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Chronic Myelogous Leukemia/Other LeukemiaPhase 2NCT01479582

Providing Access to Cord Blood Units for Transplants

A Phase 2 study testing Access to unlicensed cord blood units in people with Chronic Myelogous Leukemia/Other Leukemia and Acute Leukemias, run by National Cancer Institute (NCI).

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages Up to 99 years
What is being tested?
Access to unlicensed cord blood units
How many people
0 participants
Where
No sites listed yet
Study dates
27 Oct 2011to 26 Nov 2012
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 99 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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
Show all 11Show fewer
  • Primary immunodeficiency diseases
  • Bone marrow failure
  • Beta-thalassemia
  • Signed informed consent (and signed assent, if applicable)
  • Pediatric and adult patients of any age

Who cannot join

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

About this study

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

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.
Read more

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.
Read the detailed description

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.

Being tested
Access to unlicensed cord blood units
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
Medical subject headings
Multiple Myeloma, Lymphoma, Non-Hodgkin, Hematologic Neoplasms, Leukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Thalassemia, Lysosomal Storage Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Access to unlicensed cord blood unitsDrug

What is being measured

The primary outcome is the main question the study is designed to answer.

  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)

  1. Assess incidence of graft rejection.

  2. Assess incidence of transmission of infection.

  3. Assess incidence of serious infusion reaction.

  4. Determine 1 year survival after cord blood transplantation.

  5. Assess cumulative incidence of aGVHD vs cGVHD.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Nov 2011 (estimated)
  2. Study start27 Oct 2011
  3. Primary completion26 Nov 2012
  4. Study completion26 Nov 2012

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01479582
Sponsor's protocol ID
120027
Other IDs
12-C-0027
Status last verified
26 Nov 2012
First submitted
22 Nov 2011
Last updated
21 Dec 2018
Results posted
No

Publications

  • 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.

    PubMed 11407342

  • 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.

    PubMed 8657213

  • 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.

    PubMed 15564544

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Access to unlicensed cord blood units in people with Chronic Myelogous Leukemia/Other Leukemia and Acute Leukemias, run by National Cancer Institute (NCI).

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 21 Dec 2018. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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.... It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01479582, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Dec 2018.

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