Protocol Number: 04-I-0289

Title:

Secondary Transplantation Using Moderate Dose Busulfan as Conditioning for a Patient with Partial Reconstitution Post Initial Allogeneic Transplantation

Number:

04-I-0289

Summary:

This protocol is designed for a single specific patient. It uses busulfan as a conditioning agent in a second stem cell transplant procedure for a patient with chronic granulomatous disease (CGD), a disorder in which a certain type of white cells, called myeloid cells, do not function properly. This causes increased risk of serious bacterial and fungal infections that can lead to organ dysfunction, such as kidney disease, as well as formation of granulomas-non-cancerous masses that can cause obstructions in the esophagus, stomach, and intestines, and block urine flow from the kidneys and bladder.). The child in this study has previously undergone a stem cell transplant to treat CGD, and, as a result, he is now producing normal lymphocytes (another type of white cell). However, the myeloid cells from the donor did not engraft successfully, and the patient is still producing his own defective myeloid cells. In this study, the child will undergo a second stem cell transplant in combination with busulfan, a drug that targets myeloid cells, killing them to make way for healthy, donated myeloid cells.

Treatment includes the following procedures:

- Medical evaluation to confirm that the patient is healthy enough to undergo the transplantation

- Treatment with busulfan, injected through the patient's central venous line

- Stem cell transplantation through the central venous line

- Blood tests on days 25, 56, and 91 after the transplant to assess how many cells are of donor origin

- Bone marrow aspiration on day 100, and then at 12, 24, and 36 months to assess how many cells are of donor origin

- Pulmonary function (breathing) test at 12 and 24 months

- Physical examination and blood tests, weekly or twice weekly for the first 2 to 3 months and at 4, 6, 12, 18, 24, 36, 48, and 60 months after transplant

- Treatment for graft-versus-host disease (GVHD), if this complication develops. GVHD is the attack of lymphocytes from the donor against the patient's own cells. This is good if it is against abnormal cells, but bad if serious damage occurs to the patient's vital organs. GVHD is treated with steroids and cyclosporine, and possibly other drugs if needed.

Sponsoring Institute:

National Institute of Allergy and Infectious Diseases (NIAID)

Recruitment Detail

Type: No longer recruiting/follow-up only

Gender: Male & Female

Referral Letter Required: Yes

Population Exclusion(s): None

Eligibility Criteria: This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Special Instructions:

Currently Not Provided

Keyword(s):

Chronic Granulomatous Disease

Nonmyeloablative

CD34 Positive Cells

Donor Engraftment

Myeloid Chimerism

Recruitment Keyword(s):

None

Condition(s):

Granulomatous Disease

Chronic

Investigational Drug(s):

None

Investigational Device(s):

None

Interventions:

Drug: Busulfan

Supporting Site:

National Institute of Allergy and Infectious Diseases

Contact(s):

This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Citation(s):

Winkelstein JA, et al. Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine (Baltimore). 2000 May;79(3):155-69.

Johnston RB Jr. Clinical aspects of chronic granulomatous disease. Curr Opin Hematol. 2001 Jan;8(1):17-22. Review.

Roesler J, et al. Third-generation, self-inactivating gp91(phox) lentivector corrects the oxidase defect in NOD/SCID mouse-repopulating peripheral blood-mobilized CD34+ cells from patients with X-linked chronic granulomatous disease. Blood. 2002 Dec 15;100(13):4381-90. Epub 2002 Aug 01.

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department Clinical Research Informatics, CC.

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