A Phase 2 interventional study of CliniMACS® (T cell depletion) in Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia and Chronic Myeloid Leukemia, sponsored by Wake Forest University Health Sciences. Terminated at 1 site in United States. Open to participants aged Up to 30 Years. Per ClinicalTrials.gov, last updated 2022-04-22.
Sponsored by Wake Forest University Health Sciences · Phase 2, Interventional, and Treatment
The primary purpose is to determine the ability of CD34+ selection and T cell depletion using the CliniMACS® device to prevent severe acute graft-versus-host disease (GVHD) in patients receiving a stem cell transplant from an alternative (unrelated and mismatched related) donor. The secondary objectives include evaluation of engraftment, immune recovery, and post-transplant infections.
Patients requiring stem cell transplants for either malignant (cancerous) or non-malignant disease will be included in the study. The recipients will be grouped into one of two groups based on whether the donor is mismatched related (Cohort A) or unrelated (Cohort B). The patient will receive a conditioning regimen including chemotherapy drugs and/or total body irradiation based on the disease for which the transplant is performed.
A major issue in alternative donor (mismatched related and unrelated donor transplantation is the development of graft-versus-host disease (GVHD). Several clinical trials have shown that the use of T-cell depleted peripheral blood stem cells (PBSC) reduces GVHD in alternative donor transplants. The purpose of this study is to determine the ability of CD34 positive selection and T cell depletion using the CliniMACS® Device as the only GVHD prophylaxis to prevent severe acute GVHD in recipients of an alternative donor PBSC transplant. Mismatched related donors will match at least 3 of 6 Human leukocyte antigens(HLA)(haplocompatible) and unrelated donors will match at least 6 out of 8 HLA antigens with the transplant recipient. The conditioning therapy including chemotherapy, anti-thymocyte globulin (ATG), +/- total body irradiation (TBI) will be based on the patient's diagnosis. The transplant recipient will be followed for 5 years after transplant for GVHD, engraftment, post-transplant infections, disease relapse, and overall survival. In addition, this study will serve as a platform for a companion study of therapy to accelerate immune recovery after transplant.
2,584 studies on the registry are indexed under Precursor Cell Lymphoblastic Leukemia-Lymphoma; 473 are open to participants now.
This study's enrollment of 53 is above the median of 40 across 1,952 interventional studies indexed under Precursor Cell Lymphoblastic Leukemia-Lymphoma.
Browse Precursor Cell Lymphoblastic Leukemia-Lymphoma studies →Wake Forest University Health Sciences is the lead sponsor of 1,351 studies on the registry; 201 are open to participants now.
Of its 332 completed or terminated interventional studies of FDA-regulated products, 251 (76%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patient must have a mismatched related or an unrelated donor who is:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Recipients will receive T cell-depleted PBSC from eligible donors after receiving conditioning therapy using CliniMACS® device.
Device: CliniMACS® (T cell depletion)
Stem cells will be collected from donors after they receive Granulocyte colony-stimulating factor (G-CSF). The cells will be processed using the CliniMACS device to select for CD34+ stem cells and to deplete T cells. Recipients will receive conditioning therapy that is based on their disease type and then receive the CD34+ stem cells.
Also known as: CliniMACS device, T-cell depletion
Number of Participants With Severe Graft vs. Host Disease (GVHD).
Severe GVHD defined as grade III/IV GVHD.
Time frame: Within 30 days after stem cell transplant
Number of Participants With Engraftment and Time to Engraftment
Engraftment was measured as time to absolute neutrophil count \>500
Time frame: Within 28 days after stem cell transplant
Number of Participants With Post-transplant Infections
Time frame: 1 year
Number of Participants With EBV-related Post Transplant Lymphoproliferative Disorder (PTLD)
Time frame: 5 years
Number of Participants With Post-transplant Leukemia Relapse
Time frame: 5 years
Number of Participants With Transplant-related Mortality
Transplant-related mortality includes death due to regimen-related toxicity or GVHD (all causes other than disease relapse). Those who died due to disease relapse are not included in the analyzed population for that time point.
