PNH drug iptacopan tested in Real-World setting – but study cut short
NCT ID NCT07036718
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Novartis Investigative Site
Syktyvkar, Komi, 167904, Russia
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Novartis Investigative Site
Grozny, 364047, Russia
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Novartis Investigative Site
Moscow, 129110, Russia
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Novartis Investigative Site
Murmansk, 183047, Russia
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Novartis Investigative Site
Yuzhno-Sakhalinsk, 693000, Russia