CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01966367
• Date of registration: 17/10/2013
• Prospective Registration: No
• Primary sponsor: Diane George
• Public title: CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
• Scientific title: CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant Disease
• Date of first enrolment: March 2013
• Target sample size: 37
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT01966367
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Diane George, MD
• Affiliation: Columbia University

Key inclusion & exclusion criteria

Inclusion Criteria:

- General Eligibility (All Patients)

- Patient must be < or = 40 years of age. Patients with sickle cell anemia must be
at least 2 years of age.

- Patient or the patient's legally authorized guardian must be fully informed about
their illness and the investigational nature of the study protocol (including
foreseeable risks and possible side effects) and must sign an informed consent in
accordance with the institutional policies approved by the U.S. Department of
Health and Human Services.

- Approval for the use of this treatment protocol by the individual institution's
Human Rights Committee must be obtained, in accordance with the institutional
assurance policies of the U. S. Department of Health and Human Services.

- Human leukocyte antigen (HLA) typing will be performed by high-resolution
molecular DNA typing for HLA Class I A, B, and C and HLA Class II DRB1 and DQB1
alleles.

- Unrelated donor: An 8/10, 9/10 or 10/10 matched unrelated adult donor (MUD)
will be required for study entry.

- Related Donor: A 5/10, 6/10, 7/10, 8/10, 9/10 or 10/10 matched (or partially
matched) family donor will be required for study entry.

- Non-malignant Disorders per protocol.

- Hemoglobinopathies per protocol.

- Requirement for CD34+ stem cell selection for a second infusion of stem cells
following an allogeneic stem cell transplant from a related or unrelated adult
donor.

- Additional eligibility for patients with non-malignant disorders receiving
myeloablative conditioning

- Adequate renal function as determined by the institutional normal range.

- Adequate liver function per protocol.

- Adequate cardiac function defined by radionucleotide angiogram or echocardiogram.

- Adequate pulmonary function 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.

- Additional eligibility for patients with non-malignant disorders receiving reduced
intensity conditioning

- Adequate renal function as determined by the institutional normal range.

- Adequate liver function per protocol.

- Adequate cardiac function per protocol.

- Adequate pulmonary function per protocol.

Exclusion Criteria:

- Patients with documented uncontrolled infection at the time of study entry are not
eligible.

- Pregnancy/Breast-Feeding Females who are pregnant or breast feeding at the time of
study entry are not eligible.

-- The following additional exclusion criteria for patients with sickle cell anemia
the following exclusion criteria also apply

- Patients with bridging fibrosis or cirrhosis of the liver.

- Uncontrolled bacterial, viral or fungal infection in the past month.

- Seropositivity for HIV.

- Patients who have received prior hematocrit (HCT) within three months of enrollment
for reduced intensity regimen and within six months for myeloablative regimen/reduced
toxicity regimens.



Age minimum: N/A
Age maximum: 40 Years
Gender: All

Health Condition(s) or Problem(s) studied

Bone Marrow Failure Syndrome

Diamond-Blackfan Anemia

Acquired Immunodeficiency Syndromes

Lymphohistiocytosis

Macrophage Activation Syndrome

Severe Aplastic Anemia

Sickle Cell-beta-thalassemia

Amegakaryocytic Thrombocytopenia

Hemoglobinopathies

Histiocytic Syndrome

Langerhans Cell Histiocytosis (LCH)

Schwachman Diamond Syndrome

Severe Congenital Neutropenia

Familial Hemophagocytic Lymphocytosis

Sickle Cell Disease

Primary Immunodeficiency Syndromes

Intervention(s)

Biological: CD34 Stem Cell Selection Therapy

Primary Outcome(s)

Incidence of acute graft versus host disease (GVHD) [Time Frame: 100 days]

Secondary Outcome(s)

Incidence of infectious complications [Time Frame: 2 years]

Incidence of primary graft failure [Time Frame: 1 year]

Survival Rate [Time Frame: 5 years]

Incidence of secondary graft failure [Time Frame: 1 year]

Time to neutrophil and platelet engraftment [Time Frame: 1 year]

Time to immune reconstitution [Time Frame: 2 years]

Secondary ID(s)

AAAL0156

Source(s) of Monetary Support

Please refer to primary and secondary sponsors