Acute LeukemiaPhase 2BUSEQNCT04451200
Sequential and Personalized PK-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allo-HSCT in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning
A Phase 2 study testing Busulfan Injection in people with Acute Leukemia and Mielodysplasic Syndrome, run by Institut Paoli-Calmettes.
Trial summary
Because the anti-leukemic activity of busulfan, this dug is largely used in graft conditioning but in elderly and/or cormobid patienth an excess of toxicity is observed. This study focus on the possibility of significanty reducing this toxicity by customizing the doses of busulfan to individual PK parameters. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Acute Leukemia, Mielodysplasic Syndrome, Myeloproliferative Neoplasm
- What is being tested?
- Busulfan Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 82 participants
- Where
- No sites listed yet
- Study dates
- Nov 2020to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Adult patient up to 65 years old
- Acute leukemia, myelodysplastic syndrome or myeloproliferative neoplasia eligible for an allogeneic transplant
- Chemosensitive disease, in complete or partial or stable remission
- Allograft from an identical HLA related donor, Haplo-identical or unrelated (HLA compatibility from 8/10 to 10/10 according to HLA-A, -B, -C, -DR, -DQ allelics)
- Signed consent to participate
- -. Affiliation to a social security regimen or beneficiary of this regimen
- Patient not eligible for standard myeloablative conditioning due to age> = 45 years and / or the presence of an HCT-CI comorbidity score> = 3
Who cannot join
- Pregnant woman, without effective contraception or breastfeeding
- Person in emergency situation, patient deprived of liberty or placed under the authority of a tutor,
- Impossibility of undergoing medical follow-up of the trial for geographic, social or psychological reasons
- Contraindications to performing an allogeneic transplant
- Previous allograft
- Placental blood allograft
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Sequential and Personalized Pharmacokinetic-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allogeneic Haematopoietic Stem Cell Transplantation in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning
Because the anti-leukemic activity of busulfan, this dug is largely used in graft conditioning but in elderly and/or cormobid patienth an excess of toxicity is observed. This study focus on the possibility of significanty reducing this toxicity by customizing the doses of busulfan to individual PK parameters.
- Being tested
- Busulfan Injection
- Medical subject headings
- Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Busulfan InjectionDrug
injections doses will be personalized by PK at days -7 and -4
| Study group | Role | What they receive |
|---|---|---|
| busulfan treatment | Experimental | Personalized BU administration Receives: Drug: Busulfan Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
non-relapse mortality evaluation
non-relapse mortality evaluation
Measured: 100 days post graft
Secondary outcomes (14)
incidence of grade 3 or 4 toxicities
To evaluate toxicities linked to sequential bususlfan administration
Measured: 1 month
graft taking after sequential busulfan conditioning
incidence of hematological reconstituation
Measured: day 30 and day 100 post graft
incidence of transfusion needs for red blood cells
number of transfusions after graft
Measured: day 30 post graft
incidence of transfusion needs for red blood cells
number of transfusions after graft
Measured: day 60 post graft
incidence in graft taking after sequential busulfan conditioning
Lymphocyte chimerism
Measured: day 100 post graft
anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
incidence of acute GVH
Measured: 1 year
anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
incidence of acute GVH
Measured: 5 years
the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
incidence of chronic GVH
Measured: 1 and 5 years
the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
incidence of chronic GVH
Measured: 1 year
anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
progression-free survival
Measured: 5 years
anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
overall survival
Measured: 1 year
anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
overall survival
Measured: 5 years
anti-tumoral efficacy of sequential busulfan conditioning: Incidence of relapse
Incidence of relapse
Measured: 1 year
Differences between the theoretical target AUC and the measured a posteriori
To study the pharmacokinetics of the sequential administration of busulfan
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2020
- Study startNov 2020 (estimated)
- Primary completionNov 2023 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiaStudies of the same condition, open and under way.
