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CompletedNCT01596257Updated Dec 29, 2022Results posted

Bulk Versus Fractionated Stem Cell Infusions in Patients With Hematologic Malignancies Undergoing Stem Cell Transplantation

A Phase 2 interventional study of allogeneic hematopoietic stem cell transplantation and CliniMACS in Leukemia, sponsored by Memorial Sloan Kettering Cancer Center. Completed at 1 site in United States. Open to participants aged Up to 75 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2022-12-29.

Sponsored by Memorial Sloan Kettering Cancer Center · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
116
Allocation
Randomized
Ages
Up to 75 Years
Sex
All
01

Study summary

The purpose of this study is to find out if getting a blood stem cell transplant with donor stem cells given over several days is better than getting a blood stem cell transplant with donor stem cells given over 1 day. We want to find out which procedure over will result in improved recovery of blood and immune function after transplant. When donor stem cells are given over various days in mice, the blood and immune system recovery is quicker.

Read the detailed description

This is a complex study that involves various interventions, Intervention #1: Donor Initial Stem Cell Collection; Intervention #2: Stem Cell Product Initial Processing Orders; Intervention #3 Patient Admission and Transplantation; Intervention #4: Stem cell infusion; Intervention #5: Post infusion follow up; Intervention #6: Off Study Patient and Donor Evaluation.

Patients whose donor fails to collect the appropriate number of cells will receive all their cells as a bulk infusion. These patients will continue to receive a transplant on protocol but will be replaced until both arms have reached the target accrual.

02

Conditions studied

  • Leukemia

Keywords

  • CliniMACS-CD34 Reagent System
  • Acute Leukemia
  • Myelodysplastic syndrome
  • Non Hodgkins Lymphoma
  • Hodgkins Disease
  • Multiple Myeloma
  • 12-016
03

In context

Hematologic Neoplasms

1,464 studies on the registry are indexed under Hematologic Neoplasms; 433 are open to participants now.

This study's enrollment of 116 is above the median of 45 across 1,068 interventional studies indexed under Hematologic Neoplasms.

Browse Hematologic Neoplasms studies →

Lead sponsor

Memorial Sloan Kettering Cancer Center is the lead sponsor of 1,930 studies on the registry; 328 are open to participants now.

Of its 129 completed or terminated interventional studies of FDA-regulated products, 66 (51%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 75 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

Inclusion criteria

  • Patients who are considered candidates for an allogeneic stem cell transplantation as treatment for any of the following hematologic disorders:
  • Acute Leukemia
  • Myelodysplastic syndrome
  • Other myeloproliferative disorder (i.e. myelofibrosis, chronic myelomonocytic leukemia, or chronic myelogenous leukemia)
  • Non Hodgkins Lymphoma
  • Hodgkins Disease
  • Multiple Myeloma
  • Age includes from birth to \< 75 years old.
  • Patients must have a Karnofsky (adult) or Lansky (pediatric) Performance Status > 70%
  • Patients must have adequate organ function measured by:
  • Cardiac: asymptomatic or if symptomatic then LVEF at rest must be > 40%
  • Hepatic: \< 5x ULN ALT and \< 1.5 total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
  • Renal: serum creatinine \<1.5 mg/dl or if serum creatinine is outside the normal range, then CrCl > 40 ml/min (measured or calculated/estimated)
  • Pulmonary: asymptomatic or if symptomatic, DLCO > 40% of predicted (corrected for hemoglobin).

Exclusion criteria

Exclusion Criteria:

  • Female patients who are pregnant or breast-feeding.
  • Active viral, bacterial or fungal infection
  • Patient seropositive for HIV-I/II; HTLV -I/II
  • Presence of leukemia in the CNS
  • Candidate for a protocol of higher priority. For the purpose of this study, the following protocols will be considered of higher priority: 10-051

Donor Inclusion Criteria:

  • HLA compatible related or unrelated donor, (i.e. a fully matched unmanipulated grafts or 1-2 HLA allele disparate donor for CD34 selected grafts).
  • Meets criteria outlined in the FACT-approved SOP for "DONOR EVALUATION AND SELECTION FOR ALLOGENEIC TRANSPLANTATION" in the Blood and Marrow Transplant Program Manual, document E-1 see http://mskweb5.mskcc.org/intranet/html/80312.cfm
  • Donor must have adequate peripheral venous catheter access for leukapheresis or must agree to placement of a central catheter.
  • Wt >25kg

Donor Exclusion Criteria:

