New pill could replace infusions for rare blood disorder

NCT ID NCT07177859

First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This Phase 3 trial tests NTQ5082, an oral capsule, against the standard infusion eculizumab for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 78 adults who have not had complement inhibitor therapy will be enrolled. The main goal is to see if NTQ5082 can raise hemoglobin levels without needing blood transfusions.

What this could mean

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

Active substance

NTQ5082 capsules

What this could lead to

If successful, NTQ5082 could offer a new oral treatment option for PNH, potentially improving hemoglobin levels without the need for injections.

What could go wrong

This is an early Phase 3 trial with only 78 participants, so results may not apply to all PNH patients. The drug is compared to an existing treatment, and its safety and effectiveness are still being evaluated.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 300020, China

    Contact

    Contact