- 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
- 82 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institut Paoli-Calmettes
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04451200
- Sponsor's protocol ID
- BUSEQ-IPC 2020-006
- Acronym
- BUSEQ
- Status last verified
- Oct 2020
- First submitted
- 3 Jun 2020
- Last updated
- 17 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sequential and Personalized Pharmacokinetic-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allogeneic Haematopoietic Stem Cell Transplantation in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning
- ClinicalTrials.gov ID
- NCT04451200
- Acronym / sponsor ID
- BUSEQ / BUSEQ-IPC 2020-006
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 82 participants (estimated)
- Lead sponsor
- Institut Paoli-CalmettesUniversity, hospital or other
- Study start
- Nov 2020 (estimated)
- Primary completion
- Nov 2023 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 30 Jun 2020 (actual)
- 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.
Because the anti-leukemic activity of busulfan, this dug is largely used in graft conditioning but in elderly and/or cormobid patienth an excess of toxicity is observed. This study focus on the possibility of significanty reducing this toxicity by customizing the doses of busulfan to individual PK parameters.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| busulfan treatment | Experimental | Drug: Busulfan InjectionPersonalized BU administration |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Adult patient up to 65 years old
- Acute leukemia, myelodysplastic syndrome or myeloproliferative neoplasia eligible for an allogeneic transplant
- Chemosensitive disease, in complete or partial or stable remission
- Allograft from an identical HLA related donor, Haplo-identical or unrelated (HLA compatibility from 8/10 to 10/10 according to HLA-A, -B, -C, -DR, -DQ allelics)
- Signed consent to participate
Key exclusion criteria (6 listed)
- Pregnant woman, without effective contraception or breastfeeding
- Person in emergency situation, patient deprived of liberty or placed under the authority of a tutor,
- Impossibility of undergoing medical follow-up of the trial for geographic, social or psychological reasons
- Contraindications to performing an allogeneic transplant
- Previous allograft
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 |
|---|---|---|
| Busulfan Injectioninjections doses will be personalized by PK at days -7 and -4 | Drug | busulfan treatment |
Primary outcome
non-relapse mortality evaluation
Time frame: 100 days post graft
non-relapse mortality evaluation
14 secondary outcomes
incidence of grade 3 or 4 toxicities
Time frame: 1 month
graft taking after sequential busulfan conditioning
Time frame: day 30 and day 100 post graft
incidence of transfusion needs for red blood cells
Time frame: day 30 post graft
incidence of transfusion needs for red blood cells
Time frame: day 60 post graft
incidence in graft taking after sequential busulfan conditioning
Time frame: day 100 post graft
anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
Time frame: 1 year
anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
Time frame: 5 years
the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
Time frame: 1 and 5 years
the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
Time frame: 1 year
anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
Time frame: 5 years
anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
Time frame: 1 year
anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
Time frame: 5 years
anti-tumoral efficacy of sequential busulfan conditioning: Incidence of relapse
Time frame: 1 year
Differences between the theoretical target AUC and the measured a posteriori
Time frame: 1 month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2020.
- Individual participant dataSharing planned: No
Related trials for Acute Leukemia
Not yet recruitingPhase 1/2
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
RecruitingPhase 1
Anti-CRLF2-R/TSLPR Chimeric Antigen Receptor T Cells (TSLPR-CART) in Participants With Recurrent or Refractory CRLF2-R/TSLPR-Overexpressing B-Cell Acute Lymphoblastic Leukemia (B-ALL)
B-All, Acute Lymphoblastic Leukemia
RecruitingPhase 2
CD22 CAR T-cells to Extend Remission Following Commercial CD19 CAR T-cells in Children, Adolescents, and Adults With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia
Acute Lymphoblastic Leukemia, B-All
RecruitingPhase 1
Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies
Malignancy, Hematologic, Neoplasms, Hematologic
RecruitingPhase 3
International Study for Treatment of Childhood Relapsed Precursor B-Cell ALL 2020 (IntReALL BCP 2020)
B-Cell Acute Lymphoblastic Leukaemia
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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Busulfan Injection in people with Acute Leukemia and Mielodysplasic Syndrome, run by Institut Paoli-Calmettes.
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 6 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: opening soon, as of the sponsor's last update on 17 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dominique Genre, MD (+33491223778, drci.up@ipc.unicancer.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
non-relapse mortality evaluation (time frame: 100 days post graft). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Nov 2020.