Hematological DiseasePhase 1HaploOnlyNCT02660281
URMC Related Haplo-identical Donor BMT
A Phase 1 study testing Total Body Irradiation 1200 cGy, Fludarabine and Pre-Stem Cell Infusion Cyclophosphamide in people with Hematological Disease and Immune Deficiencies, run by University of Rochester at 1 site in United States.
Trial summary
This study will be a single-center treatment protocol, designed to validate the process of related donor haploidentical-SCT at the Wilmot Cancer Institute Blood and Marrow Transplant Unit. Read more about this study
- Who is it for?
- Ages 6 months to 75 years
- Conditions the study is for
- Hematological Disease, Immune Deficiencies, Solid Tumors
- What is being tested?
- Total Body Irradiation 1200 cGy, Fludarabine and Pre-Stem Cell Infusion Cyclophosphamide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 74 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2015to 7 Jan 2021
- Phase
- Phase 1
- 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 6 months to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient Age:
- Pediatric (ages 6 months to 18 years)
- Adult (ages 18-75 years)
- Disease:
- Congenital and Other Non-malignant Disorders
- Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome)
Show all 36Show fewer
- Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital Osteopetrosis, Osteogenesis Imperfecta)
- Metabolic disorders (e.g. Hurler's Syndrome)
- Hemoglobinopathies (e.g. Sickle Cell Disease, Thalassemia)
- Severe aplastic anemia
- High-Risk Leukemias
- Acute Myelogenous Leukemia
- Refractory to standard induction therapy (more than 1 cycle required to achieve remission)
- Recurrent (in CR≥2)
- Treatment-related AML or MDS
- Evolved from myelodysplastic syndrome
- Presence of Flt3 abnormalities
- FAB M6 or M7
- Adverse cytogenetics
- Myelodysplastic Syndrome
- Acute Lymphoblastic Leukemia including T lymphoblastic leukemia
- Refractory to standard induction therapy (time to CR >4 weeks)
- Recurrent (in CR ≥2)
- WBC count >30,000/mcL at diagnosis
- Age >30 at diagnosis
- Adverse cytogenetics, such as (t(9:22), t(1:19), t(4:11), other MLL rearrangements.
- Chronic Myelogenous Leukemia in accelerated phase or blast crisis
- Biphenotypic or undifferentiated leukemia
- Burkitt's leukemia or lymphoma
- Lymphoma:
- Large cell, Mantle cell, Hodgkin lymphoma refractory or recurrent, chemosensitive, and ineligible for an autologous stem cell transplant or previously treated with autologous SCT
- Marginal zone or follicular lymphoma that is progressive after at least two prior therapies
- Multiple Myeloma, recurrent following high-dose therapy and autologous SCT or ineligible for an autologous HSCT
- Solid tumors, with efficacy of allogeneic HSCT demonstrated for the specific disease and disease status
- Graft failure following prior related donor, unrelated donor or UCB transplant
- Myelofibrosis
Who cannot join
- Patient Age below 6 months or over 75 years
- Availability of a 10/10 HLA-matched related or unrelated donor within a reasonable time-frame dictated by the clinical urgency of the transplant
- Autologous HSCT < 6 months prior to proposed haplo-SCT
- Pregnant or breast-feeding
- Current uncontrolled infection
- Evidence of HIV infection or positive HIV serology
- Anti-donor HLA antibodies with positive crossmatch and unsuccessful -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Haploidentical Donor Hematopoietic Stem Cell Transplantation
This study will be a single-center treatment protocol, designed to validate the process of related donor haploidentical-SCT at the Wilmot Cancer Institute Blood and Marrow Transplant Unit.
Read the detailed description
This study will be a single-center treatment protocol with five possible preparative regimens, designed to validate the process of related donor haploidentical-SCT at the Wilmot Cancer Institute Blood and Marrow Transplant Unit. Enrolled patients will receive chemotherapy +/- radiation as a pre-transplant conditioning regimen. Patients will then receive haploidentical stem cells, either bone marrow or mobilized peripheral blood, followed by GvHD prophylaxis that will include cyclophosphamide. Multiple data points will be collected prior to, during, and following transplantation to ensure safety of the process and to evaluate the stated objectives.
