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

NCT ID NCT02349906

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

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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 Results may not be published yet.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
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Status unknown
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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.

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

106 people

The number who actually took part.

Start date

Apr 2015

Finished

Feb 2023

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

28 days to 17 years

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. Non-malignant disease indicated for first myeloablative allogeneic HSCT, including inborn errors of metabolism, primary immunodeficiencies, haemoglobinopathies and bone marrow failure syndromes. 2. First allogeneic HSCT. 3. Available matched sibling donor (MSD), matched family donor (MFD) or matched unrelated donor (MUD). For bone marrow (BM) and peripheral blood (PB) match is defined as at least 9/10 allele matches after four digit typing in human leucocyte antigen (HLA)-A, -B, -C, -DRB1 and DQB1 antigens. For umbilical cord blood (UCB) match is defined as at least 5/6 matches after two digit typing in HLA-A and -B and four digit typing in DRB1 antigens. Exclusion Criteria: 1. Second or later HSCT. 2. HSCT from mismatched donor (less than 9/10 BM/peripheral blood stem cells (PBSC) or less than 5/6 matched cord donor). 3. Preterm newborn infants (\<37 weeks gestational age) and term newborn infants aged 0 - 27 days at time of registration. 4. Obese paediatric patients with body mass index weight (kg)/\[height (m)\]² \> 30 kg/m². 5. Diagnosis of Fanconi anaemia and other chromosomal breakage disorders, radiosensitivity disorders (deoxyribonucleic acid (DNA) Ligase 4, Cernunnos- X-ray repair cross-complementing protein 4 (XRCC4) like factor (XLF), Nijmegen Breakage Syndrome (NBS)) and Dyskeratosis Congenita.

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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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