Clinical Trial Details
— Status: Terminated
Administrative data
| NCT number |
NCT00006054 |
| Other study ID # |
199/15104 |
| Secondary ID |
UMN-MT-1995-26UM |
| Status |
Terminated |
| Phase |
N/A
|
| First received |
July 5, 2000 |
| Last updated |
October 14, 2009 |
| Start date |
March 2000 |
| Est. completion date |
December 2002 |
Study information
| Verified date |
October 2003 |
| Source |
Office of Rare Diseases (ORD) |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
Unspecified |
| Study type |
Interventional
|
Clinical Trial Summary
OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation
for patients with primary immunodeficiencies.
II. Determine relevant outcomes of this treatment in these patients including quality of
survival, extent of morbidity and mortality from complications of the treatment (e.g., graft
versus host disease, regimen related toxicities, B- cell lymphoproliferative disease), and
completeness of functional immunoreconstitution.
Description:
PROTOCOL OUTLINE: Patients with severe combined immunodeficiency (SCID) using a matched
sibling donor receive allogeneic bone marrow or umbilical cord blood transplantation on day
0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on
days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50.
Patients with SCID using donors other than histocompatible siblings, Wiskott Aldrich
syndrome using a histocompatible sibling donor, Wiskott Aldrich syndrome and under 5 years
of age using donors other than histocompatible siblings, X-linked CD40 ligand deficiency
using a histocompatible sibling donor, X-linked CD40 ligand deficiency and under 5 years of
age using donors other than histocompatible siblings, other primary immunodeficiencies
without manifestations of hemophagocytosis using a histocompatible sibling donor, or other
primary immunodeficiencies without manifestations of hemophagocytosis and under 5 years of
age using donors other than histocompatible siblings receive busulfan IV over 2 hours every
6 hours on days -9 to -6, cyclophosphamide IV on days -5 to -2, and antithymocyte globulin
(ATG) twice daily on days -4 to -1. Allogeneic bone marrow or umbilical cord blood
transplantation takes place on day 0. Patients receive graft versus host disease (GVHD)
prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to
50.
Patients with hemophagocytic lymphohistiocytosis, Chediak Higashi syndrome, X-linked
lymphoproliferative syndrome, severe progressive Langerhans cell histiocytosis, or other
primary immunodeficiencies with complications of hemophagocytosis receive busulfan IV over 2
hours every 6 hours on days -9 to -6, cyclophosphamide IV over 2 hours on days -5 to -2,
etoposide IV over 22 hours on days -5 to -3, and ATG IV twice daily on days -2, -1, 1, and
2. Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0.
Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days
1, 3, 6, and 11 and cyclosporine IV on days -3 to 50.
Patients with Wiskott Aldrich syndrome or other primary immunodeficiencies without
manifestations of hemophagocytosis, who are over 5 years of age and using donors other than
histocompatible siblings, receive busulfan IV over 2 hours every 6 hours on days -6 and -5,
cyclophosphamide IV over 2 hours on days -4 and -3, total body irradiation on day -2, and
ATG IV over 2 hours twice daily on days -2, -1, 2, and 3. Allogeneic bone marrow or
umbilical cord blood transplantation takes place on days 0 and 1. Patients receive GVHD
prophylaxis with methylprednisolone IV every 12 hours on days 2-21, oral prednisone every 12
hours on days 22-100 and then tapered off over days 101 to 128, and cyclosporine IV over 2
hours every 8-12 hours on days -3 to 100.
All patients are followed as determined by their primary physician.
