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Complement Inhibitor

Iptacopan for Blood Diseases

Phase 2
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
Participants with a diagnosis of persistent or chronic primary ITP
Sustained anemia
Timeline
Screening 3 weeks
Treatment Varies
Follow Up screening, baseline, day 1, day 15, day 29, day 85, day 99, day 113
Awards & highlights

Study Summary

This trialis testing a new drug to treat blood disorders like low platelet count or cold agglutinin disease.

Who is the study for?
This trial is for adults with certain blood disorders like low platelet count and autoimmune hemolytic anemia who've had at least one prior treatment. They must weigh over 35 kg, have updated vaccinations against specific infections, and give written consent. Pregnant women or those able to become pregnant using no contraception are excluded, as well as individuals with severe bleeding history, liver disease, cancer within the last 5 years (except some cases), organ transplants, immune deficiencies including HIV/AIDS, chronic hepatitis B or C infection.Check my eligibility
What is being tested?
The study tests Iptacopan's effectiveness and safety in treating autoimmune benign hematological disorders. It aims to see if this medication can help manage conditions that affect blood cells such as primary immune thrombocytopenia and cold agglutinin disease by reducing symptoms like low platelet counts and anemia caused by these diseases.See study design
What are the potential side effects?
While not explicitly listed in the provided information, common side effects of drugs similar to Iptacopan may include headache, nausea, diarrhea or constipation. There could also be a risk of infections due to its effect on the immune system.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with long-term primary ITP.
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I have long-term low red blood cell counts.
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I have been diagnosed with coronary artery disease.
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I weigh at least 35 kg.
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I have been treated for ITP with at least one type of therapy.
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I have had at least one treatment for coronary artery disease.
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I am vaccinated against meningitis and pneumonia, and it's recommended I get the flu shot before treatment starts.
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I have CAD, received at least one treatment for it, and I have ongoing hemolysis and anemia.
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I weigh at least 35 kg.
Select...
I have chronic ITP and have tried at least one treatment for it.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~screening, baseline, day 1, day 15, day 29, day 85, day 99, day 113
This trial's timeline: 3 weeks for screening, Varies for treatment, and screening, baseline, day 1, day 15, day 29, day 85, day 99, day 113 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Secondary outcome measures
Haptoglobin
Hemoglobin levels
Lactate dehydrogenase (LDH)
+10 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Iptacopan 200 mg BIDExperimental Treatment1 Intervention
Iptacopan 200 mg BID
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
2,851 Previous Clinical Trials
4,197,570 Total Patients Enrolled

Media Library

Iptacopan (Complement Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05086744 — Phase 2
Cold Agglutinin Disease Research Study Groups: Iptacopan 200 mg BID
Cold Agglutinin Disease Clinical Trial 2023: Iptacopan Highlights & Side Effects. Trial Name: NCT05086744 — Phase 2
Iptacopan (Complement Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05086744 — Phase 2

Frequently Asked Questions

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

Has the FDA granted authorization for Iptacopan 200 mg BID?

"The safety of Iptacopan 200 mg BID is estimated to be a 2 as this Phase 2 trial has yielded evidence regarding its security, yet no data that can attest to its effectiveness."

Answered by AI

How many subjects are receiving the experimental treatment in this clinical experiment?

"Affirmative, the clinicaltrials.gov page indicates that this research is actively enrolling participants. It was posted on December 21st 2021 and recently revised November 30th 2022. Thirty individuals are required for the single site trial."

Answered by AI

Does this clinical trial represent a pioneering effort?

"Since 2019, Novartis Pharmaceuticals has been researching the effects of Iptacopan 200 mg BID. After its initial trial in which 95 participants were enrolled, this medication received Phase 2 drug approvals and is currently being studied by 10 active teams present in 169 cities and 39 nations."

Answered by AI

Are there any opportunities for volunteers to participate in this research project?

"Based on the information available through clinicaltrials.gov, this research study is still accepting participants. The trial was initially posted in December of 2021 and most recently amended on November 30th 2022."

Answered by AI

Who else is applying?

What state do they live in?
Pennsylvania
What site did they apply to?
Massachusetts General Hospital
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
3+

How responsive is this trial?

Most responsive sites:
  1. Massachusetts General Hospital: < 24 hours
Average response time
  • < 1 Day
Typically responds via
Phone Call
Email
~6 spots leftby Mar 2025