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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.
Must not have
History of hematopoietic stem cell transplantation
History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 60 months
Awards & highlights
No Placebo-Only Group
Pivotal Trial

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.
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.
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
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I have been vaccinated against meningitis, pneumonia, and Haemophilus influenzae.
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I am over 18 and have finished specific iptacopan study phases for PNH without reducing dosage.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had a bone marrow transplant.
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I have had repeated serious infections like meningitis or pneumonia.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Proportion of participants with adverse events
Secondary study objectives
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

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: 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
2023
Completed Phase 3
~90

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,921 Previous Clinical Trials
4,254,062 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
~111 spots leftby Oct 2027
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