Providing Access to Cord Blood Units for Transplants

NCT01479582 | Withdrawn Phase 2

• 2 other identifiers: 12002712-C-0027
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

Background: \- Cord blood banks have been set up to collect and store umbilical cord blood for transplants. These transplants are used to treat different types of cancer. In October 2011, the Food and Drug Administration (FDA) began considering cord blood as a biological drug. Most of the cord blood units currently available in cord blood banks in the United States and other countries were collected before the FDA set these new standards. The units meet standards set by the National Marrow Donor Program (NMDP), but they were not collected, tested, or stored exactly according to FDA standards. As a result, the new guidelines state that they may only be used for transplant if the transplant is done as part of a study. Researchers have set up a study to provide these cord blood units to recipients and to study the effects of their use. Objectives:

• To provide access to cord blood units for recipients whose best choice for a unit meets NMDP but not FDA standards.
• To study the effects of these cord blood transplants. Eligibility: \- Individuals who need to have a cord blood transplant to treat certain types of cancer. Design:
• Participants will be screened with a physical exam, medical history. They will also have blood tests and imaging studies.
• Participants will have the cord blood transplant and allow their medical data to be collected by the study researchers.

Conditions

Chronic Myelogous Leukemia/Other Leukemia; Acute Leukemias; MDS/MPS; Multiple Myeloma; Non-Hodgkin Lymphoma

Keywords

Adult and Pediatric; Unlicensed Cord Blood Unit IND; NMDP; Transplant; Hematologic Malignancies; Leukemia; Multiple Myeloma; Non-Hodgkin Lymphoma; CML; Chronic Myelogenous Leukemia; Myelodyplastic Syndrome; MDS; Thalassemia; Lysosomal Storage Diseases

Outcome Measures

Primary Outcomes (1)

• The primary aim of this study is to examine the incidence of neutrophil recovery of greater than or equal to 500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licen...

Secondary Outcomes (5)

• 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 aGVHD vs cGVHD.

Interventions

Access to unlicensed cord blood units DRUG

Eligibility Criteria

• Age: Up to 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with FDA-specified indications:
• Hematological malignancies
• Certain lysosomal storage and peroxisomal enzyme deficiency disorders
• Hurler syndrome (MPS I)
• Krabbe Disease (Globoid Leukodystrophy)
• X-linked Adrenoleukodystrophy
• Primary immunodeficiency diseases
• Bone marrow failure
• Beta-thalassemia
• Signed informed consent (and signed assent, if applicable)
• Pediatric and adult patients of any age

You may not qualify if:

• Patients who are receiving only licensed CBUs
• Cord blood transplant recipients at international transplant centers

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Related Publications (3)

• 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. doi: 10.1056/NEJM200106143442402.

  PMID: 11407342 BACKGROUND

• 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. doi: 10.1056/NEJM199607183350303.

  PMID: 8657213 BACKGROUND

• Rocha V, Labopin M, Sanz G, Arcese W, Schwerdtfeger R, Bosi A, Jacobsen N, Ruutu T, de Lima M, Finke J, Frassoni F, Gluckman E; Acute Leukemia Working Party of European Blood and Marrow Transplant Group; Eurocord-Netcord Registry. Transplants of umbilical-cord blood or bone marrow from unrelated donors in adults with acute leukemia. N Engl J Med. 2004 Nov 25;351(22):2276-85. doi: 10.1056/NEJMoa041469.

  PMID: 15564544 BACKGROUND

MeSH Terms

Conditions

Multiple Myeloma; Lymphoma, Non-Hodgkin; Hematologic Neoplasms; Leukemia; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Thalassemia; Lysosomal Storage Diseases

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Lymphoma; Lymphatic Diseases; Neoplasms by Site; Leukemia, Myeloid; Myeloproliferative Disorders; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Ronald E Gress, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 22, 2011
• First Posted: November 24, 2011
• Study Start: October 27, 2011
• Primary Completion: November 26, 2012
• Study Completion: November 26, 2012
• Last Updated: December 21, 2018

Record last verified: 2012-11-26