Softer chemo before stem cell transplant aims to cure blood cancers in frail children
NCT ID NCT01572181
First seen Sep 14, 2026 · Last updated Sep 15, 2026 · Updated 1 time
Summary
Researchers test a reduced-toxicity conditioning regimen before allogeneic stem cell transplant in children and young adults with blood cancers. The regimen combines fludarabine, full-dose busulfan, and anti-thymocyte globulin. The trial measures transplant-related mortality at one year and tracks engraftment and survival. Participants are aged 1 to 25 who cannot tolerate standard myeloablative conditioning.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- A conditioning regimen of fludarabine, busulfan, and anti-thymocyte globulin given before stem cell transplant
- What this could lead to
- If successful, this regimen could offer a safer path to curative stem cell transplant for children with blood cancers who are too frail for standard conditioning.
- What could go wrong
- This is a Phase 2 trial with 50 participants, so results may not apply broadly. The regimen still carries risks of transplant-related complications, and it may not control the cancer as well as standard treatment.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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50 people
The number who actually took part.
- Started
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Apr 2012
- Finished
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Oct 2017
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 months to 25 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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 * Children and adolescents aged over 12 months and under 25 years * Availability of an HLA identical family donor or an HLA-matched unrelated donor (10/10 or 9/10 if the mismatch level is at HLACw for an unrelated donor) or availability of an HLA matched cord blood (5/6 or 6/6) * Informed consent signed by patients (18-25 years) and patient's legal representative, parent(s) or guardian (cf p13) * Diagnosis of a hematologic malignancy which is a candidate for allo-HSCT, but not eligible for standard or conventional myeloablative conditioning regimens because of high risk for toxicity. * Are considered as criteria of non-eligibility for standard or conventional myeloablative conditioning: * a history of autologous or allogeneic stem cell transplantation * comorbidities or medical history predictive of a prohibitive rate of TRM and toxicity with the use of standard high dose chemotherapy and / or radiotherapy. Exclusion Criteria: * Patient has been administered any other systemic chemotherapeutic drug (including Gemtuzumab) within 21 days prior to trial enrollment and start of the conditioning regimen. Hydroxyurea is permitted if indicated to control induction refractory disease, and IT chemotherapy is allowed if indicated as maintenance treatment for previously diagnosed leptomeningeal disease, that has been in remission for at least 3 months prior to enrollment on this study. * Active infection. Protocol PI will be final arbiter if there is uncertainty regarding whether a previous infection is resolved. * Children and adolescents who are not older than 12 months and under 25 years * A donor who is HLA mismatched at the level of more than one locus. * Poor performance status (Lansky \< 50%) * Life expectancy is severely limited by concomitant illness and expected to be \<12 weeks. * Left ventricular ejection fraction \< 30%. Uncontrolled arrhythmias or symptomatic cardiac disease. * Symptomatic pulmonary disease. FEV1, FVC and DLCO \<30% of expected corrected for hemoglobin. * Creatinine clearance less than 30 mL/m per 1.73 m2 or requiring dialysis * Evidence of chronic active hepatitis or cirrhosis. If positive hepatitis serology, discuss with Study Chairman and consider liver biopsy. * Effusion or ascites \>1L prior to drainage. * HIV-positive. * Female pregnancy * Absence of effective contraception among boys and girls of childbearing potential (that contraception should be continued until 6 months after stopping treatment) * Breastfeeding * Patient's legal representative, parent(s) or guardian not able to sign informed consent. * children's refusal * Hypersensitivity to rabbit proteins, to the active substance or to any of the excipients of experimental products
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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University Hopsital
Paris, France
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University Hopsital
Rouen, France
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University Hospital
Besançon, France
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University Hospital
Bordeaux, France
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University Hospital
Clermont-Ferrand, France
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University Hospital
Grenoble, France
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University Hospital
Lille, France
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University Hospital
Lyon, France
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University Hospital
Marseille, France
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University Hospital
Montpellier, France
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University Hospital
Nancy, France
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University Hospital
Nantes, France
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University Hospital
Paris, France
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University Hospital
Rennes, France
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University Hospital
Strasbourg, France
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