Time frame: 2 year
Number of Participants With Transplant-related Toxicities
Time frame: 1 year
Overall Survival
Time frame: 2 years
Device Performance: Dose of CD34+ Cells and CD3+ Cells Given
For mismatched related donors, the target cell dose after processing is \>/= 20 x 10\^6 CD34+ cells/kg patient body weight, but \>/= 8 x 10\^6 is acceptable. For unrelated donors the target cell dose after processing is \>/= 10 x 10\^6 CD34+ cells/kg patient body weight, but \>/= 4 x 10\^6 is acceptable. The target T cell dose is \</= 3 x 10\^4 CD3+ cells/ kg.
Time frame: Length of the trial (5 years)
Candidates for bone marrow transplant who did not have a healthy HLA-identical related donor, at Levine Children's Hospital between December 2009 and June 2016.
| Milestone | T Cell Depletion Using CliniMACS® |
|---|---|
| Started | 53 |
| Completed | 52 |
| Not completed | 1 |
| Withdrew: Protocol exception/ not evaluable | 1 |
Severe GVHD defined as grade III/IV GVHD.
| Participants | T Cell Depletion Using CliniMACS® |
|---|---|
| Number of Participants With Severe Graft vs. Host Disease (GVHD). | 0 |
Engraftment was measured as time to absolute neutrophil count \>500
| days | Mismatched Related Donor Cohort | Matched Unrelated Donor Cohort |
|---|---|---|
| Number of Participants With Engraftment and Time to Engraftment | 15 (9 to 19) | 13 (10 to 15) |
| Participants | T Cell Depletion Using CliniMACS® |
|---|---|
| HHV-6 reactivation, no disease | 39 |
| Viral URI (adeno, paraflu, rhino, rsv) | 25 |
| Clostridium difficile colitis | 24 |
| Adenovirus reactivation, no disease | 15 |
| Bacteremia, gram positive | 12 |
| Candidiasis (not invasive) | 11 |
| Sinusitis | 11 |
| Bacteremia, gram negative | 9 |
| CMV reactivation, no disease | 9 |
| BK virus, no disease | 7 |
| EBV-related lymphoproliferative disease | 6 |
| BK virus, hemorrhagic cystitis | 5 |
| Adenovirus disease | 5 |
| Infection with normal ANC - grade 3 | 5 |
| Norovirus | 5 |
| Positive Galactomannan | 5 |
| Acute otitis media | 4 |
| HSV - mouth sores | 4 |
| EBV reactivation, no disease | 4 |
| Pneumonia | 3 |
| Varicella zoster | 3 |
| Candidiasis (invasive) | 2 |
| CMV, GI disease/ encephalitis | 2 |
| Disseminated aspergillus | 2 |
| Methicillin-Resistant Staph Aureus | 2 |
| Rotavirus | 2 |
| Nocardia | 2 |
| Pneumonia - fungal (non-aspergillus) | 1 |
| Influenza | 1 |
| Parvovirus B19 (low level) | 1 |
| Sapovirus | 1 |
| Septic arthritis (prosthetic joint) | 1 |
| Possible respiratory infection (rhizomucor) | 1 |
| Participants | T Cell Depletion Using CliniMACS® |
|---|---|
| Number of Participants With EBV-related Post Transplant Lymphoproliferative Disorder (PTLD) | 6 |
| Participants | T Cell Depletion Using CliniMACS® |
|---|---|
| Number of Participants With Post-transplant Leukemia Relapse | 5 |
Transplant-related mortality includes death due to regimen-related toxicity or GVHD (all causes other than disease relapse). Those who died due to disease relapse are not included in the analyzed population for that time point.
