Providing Access to Cord Blood Units for Transplants
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
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.
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Detaillierte Beschreibung
Study Design:
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.
Primary Objective:
The primary aim of this study is to examine the incidence of neutrophil recovery of (Bullet)500/mm3
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 a 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 platelet engraftment of >20,000 mcL and >50,000 mcL
- Determine CBU-derived engraftment
Eligibility Criteria:
Inclusion Criteria
- Patients with FDA-specified indications (see Appendix B for further details):
- 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
Exclusion Criteria
- Patients who are receiving only licensed CBUs
- Cord blood transplant recipients at international transplant centers
Treatment Description:
Treatment, including pre-transplant conditioning and GVHD prophylaxis, will occur per each
transplant center s specifications.
Accrual Objective:
In this access and distribution protocol, U.S. patients undergoing transplant using unlicensed
CBUs will be enrolled and there is no accrual maximum.
Accrual Period:
The accrual period is open ended.
Studientyp
Phase
- Phase 2
Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Beschreibung
- INCLUSION CRITERIA:
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
EXCLUSION CRITERIA:
- Patients who are receiving only licensed CBUs
- Cord blood transplant recipients at international transplant centers
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: Nicht randomisiert
- Interventionsmodell: Einzelgruppenzuweisung
- Maskierung: Keine (Offenes Etikett)
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
|---|
|
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...
|
Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
|---|
|
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.
|
Mitarbeiter und Ermittler
Sponsor
Publikationen und hilfreiche Links
Allgemeine Veröffentlichungen
- 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.
- 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.
- 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.
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn
Primärer Abschluss (Tatsächlich)
Studienabschluss (Tatsächlich)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Schätzen)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
- Herz-Kreislauf-Erkrankungen
- Gefäßerkrankungen
- Erkrankungen des Immunsystems
- Neubildungen nach histologischem Typ
- Neubildungen
- Lymphoproliferative Erkrankungen
- Lymphatische Erkrankungen
- Immunproliferative Erkrankungen
- Hämatologische Erkrankungen
- Hämorrhagische Störungen
- Hämostasestörungen
- Paraproteinämien
- Bluteiweißstörungen
- Neubildungen, Plasmazelle
- Lymphom
- Multiples Myelom
- Leukämie
- Lymphom, Non-Hodgkin
Andere Studien-ID-Nummern
- 120027
- 12-C-0027
Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .