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PNH drug iptacopan tested in Real-World setting – but study cut short

NCT ID NCT07036718

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

First seen Jun 24, 2026 · Last updated Jul 23, 2026 · Updated 3 times

Summary

This study looked at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) when used in everyday medical practice in Russia. Only 6 adults took part, and the study was terminated early. Researchers measured whether patients' hemoglobin levels improved and if they needed fewer blood transfusions over 6 to 12 months.

What this could mean

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

Active substance
iptacopan
What this could lead to
If successful, this could show that iptacopan helps control PNH symptoms and reduces the need for blood transfusions in everyday medical practice.
What could go wrong
This was a very small, early-terminated observational study with only 6 people, so results are limited and may not apply to broader populations. No firm conclusions can be drawn.

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.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Contacts and locations

Locations

  • Novartis Investigative Site

    Syktyvkar, Komi, 167904, Russia

  • Novartis Investigative Site

    Grozny, 364047, Russia

  • Novartis Investigative Site

    Moscow, 129110, Russia

  • Novartis Investigative Site

    Murmansk, 183047, Russia

  • Novartis Investigative Site

    Yuzhno-Sakhalinsk, 693000, Russia

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