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New pill shows promise for rare blood disorder aHUS

NCT ID NCT04889430

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 07, 2026 · Updated 1 time

Summary

This study tested a new oral medication called iptacopan in 34 adults with a rare disease called atypical hemolytic uremic syndrome (aHUS), which causes blood clots, low platelets, and kidney damage. The goal was to see if the drug could improve blood counts and kidney function without the need for other treatments. Results showed that many patients achieved a complete response, meaning their blood and kidney health improved significantly.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

34 people

The number who actually took part.

Started

Jan 2022

Finished

Apr 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Main 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 Coombs test * Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS * Receiving PE/PI, for 14 days or longer, prior to the start of screening for the current TMA * Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation * Patients with sepsis or active severe systemic bacterial, viral (including COVID-19) or fungal infection, systemic infection which confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease, active infection (or history of recurrent invasive infections) caused by encapsulated bacteria * Kidney disease suggestive of other disease than aHUS or of chronic kidney failure or family history of non-complement mediated genetic kidney disease * Liver disease or liver injury at screening * Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome * Chronic hemo- or peritoneal dialysis Other protocol-defined inclusion/exclusion criteria may apply

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Conditions

The condition(s) this trial relates to.

atypical hemolytic-uremic syndrome thrombotic microangiopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Atypical hemolytic uremic syndrome

Contacts and locations

Locations

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Comprehensive Transplant Ctr at OSU

    Columbus, Ohio, 43210, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Georgetown University Lombardi Cancer Center

    Washington D.C., District of Columbia, 20007-2197, United States

  • Harbor-UCLA Medical Center .

    Torrance, California, 90502, United States

  • Montefiore Medical Center

    The Bronx, New York, 10461, United States

  • Montefiore Medical Center .

    The Bronx, New York, 10461, United States

  • Novartis Investigative Site

    Fortaleza, Ceará, 60430 370, Brazil

  • Novartis Investigative Site

    Brasília, Federal District, 71635-580, Brazil

  • Novartis Investigative Site

    Belo Horizonte, Minas Gerais, 30150-221, Brazil

  • Novartis Investigative Site

    Recife, Pernambuco, 50740-900, Brazil

  • Novartis Investigative Site

    Porto Alegre, Rio Grande do Sul, 90035-074, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 01327 001, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 04038-002, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 05403 000, Brazil

  • Novartis Investigative Site

    Rio de Janeiro, 22270-060, Brazil

  • Novartis Investigative Site

    Salvador, 40323-010, Brazil

  • Novartis Investigative Site

    Beijing, 100034, China

  • Novartis Investigative Site

    Ostrava, Poruba, 708 52, Czechia

  • Novartis Investigative Site

    Nagpur, Maharashtra, 440015, India

  • Novartis Investigative Site

    Pune, Maharashtra, 411011, India

  • Novartis Investigative Site

    Chandigarh, Punjab, 160012, India

  • Novartis Investigative Site

    Lucknow, Uttar Pradesh, 226014, India

  • Novartis Investigative Site

    Iruma-gun, Saitama, 3500495, Japan

  • Novartis Investigative Site

    Seoul, Seoul, 03080, South Korea

  • Novartis Investigative Site

    Taichung, 40447, Taiwan

  • Novartis Investigative Site

    Taoyuan, 33305, Taiwan

  • USC Norris Cancer Center

    Los Angeles, California, 90033, United States

  • UT Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • Univ Cali Irvine ALS Neuromuscular

    Orange, California, 92868, United States

  • Univ of California at Los Angeles

    Los Angeles, California, 90095, United States

  • Univ of California at Sacramento

    Sacramento, California, 95817, United States

  • University Of Miami

    Miami, Florida, 33136, United States

  • University of Cincinnati

    Cincinnati, Ohio, 45267-0585, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.