Leukemia, Myeloid, AcuteNCT02759822
Haploidentical Hematopoietic Stem Cell Transplantation for Acute Leukemias
An observational study of Leukemia, Myeloid, Acute and Precursor Cell Lymphoblastic Leukemia-Lymphoma, run by Leonardo Javier Arcuri at 1 site in Brazil.
Trial summary
This is a prospective observational cohort study of haploidentical transplantation with post-transplant cyclophosphamide for acute leukemias using reduced intensity conditioning for acute myeloid leukemia (AML) and myeloablative conditioning for acute lymphoblastic leukemia (ALL). Read more about this study
- Who is it for?
- Ages 1 year to 60 years
- Conditions the study is for
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
- What is being tested?
- Haploidentical Stem Cell Transplantation
- Type of treatment
- Surgery or procedure
- How many people
- 30 participants
- Where
- 1 siteBrazil
- Study dates
- Apr 2016to Apr 2021
- Phase
- Observational
- 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 1 year to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients with acute leukemias referred for stem cell transplantation without HLA-matched related or unrelated donor
Who may be able to join
- Acute lymphoblastic leukemia or acute myeloid leukemia
Who cannot join
- Severe comorbidities
- Second transplant
Trial locations and contacts
1 site in 1 country.
Brazil1 site, 1 recruiting
- Recruiting
Instituto Nacional de Cancer
Rio de Janeiro, Rio de Janeiro, 20230-130
Leonardo J Arcuri, MD, +55(21)3207-1304, leonardojavier@gmail.com
Simone Lermontov, +55(21)3207-1261, simonelermontov@globo.com
About this study
This is a prospective observational cohort study of haploidentical transplantation with post-transplant cyclophosphamide for acute leukemias using reduced intensity conditioning for acute myeloid leukemia (AML) and myeloablative conditioning for acute lymphoblastic leukemia (ALL).
Read the detailed description
Hematopoietic stem cell transplantation (HSCT) is the most effective treatment for acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), with the lowest rates of relapse.
Fertility rate in Brazil is falling, and only 25% of people born today will have a matched sibling donor. On the other hand, currently donor non-related to about 50% of patients enrolled in Brazilian Receptor Registry (REREME). Consequently, at least 35% of patients won't have a matched donor.
The haploidentical transplantation is defined as a partially matched hematopoietic cell transplantation, using a partially matched family donor (parent, sibling or child). Haploidentical transplantation activity is growing worldwide, with results comparable matched unrelated donors.
The objective of this study is to test the feasibility of haploidentical transplantation with post transplant cyclophosphamide for acute leukemias in a Brazil.
- Being tested
- Haploidentical Stem Cell Transplantation
- Keywords
- Haploidentical Hematopoietic Stem Cell Transplantation, Partially Matched Hematopoietic Stem Cell Transplantation, Post-transplant Cyclophosphamide, Hematopoietic Stem Cell Transplantation
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Haploidentical Stem Cell TransplantationProcedure or surgery
Haploidentical Stem Cell Transplantation from a related donor (partially matched sibling, father, mother, son or daughter).
| Study group | Role | What they receive |
|---|---|---|
| Group A: Acute Lymphoblastic Leukemia | Haploidentical Stem Cell Transplantation with Post Transplant Cyclophosphamide (PTCy)
Preferred conditioning:
* Total Body Irradiation 1200 cGy (TBI1200) + Fludarabine 120 mg/m2 (Flu120)
Alternative conditionings:
1. Melphalan 100-140 mg/m2 (Mel100-140) + Fludarabine 160 mg/m2 (Flu160) + Total Body Irradiation 200 cGy (TBI200)
2. Busulfan 9.6 mg/kg (Bu9.6) + Fludarabine 150 mg/m2 (Flu150) + TBI200 Graft versus host disease Prophylaxis: Post-Transplant Cyclophosphamide (PTCy) + Tacrolimus (FK) or Cyclosporin (CSA) + Mycophenolate Mofetil (MMF) Receives: Procedure: Haploidentical Stem Cell Transplantation | |
| Group B: Acute Myeloid Leukemia | Haploidentical Stem Cell Transplantation with Post Transplant Cyclophosphamide (PTCy) Preferred Conditioning: Mel100-140 + Flu160 + TBI200
Alternative conditionings:
1. Bu9,6 + Flu150 + TBI200
2. Cyclophosphamide 29 mg/kg + Flu150 + TBI200 Graft versus host disease Prophylaxis: PTCy + FK or CSA + MMF Receives: Procedure: Haploidentical Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
1-year overall survival. Survival curves will be estimated by Kaplan-Meier methodology.
Measured: 1 year
Secondary outcomes (5)
Cumulative incidence of leukemia relapse
Relapse will be defined by reappearance of Leukemic Cells in bone marrow (>5%) or peripheral blood (>1%). Risk factors for leukemia relapse will be estimated by extended Cox Model for competing risks (Fine \& Gray model). Competing event will be defined as death in remission.
Measured: 5 years
Transplant Related Mortality
Transplant-related mortality (TRM) will be defined as death in remission. Risk factors for TRM will be estimated by extended Cox Model for competing risks (Fine \& Gray model). Competing event will be defined as leukemia relapse.
Measured: 6 months
Cumulative Incidence of acute Graft Versus Host Disease
Acute graft versus host disease will be diagnosed and graded by modified Glucksberg criteria. Risk factor for acute graft versus host disease will be estimated by extended Cox Model for competing risks (Fine \& Gray model). Cumulative incidence curves will be estimated by Gray methodology.
Measured: 5 years
Cumulative Incidence of chronic Graft Versus Host Disease
Chronic graft versus host disease will be diagnosed and graded by National Institute of Health Consensus (NIH Consensus). Risk factor for chronic graft versus host disease will be estimated by extended Cox Model for competing risks (Fine \& Gray model). Cumulative incidence curves will be estimated by Gray methodology.
