ATYPICAL HEMOLYTIC-UREMIC SYNDROME
Clinical trials for ATYPICAL HEMOLYTIC-UREMIC SYNDROME explained in plain language.
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New pill could replace injections for devastating kidney disease
Disease control Recruiting nowThis study is testing if switching adults with atypical hemolytic uremic syndrome (aHUS) from their current injected treatment to a new oral pill called iptacopan is safe and keeps the disease under control. It will enroll about 50 adults who are currently stable on their standar…
Matched conditions: ATYPICAL HEMOLYTIC-UREMIC SYNDROME
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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New hope for controlling rare blood disorder that attacks kidneys
Disease control Recruiting nowThis study is testing how well the drug ravulizumab works to control atypical hemolytic uremic syndrome (aHUS), a rare disease where the immune system attacks blood cells and damages kidneys. The research will follow 20 participants for 26 weeks to see if the treatment improves t…
Matched conditions: ATYPICAL HEMOLYTIC-UREMIC SYNDROME
Phase: PHASE4 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC
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New test aims to solve medical mysteries for undiagnosed patients
Diagnosis Recruiting nowThis study aims to develop and test a new diagnostic tool using RNA sequencing (RNA-Seq) to find the genetic causes of rare diseases. It will enroll about 105 participants, including healthy people, patients with known diagnoses, and patients who remain undiagnosed after standard…
Matched conditions: ATYPICAL HEMOLYTIC-UREMIC SYNDROME
Phase: NA • Sponsor: Mario Negri Institute for Pharmacological Research • Aim: Diagnosis
Last updated Apr 01, 2026 14:41 UTC
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Massive 3,000-Patient study launches to track rare blood disorder
Knowledge-focused Recruiting nowThis is an observational registry study that will follow 3,000 patients diagnosed with atypical Hemolytic-Uremic Syndrome (aHUS), a rare and serious blood disorder. The study aims to collect long-term safety information on patients who are treated with specific medications (eculi…
Matched conditions: ATYPICAL HEMOLYTIC-UREMIC SYNDROME
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:43 UTC
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Researchers map the hidden toll of a devastating rare disease
Knowledge-focused Recruiting nowThis study aims to understand the real-world impact of a rare and serious kidney disease called atypical hemolytic uremic syndrome (aHUS) in Egypt. Researchers will look back at the medical records of up to 200 patients diagnosed over the past 14 years to describe their symptoms,…
Matched conditions: ATYPICAL HEMOLYTIC-UREMIC SYNDROME
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC
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Scientists unlock genetic secrets of rare blood disorder
Knowledge-focused Recruiting nowThis study aims to understand how rare genetic mutations cause atypical Hemolytic Uremic Syndrome (aHUS), a serious blood disorder that damages kidneys. Researchers will study cells from 112 patients with aHUS to learn how specific gene defects affect blood vessel function. The g…
Matched conditions: ATYPICAL HEMOLYTIC-UREMIC SYNDROME
Phase: NA • Sponsor: Mario Negri Institute for Pharmacological Research • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:38 UTC