CD34+ Stem Cell Infusion to Augment Graft Function

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01856582
• Date of registration: 15/05/2013
• Prospective Registration: No
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: CD34+ Stem Cell Infusion to Augment Graft Function
• Scientific title: Post Transplant CD34+ Selected Stem Cell Infusion to Augment Graft Function in Children With Primary Immunodeficiency Diseases and Bone Marrow Failure Syndromes
• Date of first enrolment: October 2010
• Target sample size: 23
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01856582
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Rebecca Marsh, MD
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion criteria

Inclusion Criteria:

To be eligible for this protocol, patients must have the following:

1. 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.

2. 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.

3. Available primary donor.

4. Must not have other organ dysfunction deemed by the attending physician to preclude
this procedure.

5. Age < 35 years at time of transplant

6. 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).

Exclusion Criteria:

1. Absence of an available original donor

2. Failure to sign consent form, or inability to undergo informed consent process

3. Pregnant or lactating female

4. Uncontrolled GVHD



Age minimum: N/A
Age maximum: 35 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Immunodeficiency Disease(s)

Waning Donor Chimerism

Bone Marrow Failure

Waning Immune Function

Intervention(s)

Biological: CD34+

Primary Outcome(s)

Number of Participants Who Showed Successful Augmentation of Graft Function [Time Frame: 12 months]

Secondary ID(s)

2010-2344

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Hoxworth Blood Center

Results

• Results available: Yes
• Date Posted: 22/10/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01856582