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New drug combo shows promise for rare bone marrow failure

NCT ID NCT02099747

Disease control Sponsor: European Society for Blood and Marrow Transplantation Source: ClinicalTrials.gov ↗

What the study statuses mean

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This study tested whether adding eltrombopag to standard immunosuppressive therapy improves early recovery in patients with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. 202 participants received either standard treatment (horse antithymocyte globulin plus cyclosporine) or the same treatment plus eltrombopag. The main goal was to see if more patients achieved a complete blood count response within three months.

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

202 people

The number who actually took part.

Started

Jul 2015

Finished

Dec 2020

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

Who can take part

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

Ages

15 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. Diagnosis of severe or very severe aplastic anemia, defined by \[29\]: * At least two of the following: * Absolute neutrophil counts \<0.5 x 109/L (severe) or \<0.2 x 109/L (very severe) * Platelet counts \<20 x 109/L * Reticulocyte counts \<60 x 109/L * Hypocellular bone marrow (\<30% cellularity), without evidences of fibrosis or malignant cells 2. Male or female age \> 14 years; 3. Written informed consent 4. Willing and able to comply with all of the requirements and visits in the protocol 5. Understands that they can be randomised to either treatment arm 6. Negative pregnancy test for women of child bearing age 7. Written acceptance to use contraception (hormonal or barrier method of birth control; abstinence) for the entire duration of study participation. Exclusion Criteria: 1. Prior immunosuppressive therapy with ATG (horse of rabbit) or any other lymphocyte depleting agent (i.e., alemtuzumab) 2. Eligibility to a sibling allogeneic stem cell transplantation 3. Evidence of a myelodysplastic syndrome, defined by the presence of myelodysplastic features, excess of blasts or karyotypic abnormalities typical of MDS (according to revised WHO 2008 criteria) \[30\],, as well as other primitive marrow disease. Patients with diagnosis of AA with cytogenetic abnormalities which are recurrent in MDS (according to revised WHO 2008 criteria) \[30\] should be included in this category, and are not eligible for the study; patients with del(20q), +8 and -Y are not included in this category, and thus are eligible for this study. The list of karyotypic abnormalities which qualifies for the diagnosis of MDS are listed in the Appendix. 4. History or clinical suspect of constitutional aplastic anemia (i.e. Fanconi Anemia with positive DEB/MMC test or Dyskeratosis Congenita) 5. History of malignant tumors with active disease within 5 years from enrollment, and/or previous chemo-radiotherapy 6. Previous history of stem cell transplantation 7. Treatment with cyclosporin A unless * \<4 weeks of cyclosporin A treatment before enrolement and * wash out period of 2 weeks before enrollment 8. CMV viremia, as defined by positive PCR or pp65 test 9. WHO performance status ≥3 10. Pregnant or breast feeding patients 11. Patients with hepatic, renal or cardiac failure, or any other life- threatening concurrent disease 12. Patients with HIV infection 13. Patients without social health care assistance 14. Participation in another clinical trial within 1 month before the start of this trial 15. Patients and/or female partners of male patients not using highly effective method of birth control i.e. intrauterine device (IUD), hormonal (oral pill, injection, implants), tubal ligation or partner's vasectomy 16. subjects with known hypersensitivity to any of the component medications The presence of a Paroxysmal Nocturnal Hemoglobinuria clone is not an exclusion criterion.

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia

As listed by the trial registrant

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

Severe aplastic anemia

Contacts and locations

Locations

  • 'Federico II' Medical School

    Naples, Italy

  • AMC

    Amsterdam, Netherlands

  • AOU Città della Salute e della Scienza di Torino

    Turin, Italy

  • Azienda Ospedaliera Papa Giovanni XXIII

    Bergamo, Italy

  • Centre Hospitalier Lyon-Sud

    Lyon, France

  • City Hospital

    Nottingham, United Kingdom

  • Donostia Hospital

    Donostia / San Sebastian, Spain

  • Fondazione IRCCS ca Granda Ospedale

    Milan, Italy

  • Hopital Jean Minjoz

    Besançon, France

  • Hospital La Fe

    Valencia, Spain

  • Hospital Universitari Germans Trias I Pujol

    Badalona, Spain

  • Hôpital Haut-Lévèque

    Bordeaux, France

  • Hôpital Huriez

    Lille, France

  • Hôpital Purpan

    Toulouse, France

  • Institut Català d'Oncologia - Hospital Duran i Reynals

    Barcelona, Spain

  • Istituto G. Gaslini children's Hospital

    Genova, Italy

  • King's College Hospital

    London, United Kingdom

  • La Sapienza University Hospital

    Rome, Italy

  • Leiden University Medical Center

    Leiden, Netherlands

  • Pontchaillou Hospital

    Rennes, France

  • San Martino Hospital

    Genova, Italy

  • St. Bartholomew's Hospital

    London, United Kingdom

  • St. James Hospital

    Leeds, United Kingdom

  • St. Louis Hospital

    Paris, France

  • UMCG

    Groningen, Netherlands

  • UMCU

    Utrecht, Netherlands

  • University Hospital Basel

    Basel, Switzerland

  • University Hospital Bern

    Bern, Switzerland

  • University Hospital Zürich

    Zurich, Switzerland

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