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Complement Inhibitor

Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

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
Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 60 months
Awards & highlights

Study Summary

This trialevaluates the long-term safety, tolerability, and effectiveness of a drug, iptacopan, for people with a rare blood disorder, PNH.

Who is the study for?
This trial is for adults over 18 with Paroxysmal Nocturnal Hemoglobinuria (PNH) who've been stable on iptacopan for at least 3 months after completing certain Phase 2 or 3 trials. They must have had vaccinations against specific infections. Those with recurrent invasive infections, serious health conditions that could increase risk, or a history of stem cell transplantation cannot join.Check my eligibility
What is being tested?
The study tests the long-term safety and effectiveness of iptacopan in PNH patients. It's an open-label, single-arm extension study providing continued access to iptacopan for those who completed prior Novartis-sponsored studies.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions similar to previous phases such as headaches, nausea, and increased risk of infections due to the nature of immunomodulatory treatments.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been vaccinated against meningitis, pneumonia, and Haemophilus influenzae.
Select...
I am over 18 and have finished specific iptacopan study phases for PNH without reducing dosage.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~60 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 60 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Proportion of participants with adverse events
Secondary outcome measures
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Proportion of participants who remain free from transfusions
Proportion of participants with Major Adverse Vascular Events MAVEs
+1 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: IptacopanExperimental Treatment1 Intervention
Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy
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
2,858 Previous Clinical Trials
4,197,593 Total Patients Enrolled

Media Library

Iptacopan (Complement Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04747613 — Phase 3
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: Iptacopan
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: Iptacopan Highlights & Side Effects. Trial Name: NCT04747613 — Phase 3
Iptacopan (Complement Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04747613 — Phase 3

Frequently Asked Questions

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

How many enrollees does this clinical trial have at the moment?

"The public clinicaltrial.gov database contains information that this trial is still recruiting patients. The first posting was on 7/27/2021, with the most recent update on 11/7/2022. There are currently 167 people enrolled at 2 different sites."

Answered by AI

Are we still looking for volunteers for this experiment?

"That is correct, the clinicaltrials.gov website has information indicating that this trial is looking for subjects. The trial was posted on 7/27/2021 and updated on 11/7/20202. A total of 167 individuals are needed for the study across 2 locations."

Answered by AI

Have similar medical trials been conducted before?

"There are 10 ongoing clinical trials for Iptacopan in 39 countries and 169 cities. The first study was sponsored by Novartis Pharmaceuticals in 2019. That initial trial had 95 participants and completed Phase 2 drug approval. In the years since 2019, 5 more studies have been concluded."

Answered by AI

What does the scientific community know about Iptacopan from other research?

"Currently, there are 10 different clinical trials underway that are studying Iptacopan. Of these 10 trials, 7 have progressed to Phase 3 testing. Most of the research pertaining to Iptacopan is based in Bologna, BO; however, 471 total locations around the world are conducting trials for this medication."

Answered by AI

Iptacopan, is it possible to get this medication approved by the FDA?

"There is some evidence from past clinical trials to support the efficacy of Iptacopan, as well as multiple rounds of data that suggest it is safe for use. Therefore, we have given it a score of 3."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
2021. "Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04747613.
~143 spots leftby Jan 2028
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