Myelodysplastic SyndromePhase 2AAANCT01985061
Evaluation of 3 Different Doses of IV Busulfan
A Phase 2 study testing BX2, BX3 and BX4-Suspended in people with Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by Institut Paoli-Calmettes at 1 site in France.
Trial summary
Albeit the safety of the stem cell transplantation procedure has been greatly improved, further refining the intensity of the conditioning is an important issue to explore, especially in patients with poor prognosis, the goal being to maintain the very favorable safety profile and improve the disease control. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- What is being tested?
- BX2, BX3 and BX4-Suspended
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 177 participants
- Where
- 1 siteFrance
- Study dates
- Dec 2013to 27 May 2026
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with poor prognosis myeloid malignancies:
- Myelodysplastic syndrome,
- Acute Myeloid Leukemia (AML) beyond Complete Response (CR1),
- CR1 AML with poor risk cytogenetics
- Adult patients: aged ≥ 55 years up to 65 or < 55 years not eligible for myeloablative conditioning regimen based on Total Body Irradiation (TBI) or double alkylating agent combinations.
- Availability of a HLA identical sibling or matched unrelated donor (10/10)
- Affiliation to social security
- Written Informed Consent
Who cannot join
- History of previous Allo-Hematological Stem Cell Transplantation (HSCT)
- HIV positivity
- Signs of chronic active hepatitis B and/or C
- Evolutive psychiatric disease
- Concomitant neoplastic disease
- Pregnant or lactating woman or without contraception (for child bearing potential wom-en)
- Usual contra-indications for Allo-HSCT
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Prospective and Multicentre Evaluation of 3 Different Doses of IV Busulfan Associated With Fludarabine and Thymoglobuline in the Conditioning of Allogeneic Stem Cell Transplantation (SCT) From a Matched Related or Unrelated Donor in Patients With Poor Prognosis Myeloid Malignancies
Albeit the safety of the stem cell transplantation procedure has been greatly improved, further refining the intensity of the conditioning is an important issue to explore, especially in patients with poor prognosis, the goal being to maintain the very favorable safety profile and improve the disease control. This is the goal our prospective trial; we aim to prospectively evaluate in a prospective multicenter trial the efficacy of different conditioning regimens in patients with high-risk myeloid malignancies.
The study is a phase II trial randomizing patients between a prospective active control arm (BX2) and two experimental arms (BX3 and BX4). A standard group was kept in this clinical trial in order to avoid the limitations induced by the comparison with historical controls in the context of continuously improving practice. Each experimental arm will be conducted in parallel according to a standard phase II trial design.
In addition, this trial will associate four ancillary studies to the main clinical objective: 1/ a prospective assessment of the quality of life of the patients over a period of 2 years 2/ an analysis of the cost effectiveness of the procedure, assessed over a period of 2 years 3/ an observational busulfan pharmacokinetic study 4/ a busulfan pharmacogenomic study
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Being tested
- BX2, BX3, BX4-Suspended
- Keywords
- Myelodysplasic syndrome, Acute Myeloid leukemia
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BX2Drug
Also called Busulfan Intravenous 2 days at 3.2 mg/kg/d
BX3Drug
Also called Busulfan Intravenous 3 days
BX4-SuspendedDrug
Also called Busulfan intravenous 4 days
Suspended
| Study group | Role | What they receive |
|---|---|---|
| BX2 | Active comparator | Fludarabine (Fludara®): 30 mg/m2 on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-4 and D-3 Thymoglobuline®: 2.5 mg/kg/d on D-3 and D-2 Receives: Drug: BX2 |
| BX3 | Experimental | Fludarabine (Fludara®): 30 mg/m² on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-5, D-4 and D-3 Thymoglobuline® : 2.5 mg/kg/d on D-3 and D-2 Receives: Drug: BX3 |
| BX4-Suspended | Active comparator | Fludarabine (Fludara®): 30 mg/m²on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-6, D-5, D-4 and D-3 Thymoglobuline® : 2.5 mg/kg/d on D-3 and D-2 Receives: Drug: BX4-Suspended |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to progression or death
2-year progression free survival rates
Measured: up to 2 years
Secondary outcomes (4)
Time to neutrophil>0.5G/l and platelets>50G/l
hematologic recovery
Measured: up to 2 months
Graft versus host disease
Measured: up to 2 years
relapse
Measured: up to 2 years
Occurrence of grade 3-4 adverse events according the CTC-AE v4.0 scale
safety
Measured: up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2013 (estimated)
- Study startDec 2013
- Primary completion27 May 2026
- Study completion27 May 2026
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 Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by Institut Paoli-CalmettesThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 177 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institut Paoli-Calmettes
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Didier BLAISE, MD PhD, Principal investigator, Institut Paoli-Calmettes
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01985061
- Sponsor's protocol ID
- AAA-IPC 2011-003
- Acronym
- AAA
- Status last verified
- Sep 2026
- First submitted
- 31 Oct 2013
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Wanquet A, Crocchiolo R, Furst S, Granata A, Faucher C, Devillier R, Harbi S, Lemarie C, Calmels B, Vey N, Weiller PJ, Chabannon C, Castagna L, Blaise D, El-Cheikh J. The efficacy and safety of a new reduced-toxicity conditioning with 4 days of once-daily 100 mg/m(2) intravenous busulfan associated with fludarabine and antithymocyte globulins prior to allogeneic stem cell transplantation in patients with high-risk myelodysplastic syndrome or acute leukemia. Leuk Lymphoma. 2016 Oct;57(10):2315-20. doi: 10.3109/10428194.2016.1146948. Epub 2016 Feb 17.
