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Safety Study of Cord Blood Units for Stem Cell Transplants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01861093
Recruitment Status : Recruiting
First Posted : May 23, 2013
Last Update Posted : July 24, 2019
Sponsor:
Collaborators:
National Cancer Institute (NCI)
National Institute of Allergy and Infectious Diseases (NIAID)
National Cord Blood Program, New York Blood Center
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Tracking Information
First Submitted Date  ICMJE May 21, 2013
First Posted Date  ICMJE May 23, 2013
Last Update Posted Date July 24, 2019
Actual Study Start Date  ICMJE October 16, 2015
Estimated Primary Completion Date November 30, 2037   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 21, 2013)
Examine the safety of administration of the unlicensed investigational NCBP HPC-CORD Blood products [ Time Frame: 30 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01861093 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety Study of Cord Blood Units for Stem Cell Transplants
Official Title  ICMJE A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs) Manufactured by the National Cord Blood Program (NCBP) and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients
Brief Summary

Background:

- Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants have been performed in children and adults for blood cancers and other diseases in the world. These transplants have helped save lives and improve treatments. However, not all available units of cord blood have been collected, stored, and licensed according to specific government requirements. These unlicensed units can still be used in transplant, but they can only be given as part of specific research studies. This study will evaluate the safety of giving these unlicensed units by recording any problems that may occur during and after giving the cord blood.

Objectives:

- To test the safety and effectiveness of unlicensed cord blood units in people who need stem cell transplants.

Eligibility:

- Individuals who are scheduled to have a stem cell transplant.

Design:

  • Participants will be screened with a medical history and physical exam.
  • Participants will receive the cord blood unit as part of their stem cell transplant procedure. The transplant will be performed according to the current standard of care for the procedure.
  • After the transplant, participants will be monitored for up to 1 year. Any problems or side effects from the transplant will be treated as necessary. All outcomes will be reported to the National Cord Blood Program and to the Center for International Blood and Marrow Transplant.
Detailed Description

Background

  • The requirement for licensure of human cell and blood products 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. As of October 20, 2011, those units of cord blood 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. In addition to the licensure guidance, the FDA published a guidance in August 2011 titled Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.
  • This is a multi-center study of unlicensed, investigational cryopreserved cord blood units (CBUs) manufactured by the National Cord Blood Program (NCBP) which provides access and distribution on an Investigational New Drug (IND) application #6637 to sites with locally IRB-approved protocols for unrelated hematopoietic stem cell transplantation of pediatric and adult patients.

Objectives

-The primary objective of this study is to examine the safety of administration of the unlicensed investigational NCBP HPC-CORD Blood products, evaluating prospectively the incidence of serious adverse reactions as well as the incidence of all infusion related reactions after administration of unlicensed, investigational NCBP CBU.

Secondary Objectives:

In patients receiving a non-licensed NCBP CBU (HPC-CORD BLOOD):

  • Assess incidence of transmission of infections
  • Assess incidence of graft rejection
  • Assess incidence of neutrophil engraftment >500 /ul
  • 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 platelet engraftment of >20,000/ul and >50,000/ul
  • Determine CBU-derived engraftment

Eligibility Criteria

Inclusion Criteria

  • Patients of any age or either gender with indications for receipt of investigational HPC-CORD BLOOD who are participating in an NIH-IRB approved clinical trial for unrelated hematopoietic stem cell transplantation.
  • Signed informed consent (and assent when applicable).

Exclusion Criteria

  • Patients who are receiving licensed CB products (only)
  • Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)

Design

This study is a multi-NIH institute endeavor designed to allow NHLBI, NCI and NIAID investigators access investigational HPC-CORD BLOOD for patients participating in NIH-IRB approved clinical trials for unrelated hematopoietic stem cell transplantation. Treatment, including pre-treatment conditioning and GVHD prophylaxis will occur per the institute s NIH-IRB approved clinical trial or the transplant center s specifications.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Aplastic Anemia
  • Leukemia
  • Myelodysplastic Syndrome (MDS)
  • Lymphoma
Intervention  ICMJE Biological: Cord Blood Units
Transplant
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 21, 2013)
500
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 30, 2037
Estimated Primary Completion Date November 30, 2037   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE
  • INCLUSION CRITERIA:
  • Patients of any age or either gender with indications for receipt of investigational HPC-CORD BLOOD who are participating in an NIH-IRB approved clinical trial for unrelated hematopoietic stem cell transplantation.
  • Signed informed consent (and assent when applicable).

EXCLUSION CRITERIA:

  • Patients who are receiving licensed CB products (only)
  • Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jennifer S Wilder, R.N. (301) 451-3722 jw621w@nih.gov
Contact: Richard W Childs, M.D. (301) 451-7128 childsr@nhlbi.nih.gov
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01861093
Other Study ID Numbers  ICMJE 130116
13-H-0116
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
Study Sponsor  ICMJE National Heart, Lung, and Blood Institute (NHLBI)
Collaborators  ICMJE
  • National Cancer Institute (NCI)
  • National Institute of Allergy and Infectious Diseases (NIAID)
  • National Cord Blood Program, New York Blood Center
Investigators  ICMJE
Principal Investigator: Richard W Childs, M.D. National Heart, Lung, and Blood Institute (NHLBI)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date December 6, 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP