Hematologic MalignancyPhase 2FB4-PEDIANCT01572181
Study of a Reduced-toxicity Myeloablative Conditioning Regimen Using Fludarabine and Full Doses of Intravenous Busulfan in Pediatric Patients Not Eligible for Standard Myeloablative Conditioning Regimens
A Phase 2 study testing Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®) in people with Hematologic Malignancy, run by Nantes University Hospital at 15 sites in France.
Trial summary
The purpose of this study is to assess transplant-related mortality (TRM) at one year after allogeneic hematopoietic stem cell transplantation (allo-HSCT) prepared by a "reduced toxicity myeloablative" conditioning regimen in young patients (children and adolescents) with hematologic malignancies. Read more about this study
- Who is it for?
- Ages 12 months to 25 years
- Conditions the study is for
- Hematologic Malignancy
- What is being tested?
- Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 15 sitesFrance
- Study dates
- Apr 2012to 24 Oct 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 18Show fewer
- 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
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
France15 sites
University Hospital
Besançon
University Hospital
Bordeaux
University Hospital
Clermont-Ferrand
University Hospital
Grenoble
University Hospital
Lille
University Hospital
Lyon
University Hospital
Marseille
University Hospital
Montpellier
University Hospital
Nancy
University Hospital
Nantes
University Hopsital
Paris
University Hospital
Paris
University Hospital
Rennes
University Hopsital
Rouen
University Hospital
Strasbourg
About this study
Official title: Phase 2 Study of a Reduced-toxicity Myeloablative Conditionning Regimen Using Fludarabine and Full Doses of iv Busulfan in Pediatric Patients Not Eligible for Standard Myeloablative Conditioning Regimens
The purpose of this study is to assess transplant-related mortality (TRM) at one year after allogeneic hematopoietic stem cell transplantation (allo-HSCT) prepared by a "reduced toxicity myeloablative" conditioning regimen in young patients (children and adolescents) with hematologic malignancies.
- Conditions
- Hematologic Malignancy
- Being tested
- Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)Drug
- IV fludarabine (30 mg/m²/day for 5 days)
- IV Busulfan (Busilvex 3.2 mg/kg/day for 4 days) (the Busulfan dose is to be adapted to the weight of the child according to the drug label)
- Anti-thymocyte globulines (Thymogolubuline, 2.5 mg/kg/day for 2 days).
| Study group | Role | What they receive |
|---|---|---|
| Drugs | Experimental | Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®) Receives: Drug: Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Transplant-related mortality (TRM)
Evaluation of the cumulative incidence of TRM at 12 months after transplantation
Measured: 12 months
Secondary outcomes (5)
Incidence of engraftment
Incidence of engraftment defined as the first day of neutrophil (>500/μl for 3 consecutive days). Engraftment failure is defined as neutrophil <500/μl at day+42 after allo-SCT.
Measured: Day+42
Evaluation of overall (OS) and disease-free survival (DFS)
Evaluation of overall (OS) and disease-free survival (DFS) at 1 year after transplantation
Measured: 12 months
Cumulative incidence of relapse, death from disease, and non-relapse mortality (NRM)
Cumulative incidence of relapse, death from disease, and non-relapse mortality (NRM)
Measured: 12 months
Cumulative incidences and severity of acute and chronic Graft-versus-Host disease
Cumulative incidences and severity of acute and chronic Graft-versus-Host disease
Measured: 12 months
Immune Recovery (to be determined in a subgroup of patients)
Immune Recovery parameters: blood counts, bone marrow aspiration with evaluation of morphological response.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2012 (estimated)
- Study startApr 2012
- Primary completion24 Oct 2017
- Study completion24 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematologic MalignancyStudies of the same condition, open and under way.
- More trials run by Nantes University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nantes University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mohamad MOHTY, Professor, Principal investigator, Nantes University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01572181
- Sponsor's protocol ID
- BRD/11/06-N
- Acronym
- FB4-PEDIA
- Status last verified
- Apr 2018
- First submitted
- 21 Feb 2012
- Last updated
- 14 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
Publications
Rialland F, Grain A, Labopin M, Michel G, Gandemer V, Paillard C, Pochon C, Clement L, Brissot E, Jubert C, Sirvent A, Rohrlich PS, Plantaz D, Dalle JH, Mohty M. Reduced-toxicity myeloablative conditioning regimen using fludarabine and full doses of intravenous busulfan in pediatric patients not eligible for standard myeloablative conditioning regimens: Results of a multicenter prospective phase 2 trial. Bone Marrow Transplant. 2022 Nov;57(11):1698-1703. doi: 10.1038/s41409-022-01769-5. Epub 2022 Aug 26.
PubMed 36028757
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of a Reduced-toxicity Myeloablative Conditionning Regimen Using Fludarabine and Full Doses of iv Busulfan in Pediatric Patients Not Eligible for Standard Myeloablative Conditioning Regimens
- ClinicalTrials.gov ID
- NCT01572181
- Acronym / sponsor ID
- FB4-PEDIA / BRD/11/06-N
- Conditions
- Hematologic Malignancy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Nantes University HospitalUniversity, hospital or other
- Study start
- Apr 2012 (actual)
- Primary completion
- 24 Oct 2017 (actual)
- Study completion
- 24 Oct 2017 (actual)
- First posted
- 6 Apr 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess transplant-related mortality (TRM) at one year after allogeneic hematopoietic stem cell transplantation (allo-HSCT) prepared by a "reduced toxicity myeloablative" conditioning regimen in young patients (children and adolescents) with hematologic malignancies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Drugs | Experimental | Drug: Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®) |
Who may qualify
- Ages
- 12 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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:
Key exclusion criteria (18 listed)
- 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%)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)* IV fludarabine (30 mg/m²/day for 5 days) * IV Busulfan (Busilvex 3.2 mg/kg/day for 4 days) (the Busulfan dose is to be adapted to the weight of the child according to the drug label) * Anti-thymocyte globulines (Thymogolubuline, 2.5 mg/kg/day for 2 days). | Drug | Drugs |
Primary outcome
Transplant-related mortality (TRM)
Time frame: 12 months
Evaluation of the cumulative incidence of TRM at 12 months after transplantation
5 secondary outcomes
Incidence of engraftment
Time frame: Day+42
Evaluation of overall (OS) and disease-free survival (DFS)
Time frame: 12 months
Cumulative incidence of relapse, death from disease, and non-relapse mortality (NRM)
Time frame: 12 months
Cumulative incidences and severity of acute and chronic Graft-versus-Host disease
Time frame: 12 months
Immune Recovery (to be determined in a subgroup of patients)
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2026.
- Results publication: PubMed 36028757Rialland F, Grain A, Labopin M, Michel G, Gandemer V, Paillard C, Pochon C, Clement L, Brissot E, Jubert C, Sirvent A, Rohrlich PS, Plantaz D, Dalle JH, Mohty M. Reduced-toxicity myeloablative conditioning regimen using fludarabine and full doses of intravenous busulfan in pediatric patients not eligible for standard myeloablative conditioning regimens: Results of a multicenter prospective phase 2 trial. Bone Marrow Transplant. 2022 Nov;57(11):1698-1703. doi: 10.1038/s41409-022-01769-5. Epub 2022 Aug 26.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mohamad MOHTY, Professor principal investigator
Nantes University Hospital
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®) in people with Hematologic Malignancy, run by Nantes University Hospital at 15 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Transplant-related mortality (TRM) (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01572181, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2026.