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New drug shows promise for rare blood disorder

NCT ID NCT05096403

Disease control Sponsor: Swedish Orphan Biovitrum 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called pegcetacoplan in 24 adults with cold agglutinin disease, a rare condition where the immune system destroys red blood cells. Participants received either the drug or a placebo twice a week for 24 weeks. The goal was to see if the drug could raise hemoglobin levels and reduce the need for blood transfusions.

What this could mean

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

Active substance
Pegcetacoplan (a drug injected under the skin twice a week)
What this could lead to
If it works, this could offer a new treatment option for people with cold agglutinin disease to improve red blood cell levels and reduce the need for blood transfusions.
What could go wrong
This is a small Phase 3 trial with only 24 participants, so results may not apply to everyone. The drug requires lifelong use and carries infection risks due to required vaccinations.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

24 people

The number who actually took part.

Started

Oct 2022

Finished

Sep 2024

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: 1. Age 18 years or older. 2. Diagnosis of primary CAD. 3. Hb level ≤ 9 g/dL. 4. Documented results from bone marrow biopsy within 1 year of screening 5. Either have vaccination against Streptococcus pneumoniae, Neisseria meningitidis (Types A, C, W, Y, and B), and Haemophilus influenzae (Type B) within 2 years prior to screening or agree to receive vaccination during screening. 6. Women of childbearing potential (WOCBP), defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last investigational medicinal product (IMP) dose. 7. Men must agree to the following for the duration of the study and 8 weeks after their last IMP dose: 1. Avoid fathering a child. 2. Use protocol-defined methods of contraception. 3. Refrain from donating sperm. 8. Willing and able to give written informed consent. Exclusion Criteria: 1. Have received other anti-complement therapies (approved or investigational) within 5 half-lives of the agent prior to randomization. 2. Treatment with rituximab monotherapy within 12 weeks prior to randomization, or rituximab combination therapies (e.g., with bendamustine, fludarabine, other cytotoxic drugs or ibrutinib) within 16 weeks prior to randomization. 3. Diagnosis of systemic lupus erythematosus or other autoimmune diseases with antinuclear antibodies. 4. History of an aggressive lymphoma or presence of a lymphoma requiring therapy. 5. Have received an organ transplant. 6. Cold agglutinin syndrome secondary to Mycoplasma pneumoniae, Epstein-Barr virus or other specific causative infection. 7. Presence or suspicion of liver dysfunction as indicated by elevated alanine aminotransferase (ALT) \> 2.5 x upper limit of normal (ULN), or direct bilirubin levels \> 2 x ULN. 8. Inability to cooperate with study procedures.

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Conditions

The condition(s) this trial relates to.

autoimmune hemolytic anemia cold agglutinin disease

As listed by the trial registrant

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

Cold agglutinin disease

Contacts and locations

Locations

  • "LTD Medinvest Institute of Hematology and Transfusiology "

    Tbilisi, Georgia

  • "FOND IRCCS Cà Granda Ospedale Maggiore Policlinico

    Milan, Italy

  • A.O.R.N. S.G. Moscati di Avellino

    Avellino, 83100, Italy

  • AOU Maggiore della Carità SCDU Ematologia

    Novara, Italy

  • ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia

    Brescia, 25123, Italy

  • Aichi Medical University Hospital

    Nagakute, 480-1195, Japan

  • Algemeen Ziekenhuis Klina

    Brasschaat, 2930, Belgium

  • Amsterdam UMC

    Amsterdam, Netherlands

  • Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello

    Palermo, 90146, Italy

  • CHU de Liège

    Liège, 4000, Belgium

  • Cancer Clinical Trials Unit, Haematology -University College London -

    London, United Kingdom

  • Churchill Hospital

    Oxford, United Kingdom

  • East Carolina University Division of Hematology/ Oncology

    Greenville, North Carolina, 27858, United States

  • Fukushima Medical University Hospital

    Fukushima, Japan

  • Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii

    Reggio Calabria, 89133, Italy

  • Haukeland University Hospital

    Bergen, Norway

  • Helsinki University Hospital - Comprehensive Cancer Center

    Helsinki, Finland

  • Hospital Clinic de Barcelona

    Barcelona, Spain

  • Hospital Clínico Universitario de Salamanca

    Madrid, Spain

  • Hospital Universitario Infanta Leonor

    Madrid, Spain

  • Hospital Universitario La Paz

    Madrid, Spain

  • Hospital Universitario Virgen del Rocio

    Seville, Spain

  • Hospital Universitario de Gran Canaria Dr. Negrin

    Las Palmas de Gran Canaria, Spain

  • Hospital Universitario y Politecnico La Fe

    Valencia, Spain

  • Institut f. Transfusionsmedizin - Universität Ulm

    Ulm, Germany

  • Ishikawa Prefectural Central Hospital

    Kanazawa, 9208530, Japan

  • Lakes Research

    Miami Lakes, Florida, 33014, United States

  • Ltd M. Zodelava Hematology Centre

    Tbilisi, Georgia

  • Medical University

    Vienna, Austria

  • Osaka University Hospital

    Osaka, 5650871, Japan

  • Royal London Hospital

    London, E1 2ES, United Kingdom

  • Russell Centre for Clinical Haematology

    Nottingham, NG5 1PB, United Kingdom

  • Semmelweis Egyetem

    Budapest, Hungary

  • Shinshu University Hospital

    Nagano, Japan

  • St James' University Hospital

    Leeds, LS9 7TF, United Kingdom

  • St Olavs Hospital, Avdeling for blodsykdommer

    Trondheim, Norway

  • St. Michael's Hospital

    Toronto, Canada

  • Sykehuset Østfold Kalnes

    Grålum, Norway

  • The Oncology Institute of Hope and Innovation

    Whittier, California, 90603, United States

  • UZ Gasthuisberg

    Leuven, 3000, Belgium

  • University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC)

    Iowa City, Iowa, 52242, United States

  • University of Tsukuba Hospital

    Ibaraki, Japan

  • Universitätsklinikum Essen Klinik f. Hämatologie - Westdeutsches Tumorzentrum

    Essen, 45147, Germany

  • Weill Cornell Medicine / NewYork Presbyterian Hospital

    New York, New York, 10021, United States

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