Donor Stem Cell Transplantation for Congenital Immunodeficiencies
Status:
Completed
Trial end date:
2019-11-01
Target enrollment:
Participant gender:
Summary
This study uses transplantation to treat patients with problems in their immune system. The
immune system cells come from the bone marrow where they grow from special cells called stem
cells. Giving patients stem cells from someone else may help to cure many patients with
certain immune diseases. This is called 'bone marrow transplantation'. This procedure can
have side effects that are life-threatening. To try to make transplantation safer we are
using lower doses of the medications used in preparing the patient for the transplant.
'Conditioning' treatments are given to patients to create space in their bone marrow. This
lets the cells of the donor go into the bone marrow and produce normal immune cells. This
study will use lower doses of a drug called busulfan and lower doses of radiation than what
are currently being used in other kinds of bone marrow transplantation for other diseases.
Another problem that can occur with bone marrow transplantation is 'graft-versus-host
disease'. This happens when the cells of the donor attacks different parts of the patient s
body. This study will use a medicine called sirolimus instead of the usual medicine,
cyclosporine, to prevent graft-versus-host disease.
To go onto this study, you must have:
1. A severe immune deficiency, such as chronic granulomatous disease or leukocyte adhesion
deficiency.
2. Have problems from the disease that call for stem cell transplantation.
3. You must also be between the ages of 2 and 40 years.
Two groups of patients are included in this study:
1. Patients who have a brother or sister that have stem cells that match the patient. This
is known as an allogeneic matched sibling transplant.
2. Patients who do not have a matched sibling donor but have a donor that matches in the
National Marrow Donor Program. This is know as matched unrelated donor transplantation.
Patients will have the following procedures:
- To create space in the bone marrow, patients are given two drugs, Campath-1H and
busulfan. To prevent the body from getting rid of the donated cells, patients are given
sirolimus. On the day before the BMT, patients in the matched unrelated donor group also
receive a low-dose of whole-body radiation. This will further improve the chances that
the patients body will accept the donor cells.
- Patients will get the donor stem cells through an intravenous (IV) line that goes into a
vein in their body. The cells make their way to the bone marrow space and slowly refill
the marrow over the next several weeks. Patients will usually stay in the hospital for
30 days after the transplant.
- For the first 3 months after the transplant, patients are watched closely. The patients
will have frequent visits to the clinic. During these visits the patient will have a
physical examination and blood tests. The doctor and nurse will also check any symptoms
the patient may have. At day 100 after the transplant a sample of bone marrow is taken.
- Patients will continue to be followed periodically for at least 5 years after the
transplant.
Phase:
Early Phase 1
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)