Acute Myeloid LeukemiaPhase 2NCT03088709
Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide
A Phase 2 study testing Cyclophosphamide, Tacrolimus and Mycophenolate mofetil in people with Acute Myeloid Leukemia and Acute Lymphocytic Leukemia, run by Loyola University at 1 site in United States.
Trial summary
Historically, the best results of allogeneic SCT have been obtained when the stem cell donor is a human leukocyte antigen (HLA)-matched sibling, however, this is only available for approximately 30 percent of patients in need for SCT. Read more about this study
- Who is it for?
- Ages 16 to 90 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome
- What is being tested?
- Cyclophosphamide, Tacrolimus and Mycophenolate mofetil
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jan 2017to 31 Jan 2022
- 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 16 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Ages 16 years old and up
- Performance Status 70 percent or above
- Patients should have the following diseases:
- Acute myelogenous leukemia (AML)
- Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL)
- Transfusion dependent myelodysplastic syndrome (MDS)
Show all 11Show fewer
- Non-Hodgkin's Lymphoma (NHL)
- Chronic lymphocytic leukemia (CLL)
- Pulmonary function as measured by forced expiratory volume at one second (FEV1) and/or corrected diffusing capacity of lung for carbon monoxide (DLCO) at 60 percent of predicted or above
- Left ventricular ejection fraction at 45 percent or above
- If the donor-specific HLA antibodies (DSA) are positive, the patient must undergo a desensitization protocol resulting in undetectable DSA prior to day of transplant
Who cannot join
- Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except for hydroxyurea)
- Uncontrolled bacterial, fungal or viral infections at time of study enrollment
- Positive for HIV, human T-cell leukemia virus (HTLV-1) and/or Hepatitis C
- Subjects with signs/symptoms of active central nervous system (CNS) disease
Trial locations and contacts
1 site in 1 country.
United States1 site
Loyola University Medical Center
Maywood, Illinois, 60153
About this study
Official title: Safety, Efficacy and Feasibility of Haploidentical Stem Cell Transplantation (Haplo-SCT) Using Post-Transplant Cyclophosphamide (PTCy) as an Alternative Donor Source for Patients Who Lack a Matched Sibling/Unrelated Donor Options
Historically, the best results of allogeneic SCT have been obtained when the stem cell donor is a human leukocyte antigen (HLA)-matched sibling, however, this is only available for approximately 30 percent of patients in need for SCT. Alternative donor sources include matched unrelated donor utilizing the donor registry, cord blood transplant and mismatched donor transplant. A human leukocyte antigen (HLA)-haploidentical donor is one who shares, by common inheritance, exactly one HLA haplotype with the recipient, and includes the biologic parents, biologic children and full or half siblings. There is strong body of evidence supporting the use of haplo-SCT in patient who lack a matched sibling or unrelated donor with high rates of successful engraftment, effective Graft Versus Host Disease (GVHD) control and favorable outcomes comparative to those seen using other allograft sources, including HLA-matched sibling SCT. Furthermore, it provides a cost-efficient donor option in a timely manner especially for patients who need to proceed quickly to transplant due to concern of disease relapse/progression.
Read the detailed description
An open label, single-arm, single-center study to evaluate the safety, efficacy and feasibility of haplo-SCT as an alternative donor source for patients who lack a matched sibling/unrelated donor options. The choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD.
GVHD Prevention Treatment:
Cyclophosphamide will be administered IV on Day 3 and Day 5 post transplant.
Tacrolimus will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant.
Mycophenolate mofetil will be administered IV until patient can take it by mouth starting on Day 1 post transplant until 28 days.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Non-hodgkin Lymphoma, Chronic Lymphocytic Leukemia
- Being tested
- Cyclophosphamide, Tacrolimus, Mycophenolate mofetil, Haploidentical Stem Cell Transplantation
- Keywords
- NHL, Non-Hodgkin Lymphoma, Haploidentical, Bone Marrow Transplant, Stem Cell Transplant, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, GVHD
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, Chronic, B-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Also called Cytoxan
IV medication given for prevention of graft versus host disease.
