Transplants With Unlicensed Preserved Cord Blood

Hematologic Diseases Clinical Trial

Official title:

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01553461
• Other study ID #: 120064
• Secondary ID: 12-H-0064
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: February 21, 2012
• Est. completion date: October 20, 2036

Study information

• Verified date: April 16, 2021
• Source: National Institutes of Health Clinical Center (CC)
• Contact: Jennifer S Wilder, R.N.
• Phone: (301) 451-3722
• Email: jw621w[@]nih.gov
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Background:

• Cord blood transplants can treat cancers and other diseases in children and adults. The U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under certain safety standards. However, most available cord blood units were collected before the FDA set these standards. These units may not meet FDA standards, but they do meet similar standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that do not meet the new FDA standards may be used for transplants only as part of a research study. Doctors want to allow people who need transplants to receive cord blood that meets NMDP standards but may not meet FDA standards.

Objectives:

• To allow selected cord blood units that do not meet current FDA standards to be used for transplant.

Eligibility:

• Individuals who need cord blood units for transplant, and who best match cord blood units that are not FDA-licensed.

Design:

• Participants will provide consent to receive cord blood that meets NMDP standards but may not meet FDA standards.

• Participants will remain on the study for observation for up to 1 year after transplant, or until they withdraw from the study for personal or medical reasons....

Description:

Background

The requirement for licensure became part of the FDA final rules for Human Cells, Tissues, and Tissue-Based Products (HCT/Ps) (March 29, 2004 and May 25, 2005) in an effort to prevent transmission of communicable disease, minimize contamination and preserved integrity and function during processing, outline safety and effectiveness requirements for cells from unrelated donors or when HCT/Ps are more than minimally manipulated, assure labeling is clear accurate and not misleading and monitor and communicate with industry via establishment registration. However, as of October 20, 2011, those units that do not meet the manufacturing requirements for licensure can only be distributed for transplantation if the transplant will occur under an IND research protocol. These units are in current and future inventory at domestic and international cord blood banks that cannot be demonstrated to meet licensing requirements. In addition to the licensure guidance, the FDA published a guidance in August 2011 entitled Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.

This study is an access and distribution protocol conducted by the National Marrow Donor Program (NMDP) for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients, in which sites wishing to receive NMDP unrelated cord blood units for treatment or research must participate with an locally IRB- approved protocol.

Primary Objectives

The primary objective of this study is to examine the incidence of neutrophil recovery of greater than or equal to 500/mm(3) after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed.

Secondary Objectives

In patients receiving non-licensed CBU

• Assess incidence of graft rejection

• Assess incidence of transmission of infection

• Assess incidence of serious infusion reaction Determine 1 year survival after cord blood transplantation

• Assess cumulative incidence of acute graft vs. host disease (GVHD) grades II to IV and grades III to IV

• Assess cumulative incidence of chronic GVHD

• Determine CBU-derived engraftment

Eligibility Criteria

Inclusion Criteria

• Patients of any age with FDA-specified indications

• Signed informed consent (and assent when applicable) obtained prior to study enrollment

Exclusion Criteria

• Patients who have licensed CBUs available

• Cord blood transplant recipients at international transplant centers

• Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)

• Patients whose selected unlicensed CBU(s) will be more than minimally manipulated

Design

-Treatment description, including pre-transplant conditioning and GVHD prophylaxis, will occur per each transplant center s specification.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: October 20, 2036
• Est. primary completion date: October 20, 2036
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 99 Years

Eligibility

• INCLUSION CRITERIA:

• Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment

• Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment

• Pediatric and adult patients of any age

EXCLUSION CRITERIA:

• Patients who are receiving only licensed CBUs

• Cord blood transplant recipients at international transplant centers

• Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)

• Patients whose selected unlicensed CBU(s) will be more than minimally manipulated

Related Conditions & MeSH terms

• Anemia, Aplastic
• Hematologic Diseases
• Hematologic Neoplasms
• Myelodysplastic Syndrome (MDS)
• Myelodysplastic Syndromes
• Preleukemia
• Severe Aplastic Anemia

Intervention

Biological:
• Cord Blood Transplant
Unlicensed CBU

Locations

Country	Name	City	State
United States	National Institutes of Health Clinical Center, 9000 Rockville Pike	Bethesda	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

References & Publications (3)

Kurtzberg J, Laughlin M, Graham ML, Smith C, Olson JF, Halperin EC, Ciocci G, Carrier C, Stevens CE, Rubinstein P. Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med. 1996 Jul 18;335(3):157-66. — View Citation

Laughlin MJ, Barker J, Bambach B, Koc ON, Rizzieri DA, Wagner JE, Gerson SL, Lazarus HM, Cairo M, Stevens CE, Rubinstein P, Kurtzberg J. Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N Engl J Med. 2001 Jun 14;344(24):1815-22. — View Citation

Wagner JE, Rosenthal J, Sweetman R, Shu XO, Davies SM, Ramsay NK, McGlave PB, Sender L, Cairo MS. Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease. Blood. 1996 Aug 1;88(3):795-802. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To examine the incidence of neutrophil recovery of greater than or equal to 500/mm3 after unlicensed CBU transplant.	incidence of neturophil recovery of greater than or equal to 500/mm3 after unlicensed CBU transplant.	100 days to 1 year
Secondary	Assess incidence of graft rejection, transmission of infection, serious infusion reactions. Determine 1 year survival. Assess cumulative incidence of acute GVHD (grades II to IV), (grades III to IV), and incidence of chronic GVHD.

External Links

•   NIH Clinical Center Detailed Web Page