PubMed 26885686
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective and Multicentre Evaluation of 3 Different Doses of IV Busulfan Associated With Fludarabine and Thymoglobuline in the Conditioning of Allogeneic Stem Cell Transplantation (SCT) From a Matched Related or Unrelated Donor in Patients With Poor Prognosis Myeloid Malignancies
- ClinicalTrials.gov ID
- NCT01985061
- Acronym / sponsor ID
- AAA / AAA-IPC 2011-003
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Keywords
- Myelodysplasic syndrome, Acute Myeloid leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 177 participants (estimated)
- Lead sponsor
- Institut Paoli-CalmettesUniversity, hospital or other
- Study start
- Dec 2013 (actual)
- Primary completion
- 27 May 2026 (actual)
- Study completion
- 27 May 2026 (actual)
- First posted
- 15 Nov 2013 (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.
Albeit the safety of the stem cell transplantation procedure has been greatly improved, further refining the intensity of the conditioning is an important issue to explore, especially in patients with poor prognosis, the goal being to maintain the very favorable safety profile and improve the disease control. This is the goal our prospective trial; we aim to prospectively evaluate in a prospective multicenter trial the efficacy of different conditioning regimens in patients with high-risk myeloid malignancies.
The study is a phase II trial randomizing patients between a prospective active control arm (BX2) and two experimental arms (BX3 and BX4). A standard group was kept in this clinical trial in order to avoid the limitations induced by the comparison with historical controls in the context of continuously improving practice. Each experimental arm will be conducted in parallel according to a standard phase II trial design.
In addition, this trial will associate four ancillary studies to the main clinical objective: 1/ a prospective assessment of the quality of life of the patients over a period of 2 years 2/ an analysis of the cost effectiveness of the procedure, assessed over a period of 2 years 3/ an observational busulfan pharmacokinetic study 4/ a busulfan pharmacogenomic study
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| BX2 | Active comparator | Drug: BX2Fludarabine (Fludara®): 30 mg/m2 on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-4 and D-3 Thymoglobuline®: 2.5 mg/kg/d on D-3 and D-2 |
| BX3 | Experimental | Drug: BX3Fludarabine (Fludara®): 30 mg/m² on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-5, D-4 and D-3 Thymoglobuline® : 2.5 mg/kg/d on D-3 and D-2 |
| BX4-Suspended | Active comparator | Drug: BX4-SuspendedFludarabine (Fludara®): 30 mg/m²on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-6, D-5, D-4 and D-3 Thymoglobuline® : 2.5 mg/kg/d on D-3 and D-2 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Patients with poor prognosis myeloid malignancies:
- Adult patients: aged ≥ 55 years up to 65 or < 55 years not eligible for myeloablative conditioning regimen based on Total Body Irradiation (TBI) or double alkylating agent combinations.
- Availability of a HLA identical sibling or matched unrelated donor (10/10)
- Affiliation to social security
- Written Informed Consent
Key exclusion criteria (7 listed)
- History of previous Allo-Hematological Stem Cell Transplantation (HSCT)
- HIV positivity
- Signs of chronic active hepatitis B and/or C
- Evolutive psychiatric disease
- Concomitant neoplastic disease
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 |
|---|---|---|
| BX2Also: Busulfan Intravenous 2 days at 3.2 mg/kg/d | Drug | BX2 |
| BX3Also: Busulfan Intravenous 3 days | Drug | BX3 |
| BX4-SuspendedAlso: Busulfan intravenous 4 daysSuspended | Drug | BX4-Suspended |
Primary outcome
Time to progression or death
Time frame: up to 2 years
2-year progression free survival rates
4 secondary outcomes
Time to neutrophil>0.5G/l and platelets>50G/l
Time frame: up to 2 months
Graft versus host disease
Time frame: up to 2 years
relapse
Time frame: up to 2 years
Occurrence of grade 3-4 adverse events according the CTC-AE v4.0 scale
Time frame: up to 6 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 1 Oct 2026.
- Results publication: PubMed 26885686Wanquet A, Crocchiolo R, Furst S, Granata A, Faucher C, Devillier R, Harbi S, Lemarie C, Calmels B, Vey N, Weiller PJ, Chabannon C, Castagna L, Blaise D, El-Cheikh J. The efficacy and safety of a new reduced-toxicity conditioning with 4 days of once-daily 100 mg/m(2) intravenous busulfan associated with fludarabine and antithymocyte globulins prior to allogeneic stem cell transplantation in patients with high-risk myelodysplastic syndrome or acute leukemia. Leuk Lymphoma. 2016 Oct;57(10):2315-20. doi: 10.3109/10428194.2016.1146948. Epub 2016 Feb 17.
- official web site of the sponsorLink provided by the sponsor.
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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.
Didier BLAISE, MD PhD principal investigator
Institut Paoli-Calmettes
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BX2, BX3 and BX4-Suspended in people with Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by Institut Paoli-Calmettes at 1 site 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 8 inclusion and 7 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 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Time to progression or death (time frame: up to 2 years). 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 NCT01985061, 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 1 Oct 2026.