New pill shows promise for rare blood disorder aHUS

NCT ID NCT04889430

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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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

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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