Inherited Metabolic Storage Diseases Clinical Trial
Official title:
Anti-CD45 (YTH-24 & YTH 54) and ANTI-CD52 (CAMPATH-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases
| Verified date | September 2015 |
| Source | Baylor College of Medicine |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | United States: Food and Drug Administration |
| Study type | Interventional |
This study treats patients with an inherited disease that prevents the body from making a
specific protein or enzyme needed for the body's metabolism. Lack of this enzyme causes
accumulation of harmful or toxic substances in the body, which leads to deterioration and
failure of organs such as the brain or the heart. This disease can be fatal.
Some patients with inherited metabolic storage disease may benefit from an allogeneic stem
cell transplant ('allogeneic' means that the stem cells come from another person). Stem
cells are created in the bone marrow. They mature into different types of blood cells that
are needed including red blood cells, white blood cells, and platelets. Stem cells, when
transplanted, can make a new blood system. Donor stem cells can make the protein or enzyme
patients with this disease cells cannot. The donor cells may prevent further accumulation of
toxic substances. It is hoped that the donor cells can prevent or stop the disease from
progressing.
This research study uses a new pre-treatment combination of two drugs, Anti-CD45 and
CAMPATH-1H. Anti-CD45 and CAMPATH-1H are antibodies against certain types of blood cells.
CAMPATH-1H is particularly important because it stays active in the body for a long time
after infusion, which means it may work longer at preventing GVHD symptoms. In addition to
antibodies, patients will receive Fludarabine, which is a chemotherapy drug. Fludarabine
kills bone marrow cells and is given to reduce the bone marrow cells so that donor stem
cells may 'take.'
| Status | Terminated |
| Enrollment | 2 |
| Est. completion date | June 2003 |
| Est. primary completion date | June 2003 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | N/A and older |
| Eligibility |
Inclusion criteria: - Patients with inherited metabolic storage diseases of all ages are eligible. - Diagnosis of inherited metabolic storage disease confirmed by standard biochemical and genetic studies in consultation with the Department of Genetics at the Baylor College of Medicine - Inherited metabolic storage diseases which may be stabilized or improved by stem cell transplantation include: Hurler, Hunter, Maroteaux-Lamy, Sly, Wolman, Gaucher, Farber, Nieman-Pick, Mannosidosis, Aspartylglucosaminuria, Fucosidosis, Neuronal Ceroid-Lipofuscinosis, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, and Adrenoleukodystrophy - Availability of an HLA matched or mismatched (up to one haplotype) donor who is not an obligate carrier for the inherited condition or an unrelated HLA matched stem cell donor. Fully matched is defined as 6/6 match by high resolution DR based DNA typing. - Female patients of childbearing age must have a negative pregnancy test and be willing to use an effective means of birth control. Exclusion criteria: - Patients with a life expectancy (<6 weeks) limited by diseases other than inherited metabolic storage disease - Patients with advanced inherited metabolic storage disease, which has not been stabilized or improved by hematopoietic stem cell transplantation. - Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction <25%) - Patients with severe renal disease (Creatinine >2 x normal for age) - Patients with known allergy to rat serum products - Patients with a Karnofsky or Lansky score <50%. - Patients with a severe infection that on evaluation by the Principal Investigator precludes ablative chemotherapy or successful transplantation - Patients with severe personality disorder or mental illness or neuropsychological evaluation indicating too much damage for the transplant to be of benefit. - Patients with documented HIV positivity. - Patients with grade III-IV liver toxicity not related to metabolic storage disease. |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| United States | Texas Children's Hospital | Houston | Texas |
| United States | The Methodist Hospital | Houston | Texas |
| Lead Sponsor | Collaborator |
|---|---|
| Baylor College of Medicine | Texas Children's Hospital, The Methodist Hospital System |
United States,