TacrolimusDrug
Also called Prograf
IV medication given for prevention of graft versus host disease.
Also called Cellcept
IV medication given for prevention of graft versus host disease.
Haploidentical Stem Cell TransplantationOther
Also called Haploidentical hematopoietic stem cell transplantation
A stem cell transplant that involves matching a patient's tissue type, specifically their human leukocyte antigen (HLA) tissue type, with that of a related donor.
| Study group | Role | What they receive |
|---|---|---|
| All patients will receive Haploidentical | Experimental | The choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD. All patients will receive a Haplo-identical stem cell transplantation.
GVHD Prevention Treatment:
Cyclophosphamide 50mg/kg will be administered IV on Day 3 and Day 5 post transplant.
Tacrolimus 0.03 mg/kg daily will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant.
Mycophenolate mofetil 15mg/kg will be administered twice a day IV until patient can take it by mouth starting on Day 1 post transplant until 28 days. Receives: Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Other: Haploidentical Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Chimerism
Blood test that measures amount of donor's cells
Measured: 100 days
Secondary outcomes (8)
Neutrophil engraftment
Blood test that measures the white cell count
Measured: Day 28
Platelet engraftment
Blood test that measures the platelet count
Measured: Day 60
Grade 3 to 4 acute graft-verus-host disease (GVHD)
National Institutes of Health Acute Graft-Versus-Host Disease Grading and Form
Measured: 100 days
Frequency and severity of chronic GVHD
National Institutes of Health Chronic Graft-Versus-Host Disease Grading and Form
Measured: 1 year
Disease status with blast counts (immature blood cell count) above 5%
Blood work and/or bone marrow biopsy will be used
Measured: 3 years
Survival status by patient contact
Contact with patient by phone or doctor's visit
Measured: 3 years
Immune reconstitution
Blood work will be used to evaluate recovery of T and B cell count subset that assess cells which make antibodies to fight infections
Measured: 3 years
Grade 3 through 5 Adverse Events
Toxicities that are possibly, probably, and definitely related to study treatment according to NCI CTCAE Version 4
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2017
- Study start18 Jan 2017
- Primary completion31 Jan 2022
- Study completion31 Jan 2022
Why it stopped: Poor accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Loyola University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hagen, Patrick A, Loyola University
- Investigators
- Zeina Al-Mansour, MD, Principal investigator, Cardinal Bernardin Cancer Center, Loyola University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03088709
- Sponsor's protocol ID
- 208941
- Status last verified
- Jul 2024
- First submitted
- 22 Feb 2017
- Last updated
- 10 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Efficacy and Feasibility of Haploidentical Stem Cell Transplantation (Haplo-SCT) Using Post-Transplant Cyclophosphamide (PTCy) as an Alternative Donor Source for Patients Who Lack a Matched Sibling/Unrelated Donor Options
- ClinicalTrials.gov ID
- NCT03088709
- Acronym / sponsor ID
- 208941
- Conditions
- Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Non-hodgkin Lymphoma, Chronic Lymphocytic Leukemia
- Keywords
- NHL, Non-Hodgkin Lymphoma, Haploidentical, Bone Marrow Transplant, Stem Cell Transplant, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor accrual
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Loyola UniversityUniversity, hospital or other
- Study start
- 18 Jan 2017 (actual)
- Primary completion
- 31 Jan 2022 (actual)
- Study completion
- 31 Jan 2022 (actual)
- First posted
- 23 Mar 2017 (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.
Historically, the best results of allogeneic SCT have been obtained when the stem cell donor is a human leukocyte antigen (HLA)-matched sibling, however, this is only available for approximately 30 percent of patients in need for SCT. Alternative donor sources include matched unrelated donor utilizing the donor registry, cord blood transplant and mismatched donor transplant. A human leukocyte antigen (HLA)-haploidentical donor is one who shares, by common inheritance, exactly one HLA haplotype with the recipient, and includes the biologic parents, biologic children and full or half siblings. There is strong body of evidence supporting the use of haplo-SCT in patient who lack a matched sibling or unrelated donor with high rates of successful engraftment, effective Graft Versus Host Disease (GVHD) control and favorable outcomes comparative to those seen using other allograft sources, including HLA-matched sibling SCT. Furthermore, it provides a cost-efficient donor option in a timely manner especially for patients who need to proceed quickly to transplant due to concern of disease relapse/progression.
