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New chemo combo may lower death risk in child stem cell transplants

NCT ID NCT02349906

Completed Disease control Sponsor: medac GmbH Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug leads to fewer deaths from transplant-related causes in the first 100 days. The approach uses a standard drug (Fludarabine) plus one of the two chemo drugs to prepare the body for donor stem cells.

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Conditions

The condition(s) this trial relates to.

Bone Marrow Failure Disorders bone marrow failure syndrome hemoglobinopathy inborn error of immunity inborn errors of metabolism inherited hemoglobinopathy

As listed by the trial registrant

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

BONE MARROW FAILURE SYNDROMES HAEMOGLOBINOPATHIES INBORN ERRORS OF METABOLISM PRIMARY IMMUNODEFICIENCIES

Contacts and locations

Locations

  • Department of Pediatric Oncology & Hematology, Charite Berlin

    Berlin, 13353, Germany

  • Dzieciecy Szpital Kliniczny im. A. Gebali w Lublinie

    Lublin, 20-093, Poland

  • Hannover Medical University, Dep. of Paediatrics, Paediatric Haematology and Oncology

    Hanover, Germany

  • Heidelberg University Hospital

    Heidelberg, 69120, Germany

  • Oncoematologia Pediatrica A.O. di Perugia Ospedale S. Maria della Misericordia

    Perugia, 06156, Italy

  • Ospedale Bambino Gesu Roma

    Rome, 00165, Italy

  • Ospedale Infantile Regina Margherita Torino

    Turin, 10126, Italy

  • Reparto Trapianti Midollo Osseo Clinica Pediatrica Universitaria Fondazione MBBM-Ospedale San Gerardo

    Monza, 20900, Italy

  • S.C. Oncoematologia Pediatrica Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100, Italy

  • SC Oncoematologia Pediatrica Ospedale Pediatrico Microcitemico "Antonio Cao" A.O. Brotzu

    Cagliari, 09121, Italy

  • Szpital Uniwersytecki im. dr Antoniego Jurasza

    Bydgoszcz, 85-094, Poland

  • U.O. Oncoematologia Pediatrica Azienda Ospedaliero Universitaria Pisana Ospedale S. Chiara

    Pisa, 56100, Italy

  • U.O.C. Oncoematologia Pediatrica Policlinico "G.B. Rossi" - AOUI Verona

    Verona, 37134, Italy

  • UOC Ematologia ed Oncologia Pediatrica con TMO AOU Policlinico Vittorio Emanuele

    Catania, 95123, Italy

  • Ulm, University Hospital, Clinic for Children and Adolescents

    Ulm, 89075, Germany

  • University Children's Hospital Essen Pediatric stem cell transplantation

    Essen, 45122, Germany

  • University Hospital Frankfurt

    Frankfurt am Main, 60590, Germany

  • University Hospital Motol, Dep. of Paediatric Haematology and Oncology

    Prague, Czechia

  • University of Jena, Department of Pediatrics

    Jena, 07747, Germany

  • Uniwersytecki Szpital Dzieciecy w Krakowie

    Krakow, 30-663, Poland

  • Wroclaw Medical University, Department of Pediatric Hematology/Oncology and BMT

    Wroclaw, 50-368, Poland

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