- Conditions
- Hematological Disease, Immune Deficiencies, Solid Tumors, Myelofibrosis, Multiple Myeloma, Lymphoma
- Being tested
- Total Body Irradiation 1200 cGy, Fludarabine, Pre-Stem Cell Infusion Cyclophosphamide, Pre-Stem Cell Infusion Mesna, Busulfan
- Medical subject headings
- Hematologic Diseases, Primary Myelofibrosis, Multiple Myeloma, Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Total Body Irradiation 1200 cGyRadiation
Also called TBI
1200 cGy TBI in 8 fractions
FludarabineDrug
Fludarabine
Pre-Stem Cell Infusion CyclophosphamideDrug
Cyclophosphamide given prior to the stem cell infusion
Pre-Stem Cell Infusion MesnaDrug
Mesna given prior to the stem cell infusion
BusulfanDrug
Busulfan
MelphalanDrug
Melphalan
Stem Cell InfusionProcedure or surgery
Stem cell infusion
Post-Stem Cell Infusion CyclophosphamideDrug
Cyclophosphamide given after the stem cell infusion
Post-Stem Cell Infusion MesnaDrug
Mesna given after the Stem Cell Infusion
ThiotepaDrug
Thiotepa
| Study group | Role | What they receive |
|---|---|---|
| Full Intensity TBI-based Conditioning | Other | Total Body Irradiation 1200 cGy of 150 cGy over 4 or 5 days, days -5 or -4 to -1 Fludarabine 30 mg/m2/day x 3 days, days -6, -5, -4 Stem Cell Infusion, day 0 Post- Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Mesna 50 mg/kg/day x 2 days, days +3 and +4 Receives: Radiation: Total Body Irradiation 1200 cGy; Drug: Fludarabine; Procedure: Stem Cell Infusion; Drug: Post-Stem Cell Infusion Cyclophosphamide; Drug: Post-Stem Cell Infusion Mesna |
| Full Intensity Chemo-Only Conditioning | Other | Fludarabine 25 mg/m2/day x 5 days, days -6, -5, -4, -3, -2 Busulfan 130 mg/m2/day x 4 days, days -6, -5, -4, -3 Pre-Stem Cell Infusion Cyclophosphamide 14.5 mg/kg/day x 2 days, days -3 and -2 Pre-Stem Cell Infusion Mesna 14.5 mg/kg/day x 2 days, days -3 and -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell Infusion Mesna 50 mg/kg/day x 2 days, days +3 and +4 Receives: Drug: Fludarabine; Drug: Pre-Stem Cell Infusion Cyclophosphamide; Drug: Pre-Stem Cell Infusion Mesna; Drug: Busulfan; Procedure: Stem Cell Infusion; Drug: Post-Stem Cell Infusion Cyclophosphamide; Drug: Post-Stem Cell Infusion Mesna |
| Reduced Intensity Conditioning | Other | Fludarabine 30 mg/m2/day x 5 days, days -6 to -2 Melphalan 140 mg/m2/day x 1 day, day -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell InfusionMesna 50 mg/kg/day x 2 days, days +3 and +4 Receives: Drug: Fludarabine; Drug: Melphalan; Procedure: Stem Cell Infusion; Drug: Post-Stem Cell Infusion Cyclophosphamide; Drug: Post-Stem Cell Infusion Mesna |
| Non-Myeloablative Conditioning | Other | Fludarabine 30 mg/m2/day x 5 days, days -6 to -2 Pre-Stem Cell Infusion Cyclophosphamide 14.5 mg/kg/day x 2 days, days -3 and -2 Pre-Stem Cell InfusionMesna 14.5 mg/kg/day x 2 days, days -3 and -2 Total Body Irradiation 200 cGy, day -1 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, day +3 and +4 Post-Stem Cell Infusion Mesna 50 mg/kg/day x 2 days, day +3 and +4 Receives: Radiation: Total Body Irradiation 1200 cGy; Drug: Fludarabine; Drug: Pre-Stem Cell Infusion Cyclophosphamide; Drug: Pre-Stem Cell Infusion Mesna; Procedure: Stem Cell Infusion; Drug: Post-Stem Cell Infusion Cyclophosphamide; Drug: Post-Stem Cell Infusion Mesna |