Measured: 5 years
Cumulative Incidence of Infectious Complications
Cumulative incidence of viral, fungal and bacterial infections. Subgroups will be compared by Cox Model for competing risks (Fine \& Gray model). Cytomegalovirus reactivation will be analyzed by multiple events Cox Model.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2016 (estimated)
- Study startApr 2016
- Primary completionApr 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myeloid, AcuteStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Leonardo Javier Arcuri
- Sponsor type
- Other government
- Responsible party
- Sponsor-investigator, Leonardo Javier Arcuri, Instituto Nacional de Cancer, Brazil
- Investigators
- Leonardo J Arcuri, MD, Principal investigator, Instituto Nacional de Cancer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02759822
- Sponsor's protocol ID
- CEMO/INCA-Haplo-01
- Status last verified
- Apr 2016
- First submitted
- 22 Apr 2016
- Last updated
- 3 May 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Hematopoietic Stem Cell Transplantation for Acute Leukemias
- ClinicalTrials.gov ID
- NCT02759822
- Acronym / sponsor ID
- CEMO/INCA-Haplo-01
- Keywords
- Haploidentical Hematopoietic Stem Cell Transplantation, Partially Matched Hematopoietic Stem Cell Transplantation, Post-transplant Cyclophosphamide, Hematopoietic Stem Cell Transplantation
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Leonardo Javier ArcuriOther government
- Study start
- Apr 2016
- Primary completion
- Apr 2021 (estimated)
- First posted
- 3 May 2016 (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.
This is a prospective observational cohort study of haploidentical transplantation with post-transplant cyclophosphamide for acute leukemias using reduced intensity conditioning for acute myeloid leukemia (AML) and myeloablative conditioning for acute lymphoblastic leukemia (ALL).
Detailed description
Hematopoietic stem cell transplantation (HSCT) is the most effective treatment for acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), with the lowest rates of relapse.
Fertility rate in Brazil is falling, and only 25% of people born today will have a matched sibling donor. On the other hand, currently donor non-related to about 50% of patients enrolled in Brazilian Receptor Registry (REREME). Consequently, at least 35% of patients won't have a matched donor.
The haploidentical transplantation is defined as a partially matched hematopoietic cell transplantation, using a partially matched family donor (parent, sibling or child). Haploidentical transplantation activity is growing worldwide, with results comparable matched unrelated donors.
The objective of this study is to test the feasibility of haploidentical transplantation with post transplant cyclophosphamide for acute leukemias in a Brazil.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A: Acute Lymphoblastic Leukemia | – | Procedure: Haploidentical Stem Cell TransplantationHaploidentical Stem Cell Transplantation with Post Transplant Cyclophosphamide (PTCy) Preferred conditioning: * Total Body Irradiation 1200 cGy (TBI1200) + Fludarabine 120 mg/m2 (Flu120) Alternative conditionings: 1. Melphalan 100-140 mg/m2 (Mel100-140) + Fludarabine 160 mg/m2 (Flu160) + Total Body Irradiation 200 cGy (TBI200) 2. Busulfan 9.6 mg/kg (Bu9.6) + Fludarabine 150 mg/m2 (Flu150) + TBI200 Graft versus host disease Prophylaxis: Post-Transplant Cyclophosphamide (PTCy) + Tacrolimus (FK) or Cyclosporin (CSA) + Mycophenolate Mofetil (MMF) |
| Group B: Acute Myeloid Leukemia | – | Procedure: Haploidentical Stem Cell TransplantationHaploidentical Stem Cell Transplantation with Post Transplant Cyclophosphamide (PTCy) Preferred Conditioning: Mel100-140 + Flu160 + TBI200 Alternative conditionings: 1. Bu9,6 + Flu150 + TBI200 2. Cyclophosphamide 29 mg/kg + Flu150 + TBI200 Graft versus host disease Prophylaxis: PTCy + FK or CSA + MMF |
Who may qualify
- Ages
- 1 year to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with acute leukemias referred for stem cell transplantation without HLA-matched related or unrelated donor
Key inclusion criteria (1 listed)
- Acute lymphoblastic leukemia or acute myeloid leukemia
Key exclusion criteria (2 listed)
- Severe comorbidities
- Second transplant
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 |
|---|---|---|
| Haploidentical Stem Cell TransplantationHaploidentical Stem Cell Transplantation from a related donor (partially matched sibling, father, mother, son or daughter). | Procedure or surgery | Group A: Acute Lymphoblastic Leukemia, Group B: Acute Myeloid Leukemia |
Primary outcome
Overall Survival
Time frame: 1 year
1-year overall survival. Survival curves will be estimated by Kaplan-Meier methodology.
5 secondary outcomes
Cumulative incidence of leukemia relapse
Time frame: 5 years
Transplant Related Mortality
Time frame: 6 months
Cumulative Incidence of acute Graft Versus Host Disease
Time frame: 5 years
Cumulative Incidence of chronic Graft Versus Host Disease
Time frame: 5 years
Cumulative Incidence of Infectious Complications
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2016.
- Individual participant dataSharing planned: No
Related trials for Leukemia, Myeloid, Acute
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
Leonardo J Arcuri, MD principal investigator
Instituto Nacional de Cancer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia, Myeloid, Acute and Precursor Cell Lymphoblastic Leukemia-Lymphoma, run by Leonardo Javier Arcuri at 1 site in Brazil.
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 1 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 3 May 2016. 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 Brazil. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Leonado J Arcuri, MD (+55(21)32071304, leonardojavier@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (time frame: 1 year). 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 3 May 2016.