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Complement Inhibitor
Iptacopan for Paroxysmal Nocturnal Hemoglobinuria (APPULSE 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
Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment.
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 168 days
Awards & highlights
APPULSE Trial Summary
This trial studies the safety and effectiveness of an experimental drug to replace current treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH).
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.Check my eligibility
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.See study design
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.
APPULSE Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been vaccinated against meningitis and pneumonia before starting iptacopan.
APPULSE Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 168 days
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 168 days
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 Hb levels to demonstrate non-inferiority of iptacopan
Secondary outcome measures
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 moreAPPULSE 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
2021
Completed Phase 1
~40
Find a Location
Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
2,860 Previous Clinical Trials
4,198,259 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been on a stable dose of eculizumab or ravulizumab for at least 6 months.I have had cancer in any part of my body in the last 5 years.I am 18 or older and have been diagnosed with PNH by my doctor.I have had a stem cell or solid organ transplant.I have been vaccinated against meningitis and pneumonia before starting iptacopan.I have not had any serious infections, including COVID-19, in the last 14 days.I have been vaccinated against Haemophilus influenzae, if it was recommended and available.I needed a blood transfusion in the last 6 months.I have had repeated serious infections like meningitis or pneumonia.I do not have any unstable health conditions like heart issues or active bleeding.I am not pregnant, breastfeeding, nor planning to become pregnant during the study.I have had a fever of 100.4°F or higher in the last week.Your average hemoglobin level is at least 10 grams per deciliter.I am HIV positive.
Research Study Groups:
This trial has the following groups:- Group 1: Iptacopan treatment in adult PNH patients
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.
Is this research project currently accepting new participants?
"That is correct. According to clinicaltrials.gov, this medical trial that was first launched on April 24th 2023 is still actively recruiting participants from a single site. So far, 50 people have signed up for the study."
Answered by AI
What is the maximum capacity of participants for this trial?
"Indeed. According to clinicaltrials.gov, this research program is looking for volunteers; it was initially started on April 24th 2023 and last modified May 4th of the same year. Around 50 patients need to be recruited from a single medical centre."
Answered by AI
What potential hazard is posed to adult PNH patients when undergoing Iptacopan treatment?
"Our experts at Power have assessed the security of Iptacopan among adult PNH patients as a 3, due to its Phase 3 categorisation. This means that there is evidence demonstrating both effectiveness and safety following multiple rounds of testing."
Answered by AI
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