| Reduced Intensity Conditioning with Addition of Thiotepa | Other | Fludarabine 30 mg/m2/day x 5 days, days -6 to -2 Thiotepa 8 mg/kg, day -3 Melphalan 140 mg/m2/day x 1 day, day -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell InfusionMesna 50 mg/kg/day x 2 days, days +3 and +4 Receives: Drug: Fludarabine; Drug: Melphalan; Procedure: Stem Cell Infusion; Drug: Post-Stem Cell Infusion Cyclophosphamide; Drug: Post-Stem Cell Infusion Mesna; Drug: Thiotepa |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of days from the date of the stem cell infusion to engraftment of absolute neutrophils (ANC) and platelets (PLT) will be determined based on daily CBC and differential counts.
The date of engraftment of ANC is the first of 3 consecutive days of ANC of 500 or higher. The date of engraftment of platelets is the first of 3 consecutive days of platelet counts of 20,000 or higher in the absence of platelet transfusions for at least 7 days prior.
Measured: 42 days following the infusion of stem cells for ANC. [If engraftment of ANC does not occur within 42 days, a subsequent transplant will be performed if a donor is available.
Rate of non-engraftment and secondary graft failure
The percentage of patients who fail to engraft ANC will be tabulated as well as the percentage of patients who have primary engraftment of ANC but whose graft then fails as evidenced by pancytopenia and failure of bone marrow function.
Measured: At 100 days, 6 months, and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's date of death up to 120 months.
Secondary outcomes (7)
Percentage of subjects who develop acute graft-versus-host disease.
Assess the presence and date of onset of acute GvHD based on the criteria published by Przepiorka et al., Bone Marrow Transplant 1995; 15(6):825-8 as used by the Center for International Blood \& Marrow Transplant Research.
Measured: 100 days following the infusion of stem cells
Assess the Maximum Overall Grades 0- IV of acute GvHD and Maximum Severity (0-4) by involved organ system in patients who develop acute graft-versus-host disease.
Assess the severity of acute GvHD based on the criteria published by Przepiorka et al., Bone Marrow Transplant 1995; 15(6):825-8 as used by the Center for International Blood \& Marrow Transplant Research.
Measured: 100 days following the infusion of stem cells
Percentage of subjects who develop chronic graft-versus-host disease.
Calculate the percentage of patients who develop chronic graft-versus-host disease based on Sullivan KM, Blood 1981; 57:267 as used by the Center for International Blood \& Marrow Transplant Research.
Measured: Minimally at intervals of 100 days, 6 months and then yearly following the infusion of stem cells until the subject's date of death.or up to 120 months.
Assess the Maximum Grade: Limited versus Extensive; Maximum Severity: Mild, Moderate or Severe) of chronic GvHD in patients who develop chronic graft-versus-
Assess the severity of chronic GvHD in patients who develop chronic graft-versus-host disease based on Sullivan KM, Blood 1981; 57:267 as used by the Center for International Blood \& Marrow Transplant Research.
Measured: Minimally at intervals of 100 days, 6 months and then yearly following the infusion of stem cells until the subject's date of death or up to 120 months.
Disease-free survival
Document and update the length of time a subject survives without recurrence of the disease for which they were transplanted minimally at 100 days. 6 months, and yearly following the infusion of stem cells for as long as the patient survives and remains disease-free.
Measured: Disease assessments are performed and reported at time points that include 100 days, 6 months, and yearly until documented progression of disease or the date of death or up to 120 months.
