04889430 — Phase 3
Atypical Hemolytic Uremic Syndrome Research Study Groups: Iptacopan 200 mg b.i.d
Atypical Hemolytic Uremic Syndrome Clinical Trial 2023: Iptacopan Highlights & Side Effects. Trial Name: NCT04889430 — Phase 3
Iptacopan (Complement Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04889430 — Phase 3
~12 spots leftby Dec 2025