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Acute Lymphoblastic LeukemiaPhase 2NCT00968864

T-cell Depleted Alternative Donor Transplantation

A Phase 2 study testing CliniMACS® (T cell depletion) in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by Wake Forest University Health Sciences at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages Up to 30 years
What is being tested?
CliniMACS® (T cell depletion)
Type of treatment
Medical device
Could I receive a placebo?
No, everyone receives an active treatment
How many people
53 participants
Where
1 siteUnited States
Study dates
Aug 2009to Nov 2016
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 Up to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age < 30 years
  • Patient must have a malignant or non-malignant disease that can benefit from alternative stem cell transplantation. Examples include acute and chronic leukemias, myelodysplastic syndrome, lymphoma, severe acquired and congenital cytopenias, white and red blood cell abnormalities, and immunodeficiencies.
  • Patients with acute lymphoblastic leukemia must be in morphological remission (< 5% blasts) at the time of transplant. Patients with acute non-lymphocytic leukemia will preferably be in morphologic remission but may be enrolled when aplastic after chemotherapy or with < 20% blasts. Patients with lymphoma must be in complete or close to complete remission (if residual adenopathy, PET scan must be negative or only have slight uptake, eg. SUV < 2).
  • Patients must lack a healthy HLA-identical related donor of at least one year of age.
  • Patient must have a mismatched related or an unrelated donor who is:
  • Able to receive G-CSF and undergo apheresis either through placement of catheters in antecubital veins or a temporary central venous catheter,
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  • Healthy,
  • Willing,
  • For recipients of an unrelated donor transplant, recipient eligibility will be restricted as follows if in the judgment of the recipients' transplant physician, the recipient cannot receive a transplant with combined positive and negative fractions as described in Section 6.1.3.2 or an unmanipulated PBSC product.
  • Meets eligibility criteria for donors.
  • If only one mismatched related relative is available, an acceptable unrelated donor must be identified as a backup.
  • Patient or authorized guardian must sign informed consent for this study.

Who cannot join

  • Patient with an anticipated life expectancy of < 1 month
  • Active infectious hepatitis or CMV infection
  • HIV or HTLV-I/II infection
  • Serious infection (bacterial, fungal, viral) within the last 4 weeks
  • Cardiac ejection fraction < 45%; can be lower if patient is not in clinical cardiac failure and a reduced intensity conditioning regimen is used.
  • Creatinine clearance <60 ml/min/1.72 m2; can be lower if a reduced intensity conditioning regimen is used.
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  • Pulmonary diffusion capacity (adjusted for Hgb), FEV1, or FVC <60% of predicted or O2 sat < 94% if unable to perform PFTs; can be lower if a reduced intensity conditioning regimen is used.
  • Serum ALT > 3 x upper limit of normal (can be up to 5 x upper limit of normal if a reduced intensity conditioning regimen is used) or bilirubin > 2. The bilirubin criteria for sickle cell disease patients is direct bilirubin >2x upper limit of normal.
  • Performance score (Lansky/Karnofsky) < 50
  • Any condition that compromises compliance with the procedures of this protocol, as judged by the principal investigator.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Levine Children's Hospital, Carolinas Medical Center

    Charlotte, North Carolina, 28204

Other trials in United States

About this study

Official title: A Phase II Study Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients

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.

Read the detailed description

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.

Being tested
CliniMACS® (T cell depletion)
Keywords
T cell depleted, Matched unrelated donors, Haplocompatible donors
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic Syndromes, Lymphoma, Bone Marrow Failure Disorders, Hemoglobinopathies, Immunologic Deficiency Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CliniMACS® (T cell depletion)Device

    Also called CliniMACS device, 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.

Study groups in NCT00968864
Study groupRoleWhat they receive
CliniMACS® (T cell depletion)ExperimentalRecipients will receive T cell-depleted PBSC from eligible donors after receiving conditioning therapy using CliniMACS® device.
Receives: Device: CliniMACS® (T cell depletion)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Severe Graft vs. Host Disease (GVHD).

    Severe GVHD defined as grade III/IV GVHD.

    Measured: Within 30 days after stem cell transplant

Secondary outcomes (8)

  1. Number of Participants With Engraftment and Time to Engraftment

    Engraftment was measured as time to absolute neutrophil count >500

    Measured: Within 28 days after stem cell transplant

  2. Number of Participants With Post-transplant Infections

    Measured: 1 year

  3. Number of Participants With EBV-related Post Transplant Lymphoproliferative Disorder (PTLD)

    Measured: 5 years

  4. Number of Participants With Post-transplant Leukemia Relapse

    Measured: 5 years

  5. 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.

    Measured: 2 year

  6. Number of Participants With Transplant-related Toxicities

    Measured: 1 year

  7. Overall Survival

    Measured: 2 years

  8. 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.

    Measured: Length of the trial (5 years)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Aug 2009 (estimated)
  2. Study startAug 2009
  3. Primary completionNov 2016
  4. Study completionNov 2016

Why it stopped: Sponsor/ PI leaving institution, no plans to continue this research at this time

Resources

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
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
53 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00968864
Sponsor's protocol ID
LCH BMT 09-01
Status last verified
Oct 2017
First submitted
28 Aug 2009
Last updated
22 Apr 2022
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Gilman AL, Eckrich MJ, Epstein S, Barnhart C, Cannon M, Fukes T, Hyland M, Shah K, Grochowski D, Champion E, Ivanova A. Alternative donor hematopoietic stem cell transplantation for sickle cell disease. Blood Adv. 2017 Jun 28;1(16):1215-1223. doi: 10.1182/bloodadvances.2017005462. eCollection 2017 Jul 11.

    PubMed 29296761

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CliniMACS® (T cell depletion) in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by Wake Forest University Health Sciences 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 12 inclusion and 10 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 22 Apr 2022. 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?

Yes. Results are posted in the registry (first posted 8 Nov 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants With Severe Graft vs. Host Disease (GVHD). (time frame: Within 30 days after stem cell transplant). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00968864, which also lists 1 linked publication.

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 22 Apr 2022; AzTrials last refreshed its copy on 30 Oct 2026.

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