  • Evidence of active infection (including urinary tract infection, or upper respiratory tract Infection) or viral hepatitis exposure (on screening), unless only HBS Ab+ and HBV DNA negative.
  • Medical or physical reason which makes the donor unlikely to tolerate or cooperate with growth factor therapy and leukapheresis.
  • Factors which place the donor at increased risk for complications from leukapheresis or GCSF therapy (e.g., autoimmune disease, sickle cell trait, symptomatic coronary artery disease requiring therapy).
  • Pregnancy (positive serum or urine β-HCG) or breastfeeding. Women of childbearing age must avoid becoming pregnant while on the study
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
116 participants (actual)

Study arms

  • Active comparator
    bulk SCT

    Patients receive reduced intensity or myeloablative conditioning regimen, GVHD prophylaxis, and undergo T cell depleted or no T cell depleted allogeneic SCT on day 0. After completion of study treatment, patients are followed up every 6-8 weeks for up to 24 months

    Procedure: allogeneic hematopoietic stem cell transplantation · Device: CliniMACS

  • Experimental
    fractionated SCT

    Patients receive reduced intensity or myeloablative conditioning regimen, GVHD prophylaxis, and undergo T cell depleted or no T cell depleted allogeneic SCT on days 0, 2, 4, and 6. After completion of study treatment, patients are followed up every 6-8 weeks for up to 24 months

    Procedure: allogeneic hematopoietic stem cell transplantation · Device: CliniMACS

Interventions

  • Procedureallogeneic hematopoietic stem cell transplantation

    Undergo allogeneic hematopoietic stem cell transplant

  • DeviceCliniMACS
06

What researchers measure

Primary outcomes

  1. Median Time to Engraftment of Neutrophils

    Determine the effects of fractionated vs. bulk stem cell infusions on neutrophil recovery as defined by number of days with an absolute neutrophil count of less than 500 neutrophils per micro liter and time to an absolute neutrophil count (ANC) of 500. Determing the median time to ANC of \>/= 0.5 x 10\^9/L

    Time frame: Up to 100 weeks

Secondary outcomes

  1. Number of Participants Assessed for Toxicities

    Participants will be assessed for toxicities with the NCI-Common Terminology for Adverse Events (CTCAE), version 4.0.

    Time frame: 2 years

  2. Median Time to Platelet Engraftment

    The area under the hematopoietic recovery curve for the factors: ALC, CD4, CD8, and platelet count. The area under the curve will be computed based on recordings at days 30, 60, 100, 180, 365. Determine days to platelet engraftment by platelet count.

    Time frame: Up to 365 days

  3. Overall Survival

    Percentage of participants alive at 24 months

    Time frame: 24 months

  4. Hematopoietic Function on Day 30

    Determine the difference of CD4+ lymphocyte recovery between the two arms on day 30

    Time frame: 30 days

  5. Hematopoietic Function on Day 180

    Determine the difference of CD4+ lymphocyte recovery between the two arms on day 180

    Time frame: 180 days

  6. Hematopoietic Function on Day 360

    Determine the difference of CD4+ lymphocyte recovery between the two arms on day 360

    Time frame: 360 days

07

Results

Posted Dec 29, 2022

Participant flow

Participant flow — Overall Study
MilestoneBulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCT
Started6155
Completed3836
Not completed2319
Withdrew: Inadequate collection2319

Outcome measures

PrimaryMedian Time to Engraftment of Neutrophils

Determine the effects of fractionated vs. bulk stem cell infusions on neutrophil recovery as defined by number of days with an absolute neutrophil count of less than 500 neutrophils per micro liter and time to an absolute neutrophil count (ANC) of 500. Determing the median time to ANC of \>/= 0.5 x 10\^9/L

Time frame:
Up to 100 weeks
Reported as:
Median · days
Median Time to Engraftment of Neutrophils
daysBulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCT
Median Time to Engraftment of Neutrophils11 (9 to 22)11 (9 to 22)
SecondaryNumber of Participants Assessed for Toxicities

Participants will be assessed for toxicities with the NCI-Common Terminology for Adverse Events (CTCAE), version 4.0.

Time frame:
2 years
Reported as:
Count of participants · Participants
Number of Participants Assessed for Toxicities
ParticipantsBulk SCTFractionated SCT
Participants who experienced toxicities3836
Participants who did not experience toxicities2319
SecondaryMedian Time to Platelet Engraftment

The area under the hematopoietic recovery curve for the factors: ALC, CD4, CD8, and platelet count. The area under the curve will be computed based on recordings at days 30, 60, 100, 180, 365. Determine days to platelet engraftment by platelet count.

