Using a special method to prepare stem cells from a close relative for transplant

Ex Vivo T-Cell Depletion of Mobilized Peripheral Blood Stem Cells Via CD34-Selection (EXCESS)

This study is testing a new way to prepare stem cells from a family member for patients who need a transplant but don’t have a perfect match, to see if it helps them do better after the procedure.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 241 (estimated)
• Ages: N/A to 70 Years
• Sex: All
• Sponsor: Baylor College of Medicine (Academic / other)
• Locations: 2 sites (Houston, Texas and 1 other locations)
• Trial ID: NCT01189786 on ClinicalTrials.gov

What this trial studies

This study involves collecting stem cells from a close relative of patients who require a stem cell transplant but do not have a perfectly matched donor. The collected white blood cells will be treated with a CD34 antibody and separated using the CliniMACS CD34 Reagent System, an investigational device. Participants will undergo follow-up evaluations at specific intervals post-transplant to monitor their health and any potential complications. The study aims to improve outcomes for patients receiving haploidentical stem cell transplants.

Who should consider this trial

Good fit: Ideal candidates include patients aged between birth and 70 years who require an allogeneic stem cell transplant and have a close relative as a donor.

Not a fit: Patients with active, severe graft-versus-host disease, significant infections, or severe organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could enhance the effectiveness of stem cell transplants for patients with limited donor options.

How similar studies have performed: Other studies using similar CD34+ selection methods have shown promise, indicating potential for success in this approach.

Eligibility criteria

Inclusion criteria for Stem Cell Transplant WITH Conditioning (COHORT 1)

1. Patient requiring allogeneic SCT
2. Age between birth and 70 years
3. Patient and/or responsible person able to understand and sign consent

Exclusion criteria for Stem Cell Transplant WITH Conditioning (COHORT 1)

1. Active, acute GvHD \> grade II or extensive, chronic GvHD
2. Severe life, threatening infection
3. Pulmonary dysfunction (FEV1, FVC or DLCO 40% of predicted or 3 SD below normal)
4. Cardiac dysfunction (LVSF less than 25%)
5. Psychiatric disturbance
6. Lansky or Karnofsky score \< 50%
7. The presence of severe hepatic disease (direct bilirubin \>3x upper limit of normal and AST \> 5x upper limit of normal).
8. Creatinine \> 3x normal
9. Known HIV Positivity
10. Pregnancy

Inclusion Criteria for CD34+ Topoff WITHOUT conditioning (COHORT 2)

1. Allogeneic SCT Recipient requiring additional cellular therapy
2. Age between birth and 70 years
3. Patient and/or responsible person able to understand and sign consent
4. At least ONE of the following must be answered YES for a patient to be eligible to receive CD34+ topoff:

   1. Evidence of mixed chimerisms (less than 95% donor cells)
   2. Evidence of poor bone marrow function (bone marrow cellularity less than 50% with at least one cytopenia)
   3. Relapsed or persistent disease

Exclusion criteria for CD34+ Topoff WITHOUT conditioning (COHORT 2)

1. Active, acute GvHD \> grade II or extensive, chronic GvHD
2. Severe life, threatening infection
3. Known HIV positivity
4. Pregnancy

Inclusion Criteria for CD34+ Topoff WITH conditioning (COHORT 3)

1. Allogeneic SCT Recipient requiring additional cellular therapy
2. Age between birth and 70 years
3. Patient and/or responsible person able to understand and sign consent
4. At least ONE of the following must be answered YES for a patient to be eligible to receive CD34+ topoff:

   1. Evidence of mixed chimerisms (less than 95% donor cells)
   2. Evidence of poor bone marrow function (bone marrow cellularity less than 50% with at least one cytopenia)
   3. Relapsed or persistent disease

Exclusion criteria for CD34+ Topoff WITH Conditioning (COHORT 3)

1. Active, acute GVHD \> grade II or extensive, chronic GvHD
2. Severe life, threatening infection
3. Pulmonary disfunction (FEV1, FVC or DLCO 40% of predicted or 3 SD below normal)
4. Cardiac dysfunction (LVSF less than 25%)
5. Psychiatric disturbance
6. Lansky or Karnofsky score \< 50%
7. The presence of severe hepatic disease (direct bilirubin \> 3x upper limit of normal and AST \> 5x upper limit of normal)
8. Creatinine \> 3x normal
9. Known HIV positivity
10. Pregnancy

Where this trial is running

Houston, Texas and 1 other locations

• Houston Methodist Hospital — Houston, Texas, United States (Recruiting)
• Texas Children's Hospital — Houston, Texas, United States (Recruiting)

Study contacts

• Principal investigator: Robert Krance, MD — Baylor College of Medicine
• Study coordinator: Robert Krance, MD

Conditions Stem Cell Transplant; Allogeneic; Haploidentical Stem Cell Transplant; CD34+ Selection; CliniMACS CD34 Reagent system