CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

Phase 2

Completed

• Conditions: Bone Marrow Failure; Leukemia; Immunodeficiencies; Histiocytosis; Sickle Cell Disease; Beta Thalassemia; Inborn Errors of Metabolism; Lymphoma
• Interventions: Drug: Full Intensity with TBI; Drug: Full Intensity; Drug: Reduced Intensity; Drug: Reduced Intensity (Fanconi)

• Registration Number: NCT01049854
• Lead Sponsor: New York Medical College

Brief Summary

CD34+ stem cell selection in children, adolescents and young adults receiving partially matched family donor or matched unrelated adult donor allogeneic bone marrow or peripheral blood stem cell transplant will be safe and well tolerated and be associated with a low incidence of serious (Grade III/IV) acute and chronic graft versus host disease (GVHD).

Detailed Description

The selection of CD34+ cells is associated with the simultaneous depletion of T cells that are responsible for severe acute and chronic graft versus host disease (GVHD). Successful engraftment is reported in adult patients with malignant and non-malignant disease who received CD34+ selected stem cells from HLA-matched or mismatched mobilized peripheral blood (PBSC) or bone marrow.

Study Design:

Selected patients defined in the eligibility criteria will enrolled on this study. Patients will receive one of either full intensity or reduced intensity regimen based on the patient's disease status, organ function and performance and determined by the PI and will have peripheral blood undergo CD34 selection.

Study Timeline

• Study First Submitted: 2008-05-05
• Study First Submitted QC: 2010-01-14
• Study First Posted: 2010-01-15
• Study Start: 2011-09
• Primary Completion: 2017-11
• Status Verified: 2018-08
• Study Completion: 2018-08
• Last Update Submitted: 2018-08-20
• Last Update Posted: 2018-08-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Adequate renal function defined as:Serum creatinine <1.5 x normal, or Creatinine clearance or radioisotope GFR >60 ml/min/m2 or an equivalent GFR as determined by the institutional normal range.
• Adequate liver function defined as:Total bilirubin <1.5 x normal, or SGOT (AST) or SGPT (ALT) <3.0 x normal
• Adequate cardiac function defined as:Shortening fraction >27% by echocardiogram, or Ejection fraction of >47% by radionucleotide angiogram or echocardiogram.
• Adequate pulmonary function defined as:Uncorrected DLCO >50% by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry >94% on room air.

Eligibility for Reduced Intensity Regimen:

• Adequate renal function defined as:Serum creatinine 2.0 x normal, or creatinine clearance or radioisotope GFR > 40 ml/min/m2 or an equivalent GFR as determined by the institutional normal range.
• Adequate liver function defined as:Total bilirubin < 2.5 x normal; or SGOT (AST) or SGPT (ALT) < 5.0 x normal.
• Adequate cardiac function defined as:Shortening fraction of >25% by echocardiogram, or Ejection fraction of >40% by radionuclide angiogram or echocardiogram.
• Adequate pulmonary function defined as:DLCO >35% by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry >94% in room air.

Read More

Exclusion Criteria

• Pregnancy/Breast Feeding: Females who are pregnant or breast-feeding are not eligible.
• Infection: Patients with documented uncontrolled infection at the time of study entry are not eligible.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Thiotepa/Cyclophosphamide/ATG	Full Intensity with TBI	Full intensity with TBI
Busulfan/Melphalan/ATG	Full Intensity	Full intensity without TBI
Busulfan/Fludarabine/Alemtuzumab	Reduced Intensity	Reduced Intensity Chemotherapy
Fludarabine/Cyclophosphamide/ATG	Reduced Intensity (Fanconi)	Reduced Intensity Chemotherapy for Fanconi Anemia

Primary Outcome Measures

Name	Time	Method
The safety CD34+ stem cell selection	100 days	serious adverse events will be monitored post transplant to determine if there is an increase vs. historical data related to the CD34+ selection

Secondary Outcome Measures

Name	Time	Method
Immune reconstitution (T, B, DC) following CD34+ selection	3 years	immune subsets will be drawn post transplant to determine the rate of reconstitution post CD34+ transplant to determine if this process increases or decreases the reconstitution time.

Trial Locations

Locations (1)

New York Medical College; 🇺🇸 Valhalla, New York, United States