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Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO (THALLO)

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ClinicalTrials.gov Identifier: NCT00578292
Recruitment Status : Terminated (Over the course of this study stem cell transplant was determined to be standard of care for this disease and as such the study is no longer relevant.)
First Posted : December 21, 2007
Last Update Posted : July 22, 2016
Texas Children's Hospital
Information provided by (Responsible Party):
Kathryn Leung, Baylor College of Medicine

Brief Summary:

Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells. Because of that, the patient has chronically low red blood cell numbers (anemia) and need regular blood transfusions to help the patient feel better and to help prevent damage to important organs such as the heart. The following treatments are currently available to patients: lifelong blood transfusions and drugs that help remove iron from the body, and long-term antibiotics to prevent infections. These treatments are difficult for patients to take, and do not stop the effects of the disease.

Currently, the only treatment that may cure thalassemia is bone marrow or blood stem cell transplantation. Special blood or bone marrow cells from a healthy person might allow the bone marrow to create healthy cells, which will replace the abnormal red blood cells of thalassemia. There is a lot of experience using special blood or bone marrow cells from a healthy brother or sister who is the same HLA (immune) type. For patients who do not have such a donor in the family, an unrelated volunteer donor can be used. It is important for the patient to realize that this kind of transplant can have more problems than a transplant from a brother or sister.

Because we do not know the long-term effects of this treatment and because this type of transplant has not been used often for people with thalassemia, this is a research study. We hope, but cannot promise, that the transplanted marrow/stem cells will produce healthy cells and the patient will no longer have severe thalassemia.

Condition or disease Intervention/treatment
Thalassemia Drug: Busulfan Drug: Fludarabine Drug: Campath 1H Drug: Cyclophosphamide Drug: MESNA

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Study of Allogeneic Stem Cell Transplantation From Unrelated Donors for Patients With Severe Homozygous Beta 0/+ Thalassemia or Severe Variants of Beta 0/+ Thalassemia
Study Start Date : February 2004
Primary Completion Date : May 2016
Study Completion Date : May 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia
U.S. FDA Resources

Arm Intervention/treatment
Experimental: Bone Marrow or Stem Cell Infusion

Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H

Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0.

Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor.

Drug: Busulfan

4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg

Days -9 through -6

Other Name: Myleran
Drug: Fludarabine
30mg/m2 Day -5 through Day -2
Other Name: Fludara
Drug: Campath 1H
Per institutional guidelines Days -5 through -2
Other Name: Alemtuzumab
Drug: Cyclophosphamide
50 mg/kg Days -5 through -2
Other Name: Cytoxan
10 mg/kg x 5 Days -5 through -2
Other Name: Mesnex

Primary Outcome Measures :
  1. Evaluate engraftment. [ Time Frame: 30 days post-transplant ]
  2. To evaluate the occurrence of transient and stable mixed hematopoietic chimerism (HC) after unrelated donor SCT, and its effect on the recurrence of clinically measurable thalassemia. [ Time Frame: 2 years ]
  3. To measure hematopoietic and immune reconstitution, and assess the effects on infectious complications. [ Time Frame: 2 years ]
  4. Toxicities. [ Time Frame: +100 days post-transplant ]
  5. Loss of chimerism. [ Time Frame: 2 years post-transplant ]
  6. GVHD [ Time Frame: 2 years post-transplant ]
  7. Morbidity and mortality post-transplant. [ Time Frame: 2 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 64 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Patients with documented diagnosis of severe (transfusion-dependent) homozygous b0/+-thalassemia or severe variants of b0/+-thalassemia requiring chronic transfusion therapy and iron chelating agents, who fulfill the following conditions:

  1. Patient does not have an HLA genotype-identical donor available and has a 5/6 or 6/6 matched unrelated donor, or a 5/6 matched related donor available.
  2. Must be between 1 and 16 yrs of age (all Pesaro risk groups).
  3. Patients older than 17 yrs of age must be in Pesaro Risk Class 2 or lower (see Appendix B).
  4. Women of childbearing potential must have a negative pregnancy test.
  5. Documentation of compliance with iron chelation, absence or presence of hepatomegaly, and presence or absence of hepatic fibrosis prior to transplant (criteria for the Pesaro Risk Classification). This information will be obtained by history, physical exam and interpretation of liver biopsy results.
  6. Documentation of awareness of alternative treatment options.

Exclusion Criteria:

  1. Biopsy-proven chronic active hepatitis or fibrosis with portal bridging.
  2. Has previous history of malignancies.
  3. Creatinine clearance < 35 mL/min/1.73 M2.
  4. Severe cardiac dysfunction defined as shortening fraction < 25%.
  5. HIV infection.
  6. Inadequate intellectual capacity to give informed consent (in the case of minors, this criteria must be fulfilled by the legal guardian).
  7. Be pregnant, lactating or unwilling to use appropriate birth control.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00578292

United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
Texas Children's Hospital
Principal Investigator: Kathryn Suet Wa Leung, MD Baylor College of Medicine/Texas Children's Hospital

Responsible Party: Kathryn Leung, Assistant Professor, Center for Cell and Gene Therapy, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00578292     History of Changes
Other Study ID Numbers: H-14539-THALLO
First Posted: December 21, 2007    Key Record Dates
Last Update Posted: July 22, 2016
Last Verified: July 2016

Keywords provided by Kathryn Leung, Baylor College of Medicine:
homozygous b0/+-thalassemia
severe variants of b0/+-thalassemia
chronic transfusion therapy
iron chelating agents
transfusion-dependent homozygous b0/+-thalassemia or severe variants of b0/+-thalassemia

Additional relevant MeSH terms:
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Fludarabine phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antimetabolites, Antineoplastic