Personalized Targeted Preparative Regimen Before T-depleted Allogeneic HSCT in Children With Chemoresistent Acute Leukemias
NCT ID: NCT04000698
Last Updated: 2020-11-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE3
25 participants
INTERVENTIONAL
2019-10-15
2022-12-31
Brief Summary
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Detailed Description
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The hypothesis of the study is that personalized targeted therapy combined with high-dose chemotherapy may improve the outcome of allogeneic HSCT in a cohort of pediatric patients with refractory leukemia.
Bcl-2, CD38, CD184 were chosen as potential targets due to frequent expression in pediatric acute leukemias, availability of marketed targeted therapies venetoclax, daratumumab and prelixafor, and expected non-overlapping toxicity profile of these agents and the conditioning regimen.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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intervention/treatment
Preparative chemotherapy before allogeneic HSCT
* Fludarabin
* Cytarabine
* Venetoclax
* Daratumomab
* Vecanoid
* treosulfan
* fludarabine
* thiophosphomide
* Venetoclax
* Plerixafor
* abatacept
* tocilizumab
* rituximab
* HSCT from the haploidentical donor, ex vivo depleted of alpha/beta T lymphocytes
Preparative regimen
Preparative chemotherapy before allogeneic HSCT
* Fludarabin
* Cytarabine
* Venetoclax
* Daratumomab
* Vecanoid Condition
* treosulfan
* fludarabine
* thiophosphomide
* Venetoclax
* Plerixafor GVHD prophylaxis
* abatacept
* tocilizumab
* rituximab
* HSCT from the haploidentical donor, ex vivo depleted of alpha/beta T lymphocytes
Interventions
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Preparative regimen
Preparative chemotherapy before allogeneic HSCT
* Fludarabin
* Cytarabine
* Venetoclax
* Daratumomab
* Vecanoid Condition
* treosulfan
* fludarabine
* thiophosphomide
* Venetoclax
* Plerixafor GVHD prophylaxis
* abatacept
* tocilizumab
* rituximab
* HSCT from the haploidentical donor, ex vivo depleted of alpha/beta T lymphocytes
Eligibility Criteria
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Inclusion Criteria
2. Disease stage
* Acute myeloid leukemia (AML), relapsed or refractory, failure to achieve hematologic remission after at least to courses of intensive chemotherapy, including at least one course with high-dose AraC and fludarabine
* Acute lymphoblastic leukemia (ALL), relapsed or refractory, failure to achieve hematologic remission after at least two high-dose therapy blocks
3. Patient eligible for current hematopoietic stem cell transplantation protocol
4. The BCL-2 expression must be detected on greater than 30% of tumor cells (AML and ALL) by flow cytometry
5. CD38 expression must be detected on greater than 30% of tumor cells (AML and ALL) by flow cytometry
6. CD184
7. Patients must have measurable or evaluable disease at the time of enrollment, which may include any evidence of disease including minimal residual disease detected by flow cytometry, cytogenetics, or polymerase chain reaction (PCR) analysis.
8. Patient Clinical Performance Status: Karnofsky \>50% or Lansky \>50%
9. Patient Life Expectancy \>12 weeks
10. Patients who agree to long-term follow up for up to 5 years
Exclusion Criteria
* Patients with uncontrolled infections
* Clearance of creatinine \< 70 ml/min
* Cardiac ejection fraction \< 40%
* Patients who can perform pulmonary function tests will be excluded if they have a diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) of \< 50% predicted; patients who are unable to perform pulmonary function tests will be excluded if the oxygen (O2) saturation is \< 92% on room air
* Patients who have liver function test (LFTs) (including total bilirubin, aspartate aminotransferase \[AST\] and alanine aminotransferase \[ALT\]) \>= twice the upper limit of normal
* Karnofsky/Lansky Scale \<70%
25 Years
ALL
No
Sponsors
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Federal Research Institute of Pediatric Hematology, Oncology and Immunology
OTHER
Responsible Party
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Locations
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Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, , Russia
Countries
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Central Contacts
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Facility Contacts
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Zhanna Shekhovtsova, MD
Role: primary
Eugene Pashanov, PhD
Role: backup
Other Identifiers
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NCPHOI-2018-08
Identifier Type: -
Identifier Source: org_study_id