Safety Study of Unlicensed IND Cord Blood Units Manufactured by the National Cord Blood Program for Unrelated Transplantation

New York Blood Center

Status and phase: Enrolling; Phase 2

Conditions: Infusion Reactions

Treatments: Biological: unlicensed CBU

Study type: Interventional

Funder types: Other

Identifiers

NCT01656603

6637-01

Details and patient eligibility

About

This study will evaluate the safety of infusion of the investigational cord blood units by carefully documenting all infusion-related problems.

Full description

The primary aim of this study is to examine the safety of administration of the unlicensed investigational NCBP hematopoietic progenitor cell-cord blood (HPC-CORD BLOOD) products in a multi-institution setting. Therefore, the study will evaluate prospectively the incidence of serious adverse reactions as well as the incidence of all infusion related reactions after administration of the unlicensed, investigational NCBP CBU.

Definitions of Infusion-related adverse reactions:

Mild - Moderate: reactions during or after the infusion of the cord blood (CB) product that require some medical intervention but do not affect the overall patient status or outcome.

Severe: serious, life-threatening or fatal infusion reactions, requiring major medical intervention. These include: anaphylactic shock, acute cardiac, pulmonary or renal failure, seizures, patient transfer to the Intensive Care Unit, or death within 48 hours of the CB infusion. Adverse Reactions will also be classified by grade, according to the Common Terminology Criteria for Adverse Events v4.0 (CTCAE).

Enrollment: 9,999 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Diagnosis: Patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment.
2. Patients: Patients of any age and either gender
3. Cord blood product manufactured by the NCBP (at least one, if the graft contains more than one units)

Exclusion criteria

1. Patients who are receiving licensed cord blood products (only)
2. Patients who are receiving unlicensed cord blood products from other banks (only)
3. Patients who are transplanted at non-US transplant centers
4. Patients who are receiving cord blood products that will be "manipulated" post-thaw (e.g., ex vivo expansion, incubation in vitro, etc.)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9,999 participants in 1 patient group

unlicensed CBU

Experimental group

Description:

The Principal Investigators will be the transplant physicians at participating US transplant centers

Treatment:

Biological: unlicensed CBU