Detailed description
An open label, single-arm, single-center study to evaluate the safety, efficacy and feasibility of haplo-SCT as an alternative donor source for patients who lack a matched sibling/unrelated donor options. The choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD.
GVHD Prevention Treatment:
Cyclophosphamide will be administered IV on Day 3 and Day 5 post transplant.
Tacrolimus will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant.
Mycophenolate mofetil will be administered IV until patient can take it by mouth starting on Day 1 post transplant until 28 days.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All patients will receive Haploidentical | Experimental | Drug: Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate mofetil, Other: Haploidentical Stem Cell TransplantationThe choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD. All patients will receive a Haplo-identical stem cell transplantation. GVHD Prevention Treatment: Cyclophosphamide 50mg/kg will be administered IV on Day 3 and Day 5 post transplant. Tacrolimus 0.03 mg/kg daily will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant. Mycophenolate mofetil 15mg/kg will be administered twice a day IV until patient can take it by mouth starting on Day 1 post transplant until 28 days. |
Who may qualify
- Ages
- 16 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Ages 16 years old and up
- Performance Status 70 percent or above
- Patients should have the following diseases:
- Acute myelogenous leukemia (AML)
- Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL)
Key exclusion criteria (4 listed)
- Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except for hydroxyurea)
- Uncontrolled bacterial, fungal or viral infections at time of study enrollment
- Positive for HIV, human T-cell leukemia virus (HTLV-1) and/or Hepatitis C
- Subjects with signs/symptoms of active central nervous system (CNS) 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 |
|---|---|---|
| CyclophosphamideAlso: CytoxanIV medication given for prevention of graft versus host disease. | Drug | All patients will receive Haploidentical |
| TacrolimusAlso: PrografIV medication given for prevention of graft versus host disease. | Drug | All patients will receive Haploidentical |
| Mycophenolate mofetilAlso: CellceptIV medication given for prevention of graft versus host disease. | Drug | All patients will receive Haploidentical |
| Haploidentical Stem Cell TransplantationAlso: Haploidentical hematopoietic stem cell transplantationA stem cell transplant that involves matching a patient's tissue type, specifically their human leukocyte antigen (HLA) tissue type, with that of a related donor. | Other | All patients will receive Haploidentical |
Primary outcome
Chimerism
Time frame: 100 days
Blood test that measures amount of donor's cells
8 secondary outcomes
Neutrophil engraftment
Time frame: Day 28
Platelet engraftment
Time frame: Day 60
Grade 3 to 4 acute graft-verus-host disease (GVHD)
Time frame: 100 days
Frequency and severity of chronic GVHD
Time frame: 1 year
Disease status with blast counts (immature blood cell count) above 5%
Time frame: 3 years
Survival status by patient contact
Time frame: 3 years
Immune reconstitution
Time frame: 3 years
Grade 3 through 5 Adverse Events
Time frame: 2 years
Results and safety
The study was stopped early (reason given: Poor accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2024.
- Individual participant dataSharing planned: No
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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.
Zeina Al-Mansour, MD principal investigator
Cardinal Bernardin Cancer Center, Loyola University
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 Cyclophosphamide, Tacrolimus and Mycophenolate mofetil in people with Acute Myeloid Leukemia and Acute Lymphocytic Leukemia, run by Loyola University at 1 site in United States.
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 11 inclusion and 4 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: stopped early, as of the sponsor's last update on 10 Jul 2024. 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 United States. 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 was stopped early (reason given: Poor accrual). Results are not available in the source record.
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?
Chimerism (time frame: 100 days). 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 10 Jul 2024.