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

NCT ID NCT07036718

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Aug 18, 2026 · Updated 5 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.

Study facts

What this study's own registry entry says, in plain language.

Participants

6 people

The number who actually took part.

Started

Jul 2025

Finished

Dec 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Enrollment of female and male patients with PNH

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Age ≥18 years at the start of iptacopan therapy. 2. The patient is undergoing treatment with iptacopan. 3. The treating physician decided to prescribe iptacopan based on the Summary of Product Characteristics during routine clinical practice, regardless of study participation. 4. Provision of written informed consent. Exclusion Criteria: 1\. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.

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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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