| Participants | T Cell Depletion Using CliniMACS® |
|---|---|
| Day 100 | 1 |
| 1 year | 9 |
| 2 years | 11 |
| Participants | T Cell Depletion Using CliniMACS® |
|---|---|
| Thrombotic Microangiopathy | 8 |
| Idiopathic Pneumonia Syndrome | 3 |
| Veno-occlusive Disease | 1 |
| Participants | Mismatched Related Donor Cohort: Malignant Disease | Mismatched Related Donor: Non-malignant Disease | Matched Unrelated Donor Cohort |
|---|---|---|---|
| Overall Survival | 9 | 9 | 6 |
For mismatched related donors, the target cell dose after processing is \>/= 20 x 10\^6 CD34+ cells/kg patient body weight, but \>/= 8 x 10\^6 is acceptable. For unrelated donors the target cell dose after processing is \>/= 10 x 10\^6 CD34+ cells/kg patient body weight, but \>/= 4 x 10\^6 is acceptable. The target T cell dose is \</= 3 x 10\^4 CD3+ cells/ kg.
| cells/ kg | Mismatched Related Donor Cohort | Matched Unrelated Donor Cohort |
|---|---|---|
| x 10^6 CD34+ cells/ kg | 20.26 (8.78 to 25.03) | 15.16 (11.06 to 23.33) |
| x 10^4 CD3+ cells/ kg | 0.02 (0 to 1.1) | 0.3 (0 to 0.5) |
Collected over From the start of the conditioning regimen for transplant until 1 year after transplant. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| T Cell Depletion Using CliniMACS® | 15/52 (28.8%) | 45/52 (86.5%) | 33/52 (63.5%) |
| Event | T Cell Depletion Using CliniMACS® |
|---|---|
| FeverGeneral disorders | 32/52 |
| HHV-6 ReactivationInfections and infestations | 9/52 |
| Graft vs. Host diseaseImmune system disorders | 9/52 |
| Thrombotic microangiopathyBlood and lymphatic system disorders | 6/52 |
| Acute gastroenteritis/ diarrheaGastrointestinal disorders | 5/52 |
| CMV reactivationInfections and infestations | 4/52 |
| EBV-related lymphoproliferative diseaseInfections and infestations | 4/52 |
| PneumoniaInfections and infestations | 4/52 |
| RelapseNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 2/26 |
| Infection w/ unknown absolute neutrophil count (ANC)Infections and infestations | 3/52 |
| Event | T Cell Depletion Using CliniMACS® |
|---|---|
| Hypoxia - grade 3Respiratory, thoracic and mediastinal disorders | 14/52 |
| Rash - grade 3Skin and subcutaneous tissue disorders | 5/52 |
| Seizures - grade 3Nervous system disorders | 5/52 |
| Hypokalemia - grade 4Metabolism and nutrition disorders | 4/52 |
| Hypoxia - grade 4Respiratory, thoracic and mediastinal disorders | 4/52 |
| Renal and Urinary disorder - grade 3Renal and urinary disorders | 4/52 |
| Thrombotic microangiopathy - grade 4Blood and lymphatic system disorders | 4/52 |
| Thrombotic microangiopathy - grade 3Blood and lymphatic system disorders | 4/52 |
| Elevated ALT - grade 4Investigations | 3/52 |
| Hypertension - grade 4Vascular disorders | 3/52 |
| Age, Customized(Participants) | T Cell Depletion Using CliniMACS® |
|---|---|
| Less than 1 year | 4 |
| 1-2 years | 3 |
| 2-4 years | 5 |
| 4-6 years | 6 |
| 6-8 years | 3 |
| 8-10 years | 4 |
| 10-12 years | 6 |
| 12-14 years | 5 |
| 14-16 years | 5 |
| 16-18 years | 8 |
| Greater than 18 years | 3 |
| Sex: Female, Male(Participants) | T Cell Depletion Using CliniMACS® |
|---|---|
| Female | 25 |
| Male | 27 |
| Race/Ethnicity, Customized(Participants) | T Cell Depletion Using CliniMACS® |
|---|---|
| Caucasian | 10 |
| African American | 26 |
| Hispanic or Latino | 13 |
| Other | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
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