Time to relapsed disease
Document the time to relapse of the disease for which the patient was Minimally assessments will be performed at 100 days. 6 months, and yearly following the infusion of stem cells for as long as the patient survives and remains disease-free.
Measured: Disease assessments are performed and reported at time points that include 100 days, 6 months, and yearly until the date of documented progression or the subject's date of death or up to 120 months.
Immune reconstitution
Evaluate immune reconstitution by measurement of immunoglobulins (IgG, IgA, and IgM), assessment of infections, and lymphocyte analysis
Measured: At 100 day, 6 month and one year intervals following the infusion of stem cells until the subject's date of death or up to 120 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2016 (estimated)
- Study startOct 2015
- Primary completion28 Oct 2019
- Study completion7 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematological DiseaseStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 74 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Rochester
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jeffrey Andolina, University of Rochester
- Investigators
- Jeffrey Andolina, MD, Principal investigator, Wilmot Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02660281
- Sponsor's protocol ID
- UBMT 15056
- Acronym
- HaploOnly
- Status last verified
- Nov 2021
- First submitted
- 24 Nov 2015
- Last updated
- 3 Nov 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Donor Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT02660281
- Acronym / sponsor ID
- HaploOnly / UBMT 15056
- Conditions
- Hematological Disease, Immune Deficiencies, Solid Tumors, Myelofibrosis, Multiple Myeloma, Lymphoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 74 participants (actual)
- Lead sponsor
- University of RochesterUniversity, hospital or other
- Study start
- Oct 2015
- Primary completion
- 28 Oct 2019 (actual)
- Study completion
- 7 Jan 2021 (actual)
- First posted
- 21 Jan 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 study will be a single-center treatment protocol, designed to validate the process of related donor haploidentical-SCT at the Wilmot Cancer Institute Blood and Marrow Transplant Unit.
Detailed description
This study will be a single-center treatment protocol with five possible preparative regimens, designed to validate the process of related donor haploidentical-SCT at the Wilmot Cancer Institute Blood and Marrow Transplant Unit. Enrolled patients will receive chemotherapy +/- radiation as a pre-transplant conditioning regimen. Patients will then receive haploidentical stem cells, either bone marrow or mobilized peripheral blood, followed by GvHD prophylaxis that will include cyclophosphamide. Multiple data points will be collected prior to, during, and following transplantation to ensure safety of the process and to evaluate the stated objectives.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Full Intensity TBI-based Conditioning | Other | Radiation: Total Body Irradiation 1200 cGy, Drug: Fludarabine, Procedure: Stem Cell Infusion, Drug: Post-Stem Cell Infusion Cyclophosphamide, Drug: Post-Stem Cell Infusion MesnaTotal Body Irradiation 1200 cGy of 150 cGy over 4 or 5 days, days -5 or -4 to -1 Fludarabine 30 mg/m2/day x 3 days, days -6, -5, -4 Stem Cell Infusion, day 0 Post- Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Mesna 50 mg/kg/day x 2 days, days +3 and +4 |
| Full Intensity Chemo-Only Conditioning | Other | Drug: Fludarabine, Drug: Pre-Stem Cell Infusion Cyclophosphamide, Drug: Pre-Stem Cell Infusion Mesna, Drug: Busulfan, Procedure: Stem Cell Infusion, Drug: Post-Stem Cell Infusion Cyclophosphamide, Drug: Post-Stem Cell Infusion MesnaFludarabine 25 mg/m2/day x 5 days, days -6, -5, -4, -3, -2 Busulfan 130 mg/m2/day x 4 days, days -6, -5, -4, -3 Pre-Stem Cell Infusion Cyclophosphamide 14.5 mg/kg/day x 2 days, days -3 and -2 Pre-Stem Cell Infusion Mesna 14.5 mg/kg/day x 2 days, days -3 and -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell Infusion Mesna 50 mg/kg/day x 2 days, days +3 and +4 |
