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New hope for PNH patients with stubborn anemia

NCT ID NCT07187401

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 Jul 10, 2026 · Updated 2 times

Summary

This study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive either the drug or a placebo, and researchers will monitor side effects and blood levels.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2026

Expected to finish

Jul 2031

An estimate. End dates often move.

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.

Key Inclusion Criteria: 1. Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing 2. Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit, as described in the protocol 3. Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol 4. Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1 Key Exclusion Criteria: 1. Has history of bone marrow transplantation or receipt of an organ transplant 2. Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms 3. Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period 4. Has laboratory evidence of bone marrow failure, as described in the protocol 5. Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol NOTE: Other Protocol Defined Inclusion / Exclusion Criteria Apply

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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, PNH Persistent anemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    8 sites in 3 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Kings College Hospital NHS Foundation Trust

    RECRUITING

    London, SE5 9RS, United Kingdom

  • Samsung Medical Center

    RECRUITING

    Seoul, 6351, South Korea

  • Seoul National University Hospital

    RECRUITING

    Seoul, 3080, South Korea

  • Severance Hospital

    RECRUITING

    Seoul, 3722, South Korea

  • St. James's University Hospital

    RECRUITING

    Leeds, West Yorkshire, LS97TF, United Kingdom

  • St. Vincent Hospital - The Catholic University of Korea

    RECRUITING

    Suwon, Gyeonggi-do, 16247, South Korea

  • The Catholic University of Korea, Seoul St. Mary's Hospital

    RECRUITING

    Seoul, 06591, South Korea

  • Toronto General Hospital

    RECRUITING

    Toronto, Ontario, M5G 2C4, Canada

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