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BCX9930 safety checked in PNH patients who benefited from treatment

NCT ID NCT05741346

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This study looked at the long-term safety of BCX9930, an oral medication, for people with paroxysmal nocturnal hemoglobinuria (PNH) who had already benefited from the drug in a previous study. It included 28 participants and aimed to monitor side effects. The study was terminated early, so results are limited.

What this could mean

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

Active substance
BCX9930 (oral medication)
What this could lead to
If successful, this could provide a long-term treatment option for people with PNH who have limited alternatives.
What could go wrong
The study was terminated early and only included 28 people, so results are limited. Long-term safety and effectiveness are not fully established.

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.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

28 people

The number who actually took part.

Started

Jan 2023

Finished

Jan 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.

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: * Male or non-pregnant, non-lactating female participants * Were receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, had benefited from treatment with BCX9930 and would have benefited from continued treatment with BCX9930, and who did not have access to other treatment options Exclusion Criteria: * Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would have interfered with the participant's ability to participate in the study or increased the risk of participation for that participant * An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromised the ability of the participant to continue study-specific procedures or it was considered not to be in the participant's best interest to continue, or benefit-risk assessment was no longer in favor of the participant's continued treatment

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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

  • Investigative Site

    Paris, France

  • Investigative Site

    Budapest, Hungary

  • Investigative Site

    Ampang, Malaysia

  • Investigative Site

    Bloemfontein, South Africa

  • Investigative Site

    Cape Town, South Africa

  • Investigative Site

    Pretoria, South Africa

  • Investigative Site

    Daejeon, South Korea

  • Investigative Site

    Barcelona, Spain

  • Investigative Site

    Valencia, Spain

  • Investigative Site

    Leeds, United Kingdom

  • Investigative Site

    London, United Kingdom

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