| Reduced Intensity Conditioning | Other | Drug: Fludarabine, Drug: Melphalan, Procedure: Stem Cell Infusion, Drug: Post-Stem Cell Infusion Cyclophosphamide, Drug: Post-Stem Cell Infusion MesnaFludarabine 30 mg/m2/day x 5 days, days -6 to -2 Melphalan 140 mg/m2/day x 1 day, day -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell InfusionMesna 50 mg/kg/day x 2 days, days +3 and +4 |
| Non-Myeloablative Conditioning | Other | Radiation: Total Body Irradiation 1200 cGy, Drug: Fludarabine, Drug: Pre-Stem Cell Infusion Cyclophosphamide, Drug: Pre-Stem Cell Infusion Mesna, Procedure: Stem Cell Infusion, Drug: Post-Stem Cell Infusion Cyclophosphamide, Drug: Post-Stem Cell Infusion MesnaFludarabine 30 mg/m2/day x 5 days, days -6 to -2 Pre-Stem Cell Infusion Cyclophosphamide 14.5 mg/kg/day x 2 days, days -3 and -2 Pre-Stem Cell InfusionMesna 14.5 mg/kg/day x 2 days, days -3 and -2 Total Body Irradiation 200 cGy, day -1 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, day +3 and +4 Post-Stem Cell Infusion Mesna 50 mg/kg/day x 2 days, day +3 and +4 |
| Reduced Intensity Conditioning with Addition of Thiotepa | Other | Drug: Fludarabine, Drug: Melphalan, Procedure: Stem Cell Infusion, Drug: Post-Stem Cell Infusion Cyclophosphamide, Drug: Post-Stem Cell Infusion Mesna, Drug: ThiotepaFludarabine 30 mg/m2/day x 5 days, days -6 to -2 Thiotepa 8 mg/kg, day -3 Melphalan 140 mg/m2/day x 1 day, day -2 Stem Cell Infusion, day 0 Post-Stem Cell Infusion Cyclophosphamide 50 mg/kg/day x 2 days, days +3 and +4 Post-Stem Cell InfusionMesna 50 mg/kg/day x 2 days, days +3 and +4 |
Who may qualify
- Ages
- 6 months to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (36 listed)
- Pediatric (ages 6 months to 18 years)
- Adult (ages 18-75 years)
- Congenital and Other Non-malignant Disorders
- Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome)
- Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital Osteopetrosis, Osteogenesis Imperfecta)
Key exclusion criteria (7 listed)
- Patient Age below 6 months or over 75 years
- Availability of a 10/10 HLA-matched related or unrelated donor within a reasonable time-frame dictated by the clinical urgency of the transplant
- Autologous HSCT < 6 months prior to proposed haplo-SCT
- Pregnant or breast-feeding
- Current uncontrolled infection
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 |
|---|---|---|
| Total Body Irradiation 1200 cGyAlso: TBI1200 cGy TBI in 8 fractions | Radiation | Full Intensity TBI-based Conditioning, Non-Myeloablative Conditioning |
| FludarabineFludarabine | Drug | Full Intensity Chemo-Only Conditioning, Full Intensity TBI-based Conditioning, Non-Myeloablative Conditioning, Reduced Intensity Conditioning, Reduced Intensity Conditioning with Addition of Thiotepa |
| Pre-Stem Cell Infusion CyclophosphamideCyclophosphamide given prior to the stem cell infusion | Drug | Full Intensity Chemo-Only Conditioning, Non-Myeloablative Conditioning |
| Pre-Stem Cell Infusion MesnaMesna given prior to the stem cell infusion | Drug | Full Intensity Chemo-Only Conditioning, Non-Myeloablative Conditioning |
| BusulfanBusulfan | Drug | Full Intensity Chemo-Only Conditioning |
| MelphalanMelphalan | Drug | Reduced Intensity Conditioning, Reduced Intensity Conditioning with Addition of Thiotepa |
| Stem Cell InfusionStem cell infusion | Procedure or surgery | Full Intensity Chemo-Only Conditioning, Full Intensity TBI-based Conditioning, Non-Myeloablative Conditioning, Reduced Intensity Conditioning, Reduced Intensity Conditioning with Addition of Thiotepa |
| Post-Stem Cell Infusion CyclophosphamideCyclophosphamide given after the stem cell infusion | Drug | Full Intensity Chemo-Only Conditioning, Full Intensity TBI-based Conditioning, Non-Myeloablative Conditioning, Reduced Intensity Conditioning, Reduced Intensity Conditioning with Addition of Thiotepa |
| Post-Stem Cell Infusion MesnaMesna given after the Stem Cell Infusion | Drug | Full Intensity Chemo-Only Conditioning, Full Intensity TBI-based Conditioning, Non-Myeloablative Conditioning, Reduced Intensity Conditioning, Reduced Intensity Conditioning with Addition of Thiotepa |
| ThiotepaThiotepa | Drug | Reduced Intensity Conditioning with Addition of Thiotepa |
Primary outcome
The number of days from the date of the stem cell infusion to engraftment of absolute neutrophils (ANC) and platelets (PLT) will be determined based on daily CBC and differential counts.
