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Complement Inhibitor

Iptacopan for Paroxysmal Nocturnal Hemoglobinuria (APPULSE Trial)

Augusta, GA
Phase 3
Waitlist Available
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment.
Be older than 18 years old
Must not have
History of cancer of any part of the body within the past 5 years
History of stem cell transplantation or any solid organ transplantation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 168 days
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing a new medication called iptacopan in adults with a blood disorder called PNH. It aims to see if iptacopan is safe and effective. Iptacopan works by stopping the immune system from attacking red blood cells. The medication has shown significant improvement in hemoglobin levels and reduction in hemolysis.

Who is the study for?
Adults over 18 with Paroxysmal Nocturnal Hemoglobinuria (PNH) who've been on a stable anti-C5 antibody treatment for at least 6 months can join. They must not have HIV, be pregnant, or have had recent infections or transfusions. Vaccinations against certain infections are required before starting the trial.
What is being tested?
The trial is testing iptacopan's effectiveness and safety in PNH patients switching from their current treatments (eculizumab/ravulizumab) to iptacopan. It's an open-label study where all participants receive iptacopan for 24 weeks.
What are the potential side effects?
While specific side effects of iptacopan aren't listed here, common ones may include symptoms like headache, nausea, or potential increased risk of infection due to immune system changes.

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 meningitis and pneumonia before starting iptacopan.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had cancer in any part of my body in the last 5 years.
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I have had a stem cell or solid organ transplant.
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I have not had any serious infections, including COVID-19, in the last 14 days.
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I needed a blood transfusion in the last 6 months.
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I have had repeated serious infections like meningitis or pneumonia.
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I am not pregnant, breastfeeding, nor planning to become pregnant during the study.
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I have had a fever of 100.4°F or higher in the last week.
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I am HIV positive.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 168 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 168 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from baseline in Hb levels to demonstrate non-inferiority of iptacopan
Secondary study objectives
Change from baseline in Absolute Reticulocytes count (ARC) levels
Change in Hb levels to demonstrate superiority of iptacopan
Change in fatigue score using FACIT-F questionnaire
+5 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 treatment in adult PNH patientsExperimental Treatment1 Intervention
Subjects will receive iptacopan at a dose of 200 mg b.i.d. orally
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
Paroxysmal Nocturnal Hemoglobinuria (PNH) is commonly treated with complement inhibitors such as eculizumab and ravulizumab, which target the C5 component of the complement system to prevent the destruction of red blood cells. Iptacopan, a Factor B inhibitor, works by inhibiting the alternative pathway of the complement system, thereby reducing hemolysis and thrombosis. These treatments are crucial for PNH patients as they help manage symptoms, reduce the risk of life-threatening complications, and improve quality of life by preventing the excessive breakdown of red blood cells and associated complications.
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Find a Location

Closest Location:Novartis Investigative Site· Augusta, GA

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,916 Previous Clinical Trials
4,253,801 Total Patients Enrolled

Media Library

Iptacopan (Complement Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05630001 — Phase 3
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: Iptacopan treatment in adult PNH patients
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: Iptacopan Highlights & Side Effects. Trial Name: NCT05630001 — Phase 3
Iptacopan (Complement Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05630001 — Phase 3
~20 spots leftby Dec 2025