Clinical Trial: Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemi

Brief Summary:

Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Thalassemia is when the body does not make enough hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The patient may or may not need to get regular blood transfusions (getting more blood) to improve their quality of life (feel better) and prevent organ damage (problems with the brain, heart, lung, kidney, and gonad, for example.). The transfusions can also cause problems, including iron overload (too much iron in the blood), which can be fatal (patients can die) without regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or SCD die sooner. There is no proven cure.

We would like to treat patients using bone marrow transplantation, a treatment that has been used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from a brother or sister who does not have sickle cell disease or severe thalassemia. If the treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been used to treat patients with sickle cell disease or thalassemia. It has worked in most cases. We hope, but cannot promise, that the transplanted marrow will make healthy cells, and patients will not have sickle cell disease or severe thalassemia anymore.

We do not know what effect this treatment will have on the damage that has already been done by the disease. Finding that out is the main reason for this study. Currently, very little has been reported about organ function after bone marrow transplants in patients with

Detailed Summary:

Prior to any bone marrow transplant, we will need patients to:

  • Answer questions about their medical history;
  • Undergo a physical examination;
  • Have tests done to see how well the lungs are working;
  • Have a chest x-ray;
  • Have an EKG to look at the heart;
  • Have an MRI & MRA (Magnetic Resonance Angiography, which looks at the blood vessels and the blood flowing through them). These 2 tests will look to see if the patient has had any strokes;
  • Have a PET scan to look at the head and body;
  • Have a liver biopsy to determine if the liver has been damaged (which can happen from iron overload that happens after many transfusions). Too much liver damage would mean a transplant cannot be done.

For the liver biopsy, the skin is numbed with medicine, and a special needle goes into the liver. The needle removes a very small piece of the liver (tissue). The tissue is taken and examined.

Also, about 30 cc (2 tablespoons) of blood will be drawn to test the blood for viruses, including HIV (the virus that causes AIDS). If the HIV test is positive, a transplant will not be done because it would be too dangerous for the patient.

At least 2 weeks before the bone marrow infusion, the patient will be immunized with Prevnar 7. Prevnar 7 is a vaccine that is used in children to protect against some types of bacteria called Streptococcus pneumoniae, which can cause the lung infection called pneumonia. People with Sickle Cell anemia are at a higher risk of dying from an infec
Sponsor: Baylor College of Medicine

Current Primary Outcome:

  • Evaluate recovery of organ function in patients with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors and if they can be improved or reversed. [ Time Frame: 12 months ]
  • Evaluate the use of PET scan examination in assessing metabolic function of organs in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+ after undergoing allogeneic SCT/BMT from HLA genotype identical donors. [ Time Frame: 12 months ]
  • Evaluate response to immunization after BMT in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+. [ Time Frame: 12 months ]


Original Primary Outcome:

  • Evaluate recovery of organ function in patients with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors and if they can be improved or reversed. [ Time Frame: 12 months ]
  • Evaulate the use of PET scan examination in assessing metabolic function of organs in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+ after undergoing allogeneic SCT/BMT from HLA genotype identical donors. [ Time Frame: 12 months ]
  • Evaluate response to immunization after BMT in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+. [ Time Frame: 12 months ]


Current Secondary Outcome:

Original Secondary Outcome:

Information By: Baylor College of Medicine

Dates:
Date Received: December 19, 2007
Date Started: July 2005
Date Completion:
Last Updated: April 22, 2014
Last Verified: April 2014