Time frame: 42 days following the infusion of stem cells for ANC. [If engraftment of ANC does not occur within 42 days, a subsequent transplant will be performed if a donor is available.
The date of engraftment of ANC is the first of 3 consecutive days of ANC of 500 or higher. The date of engraftment of platelets is the first of 3 consecutive days of platelet counts of 20,000 or higher in the absence of platelet transfusions for at least 7 days prior.
Rate of non-engraftment and secondary graft failure
Time frame: At 100 days, 6 months, and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's date of death up to 120 months.
The percentage of patients who fail to engraft ANC will be tabulated as well as the percentage of patients who have primary engraftment of ANC but whose graft then fails as evidenced by pancytopenia and failure of bone marrow function.
7 secondary outcomes
Percentage of subjects who develop acute graft-versus-host disease.
Time frame: 100 days following the infusion of stem cells
Assess the Maximum Overall Grades 0- IV of acute GvHD and Maximum Severity (0-4) by involved organ system in patients who develop acute graft-versus-host disease.
Time frame: 100 days following the infusion of stem cells
Percentage of subjects who develop chronic graft-versus-host disease.
Time frame: Minimally at intervals of 100 days, 6 months and then yearly following the infusion of stem cells until the subject's date of death.or up to 120 months.
Assess the Maximum Grade: Limited versus Extensive; Maximum Severity: Mild, Moderate or Severe) of chronic GvHD in patients who develop chronic graft-versus-
Time frame: Minimally at intervals of 100 days, 6 months and then yearly following the infusion of stem cells until the subject's date of death or up to 120 months.
Disease-free survival
Time frame: Disease assessments are performed and reported at time points that include 100 days, 6 months, and yearly until documented progression of disease or the date of death or up to 120 months.
Time to relapsed disease
Time frame: Disease assessments are performed and reported at time points that include 100 days, 6 months, and yearly until the date of documented progression or the subject's date of death or up to 120 months.
Immune reconstitution
Time frame: At 100 day, 6 month and one year intervals following the infusion of stem cells until the subject's date of death or up to 120 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 3 Nov 2021.
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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.
Jeffrey Andolina, MD principal investigator
Wilmot Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Total Body Irradiation 1200 cGy, Fludarabine and Pre-Stem Cell Infusion Cyclophosphamide in people with Hematological Disease and Immune Deficiencies, run by University of Rochester 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 36 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 3 Nov 2021. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The number of days from the date of the stem cell infusion to engraftment of absolute neutrophils (ANC) and platelets (PLT) will be determined based on daily CBC and differential counts. (time frame: 42 days following the infusion of stem cells for ANC. [If engraftment of ANC does not occur within 42 days, a subsequent transplant will be performed if a donor is available.). The study lists 2 primary outcomes in all. 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 Nov 2021.