New drug crovalimab aims to control rare blood disorder PNH

NCT ID NCT04654468

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tests crovalimab in about 50 people with PNH who have not used similar drugs before. PNH causes red blood cells to break apart, leading to fatigue, pain, and need for transfusions. Crovalimab is given as an IV infusion and then as injections under the skin to block part of the immune system and prevent red blood cell destruction. The study measures how well the drug controls hemolysis and reduces transfusion needs over 24 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
Crovalimab (a drug given by IV and injection under the skin)
What this could lead to
If successful, crovalimab could offer a new treatment option to control PNH, reducing red blood cell destruction and the need for blood transfusions.
What could go wrong
This is a single-arm, open-label trial with only 51 participants, so results may not apply broadly. Risks include infections and breakthrough hemolysis.

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

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

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

Contacts and locations

Locations

  • Guangdong General Hospital

    Guangzhou, 510115, China

  • Institute of Hematology and Hospital of Blood Disease

    Tianjin, 300020, China

  • Tianjin Medical University General Hospital

    Tianjin, 300052, China

  • Union Hospital Tongji Medical College Huazhong University of Science and Technology

    Wuhan, 430022, China

  • West China Hospital, Sichuan University

    Chengdu, 610041, China

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