Time frame:
Up to 365 days
Reported as:
Median · days
Median Time to Platelet Engraftment
daysBulk SCTFractionated SCT
Median Time to Platelet Engraftment18 (9 to 112)18 (4 to 47)
SecondaryOverall Survival

Percentage of participants alive at 24 months

Time frame:
24 months
Reported as:
Number · % of participants alive
Overall Survival
% of participants aliveBulk SCTFractionated SCT
Overall Survival6062
SecondaryHematopoietic Function on Day 30

Determine the difference of CD4+ lymphocyte recovery between the two arms on day 30

Time frame:
30 days
Reported as:
Median · cells/mm^3
Hematopoietic Function on Day 30
cells/mm^3Bulk SCTFractionated SCT
Hematopoietic Function on Day 300 (0 to 1473)6 (0 to 678)
SecondaryHematopoietic Function on Day 180

Determine the difference of CD4+ lymphocyte recovery between the two arms on day 180

Time frame:
180 days
Reported as:
Median · cells/mm^3
Hematopoietic Function on Day 180
cells/mm^3Bulk SCTFractionated SCT
Hematopoietic Function on Day 180179 (0 to 799)111 (0 to 4180)
SecondaryHematopoietic Function on Day 360

Determine the difference of CD4+ lymphocyte recovery between the two arms on day 360

Time frame:
360 days
Reported as:
Median · cells/mm^3
Hematopoietic Function on Day 360
cells/mm^3Bulk SCTFractionated SCT
Hematopoietic Function on Day 360294 (0 to 1076)280 (27 to 928)

Adverse events

Collected over 2 years. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Bulk Stem Cell Transplantation/SCT26/61 (42.6%)21/61 (34.4%)38/61 (62.3%)
Fractionated Stem Cell Transplantation/SCT23/55 (41.8%)12/55 (21.8%)36/55 (65.5%)
Most frequent serious events
Showing 10 of 49
Most frequent serious events
EventBulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCT
HypoxiaRespiratory, thoracic and mediastinal disorders7/612/55
Lung infectionInfections and infestations7/613/55
Respiratory failureRespiratory, thoracic and mediastinal disorders4/610/55
Neutrophil count decreasedInvestigations0/613/55
Death NOSGeneral disorders3/612/55
DiarrheaGastrointestinal disorders3/612/55
DyspneaRespiratory, thoracic and mediastinal disorders3/611/55
FeverGeneral disorders3/610/55
Infections and infestations - Other, specifyInfections and infestations3/611/55
HypotensionVascular disorders1/612/55
Most frequent other events
Showing 10 of 38
Most frequent other events
EventBulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCT
Mucositis oralGastrointestinal disorders30/6117/55
Febrile NeutropeniaBlood and lymphatic system disorders20/6123/55
HyperglycemiaMetabolism and nutrition disorders24/6113/55
Infections and infestations - Other, specifyInfections and infestations23/6112/55
DiarrheaGastrointestinal disorders19/6119/55
Lung infectionInfections and infestations19/617/55
AnorexiaMetabolism and nutrition disorders18/6113/55
NauseaGastrointestinal disorders18/6115/55
Rash maculo-papularSkin and subcutaneous tissue disorders14/6111/55
Alanine aminotransferase increasedInvestigations12/6111/55

Baseline characteristics

Age, Continuous
Age, Continuous(years)Bulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCTTotal
Median59 (13 to 70)59 (24 to 73)59 (13 to 73)
Sex: Female, Male
Sex: Female, Male(Participants)Bulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCTTotal
Female301949
Male313667
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Bulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCTTotal
Hispanic or Latino505
Not Hispanic or Latino5655111
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Bulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCTTotal
American Indian or Alaska Native000
Asian404
Native Hawaiian or Other Pacific Islander000
Black or African American055
White514899
More than one race000
Unknown or Not Reported628
Region of Enrollment
Region of Enrollment(Participants)Bulk Stem Cell Transplantation/SCTFractionated Stem Cell Transplantation/SCTTotal
United States6155116
08

Study locations

1 site
  • Memorial Sloan Kettering Cancer Center
    New York, New York 10065, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Jan 30, 2018

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Dec 29, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01596257
Lead sponsor
Memorial Sloan Kettering Cancer Center
Responsible party
Sponsor
First posted
May 10, 2012
Start date
May 5, 2012
Primary completion
Aug 30, 2021
Completion
Aug 30, 2021
Results posted
Dec 29, 2022
Last update
Dec 29, 2022

Study contacts

Sergio Giralt, MD
principal investigator · Memorial Sloan Kettering Cancer Center
View the source record on ClinicalTrials.gov ↗

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