Monoclonal Antibody Conditioning for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases

Stopped early · Phase 1/Phase 2

Conditions studied: Inherited Metabolic Storage Diseases

In brief

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.'

Key facts

Study ID: NCT00056979
Run by: Baylor College of Medicine
People needed: 2
Starts: 2002-06-01
Expected to finish: 2003-06-01
Last updated by the study team: 2015-09-23

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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.

Where it is running

Texas Children's Hospital — Houston, Texas, United States
The Methodist Hospital — Houston, Texas, United States

Full record on ClinicalTrials.gov

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