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Experimental drug offered to stem cell transplant patients with rare blood disorder

NCT ID NCT04247906

Disease control Sponsor: Omeros Corporation 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 Results may not be published yet.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
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 This study
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This program provides narsoplimab, an experimental drug, to patients who develop a serious blood clotting condition called thrombotic microangiopathy after a stem cell transplant. The goal is to offer treatment when no other options are available. Patients must have had a stem cell transplant and meet specific criteria to participate.

What this could mean

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

Active substance
narsoplimab
What this could lead to
If successful, this could offer a treatment option for patients with a serious complication after stem cell transplant.
What could go wrong
This is an expanded access program, not a formal trial, so results are uncertain. The drug may not work for everyone and could have side effects.

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.

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.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

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.

* Patient (adult or pediatric) must have undergone allogeneic or autologous hematopoietic stem cell transplant (HSCT) * Patient must be capable of understanding an informed consent form (ICF) or the parent or legal guardian of the patient is able to understand and sign a written ICF prior to the initiation of any treatment procedures. * Patient must have HSCT- TMA defined as having thrombocytopenia and evidence of microangiopathic hemolytic anemia.

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Conditions

The condition(s) this trial relates to.

thrombotic microangiopathy

As listed by the trial registrant

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

Hematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-tma)