CD34+ Stem Cell Infusion to Augment Graft Function

NCT01856582 | Terminated Phase 2 Results

• 1 other identifier: 2010-2344
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine if infusing additional special donor cells will help to improve graft or immune function in previously transplanted children with immune deficiencies and bone marrow failures.

Conditions

Waning Donor Chimerism; Waning Immune Function; Primary Immunodeficiency Disease(s); Bone Marrow Failure

Keywords

Primary Immunodeficiency Disease(s); Mixed Donor Chimerism; bone marrow failure syndromes; allogeneic hematopoietic stem cell transplant (HSCT); children

Outcome Measures

Primary Outcomes (1)

• Number of Participants Who Showed Successful Augmentation of Graft Function

  Successful augmentation of graft function achieved if donor chimerism is doubled compared to the value immediately pre-infusion at 12 months.

  12 months

Study Arms (1)

CD34+ selected stem cell infusion

EXPERIMENTAL

An infusion of selected CD34+ stem cells will be given without any preparative regimen.

Biological: CD34+

Interventions

CD34+ BIOLOGICAL

CD34+ cells are selected using the CliniMACS System; without preparative regimen

CD34+ selected stem cell infusion

Eligibility Criteria

• Age: Up to 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• To be eligible for this protocol, patients must have the following:
• Primary immunodeficiency (e.g. SCID, Wiskott-Aldrich and/or other more rare conditions and other bone marrow failure syndromes) with prior allogeneic stem cell transplant.
• Waning donor chimerism or immune function that is inadequate to correct their disease or clinical condition, for which primary transplant was given, as determined by their attending physician.
• Available primary donor.
• Must not have other organ dysfunction deemed by the attending physician to preclude this procedure.
• Age \< 35 years at time of transplant
• One of the following must be true:
• Patients must have evidence of persistent or recurrent immunodeficiency or thrombocytopenia.
• OR-
• Primary immunodeficiency disease with known potential to progress to malignant condition if untreated.
• OR-
• Debilitating secondary disease known to be a consequence of inadequate immune response to known agent or pathogen, uncontrollable by other available medical therapies (e.g. third patient described on page 5).

You may not qualify if:

• Absence of an available original donor
• Failure to sign consent form, or inability to undergo informed consent process
• Pregnant or lactating female
• Uncontrolled GVHD

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Hoxworth Blood Center: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

• Chandra S, Bleesing JJ, Jordan MB, Grimley MS, Khandelwal P, Davies SM, Edwards S, Leemhuis T, Marsh RA. Post-Transplant CD34+ Selected Stem Cell "Boost" for Mixed Chimerism after Reduced-Intensity Conditioning Hematopoietic Stem Cell Transplantation in Children and Young Adults with Primary Immune Deficiencies. Biol Blood Marrow Transplant. 2018 Jul;24(7):1527-1529. doi: 10.1016/j.bbmt.2018.03.013. Epub 2018 Mar 16.

  PMID: 29555312 RESULT

MeSH Terms

Conditions

Bone Marrow Failure Disorders

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Rebecca Marsh, MD/Associate Professor of Pediatrics
• Organization: Cincinnati Children's Hospital Medical Center

Rebecca.Marsh@cchmc.org

513-803-9063

Study Officials

• Rebecca Marsh, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2013
• First Posted: May 17, 2013
• Study Start: October 1, 2010
• Primary Completion: August 15, 2018
• Study Completion: August 15, 2018
• Last Updated: November 22, 2019
• Results First Posted: October 22, 2019

Record last verified: 2018-12

Locations

US (1)