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Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

(APPELHUS)

Sponsored by Novartis Pharmaceuticals

Quick Self-Assessment

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Current Status

Active, Not Recruiting

Auto-verified from ClinicalTrials.gov

Verified Feb 27, 2026

Phase

Clinical Trial

Sponsor

Novartis Pharmaceuticals

Enrollment Target

50

Start Date

Jan 2022

๐Ÿ“Š
1 of 50 participants interested
2% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

The purpose of this Phase 3 study is to determine whether iptacopan (LNP023) is efficacious and safe for the treatment of aHUS in adult patients who are treatment naive to complement inhibitor therapy.

Who Can Participate

Inclusion Criteria

Adult patients with evidence of active thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury
Vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections are required prior to the start of study treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local regulations, at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination Main

Exclusion Criteria

Treatment with complement inhibitors, including anti-C5 antibody
ADAMTS13 deficiency (\<10% activity or \<0.1U/ml), and/or Shiga toxin-related hemolytic uremic syndrome (STx-HUS), and/or Positive direct C

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

50 participants

๐ŸŽ‚

Age Range

18 Years - 100 Years

โšง

Gender

ALL

๐Ÿข

Sponsor

Novartis Pharmaceuticals

Study Locations

Choose your preferred location or select flexible during enrollment

Unknown

Los Angeles

California

Location available
View Los Angeles location page
Unknown

Los Angeles

California

Location available
View Los Angeles location page
Unknown

Orange

California

Location available
View Orange location page
Unknown

Sacramento

California

Location available
View Sacramento location page
Unknown

Torrance

California

Location available
View Torrance location page
Unknown

Washington D.C.

District of Columbia

Location available
View Washington D.C. location page
Unknown

Miami

Florida

Location available
View Miami location page
Unknown

Boston

Massachusetts

Location available
View Boston location page
Unknown

Minneapolis

Minnesota

Location available
View Minneapolis location page

๐Ÿ“ And 10 more locations available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

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Looking for Atypical Hemolytic Uremic Syndrome Treatment in Birmingham?

Join others in Alabama exploring innovative treatment options through clinical research

Atypical Hemolytic Uremic Syndrome Treatment Options in Birmingham, Alabama

If you're searching for Atypical Hemolytic Uremic Syndrome treatment in Birmingham, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Birmingham, Los Angeles, Los Angeles and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Atypical Hemolytic Uremic Syndrome. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Alabama
Now Enrolling
Up to 50 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Atypical Hemolytic Uremic Syndrome?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Atypical Hemolytic Uremic Syndrome

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Atypical Hemolytic Uremic Syndrome Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT04889430. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.