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Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism Clinical Trials Information presented on Clinical Trials Search isn't designed to be a substitute for certified healthcare advice, travels to or professional assistance using a genuine medical doctor. We are not physicians. Always confer with your dr. about Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism Clinical research trials and Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism medical trials happen in hundreds of places across the United States. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials usually measure the effectualness of new drugs. The intention of the studies / undertakings is to solve certain human healthcare questions. Clinical trials are a popular manner for mDs, government agencies, and private sector companies to locate treatments for all forms of circumstances, such as Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism. Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism Clinical Trials and other clinical trials allow for volunteers to undergo medical treatment choices before they are available to the general public. Some times the human subjects get treatment for free of charge, and sometimes they are paid for their time. Occasionally there is a cost for a Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism clinical trial. Participants frequently get the best healthcare available for their Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism condition. Risks are a reality, nonetheless, and can include extra or frequent physician trips, medical risks (possibly life-jeopardising), and/or the treatment being ineffective. Trials are federally governed with exacting guidelines to protect clinical trials subjects.
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Home > "P" Clinical Trials Conditions > Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism  Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism
Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism
For Condition: Graft Versus Host Disease,Lysosomal Storage Diseases,Peroxisomal Disorders
Status: Recruiting
Sponsor(s): Fairview University Medical Center ,
Synopsis: OBJECTIVES: I. Determine the survival at 100 days, and then at 1 and 3 years of children with lysosomal and peroxisomal inborn errors of metabolism (storage diseases) after undergoing bone marrow or umbilical cord blood transplantation. II. Determine the change in neuropsychometric function in these patients at 100 days, and then at 1, 2, and 3 years after undergoing this treatment regimen. IV. Estimate the toxicity of this treatment regimen in these patients. V. Estimate the rate of hematological donor cell engraftment with this treatment regimen in these patients. VI. Estimate the incidence of graft versus host disease in these patients after undergoing this treatment regimen.
Details: PROTOCOL OUTLINE: Patients are stratified according to donor type (related vs unrelated) and disease status (mucopolysaccharidosis or leukodystrophy with IQ at least 80 vs leukodystrophy with IQ less than 80). Patients receive cyclophosphamide IV over 2 hours on days -6 and -5; total body irradiation on days -3 to 0; methylprednisolone IV every 12 hours on days -3 to -1 and days 5 to 56; anti-thymocyte globulin IV over 4-6 hours every 12 hours on days -3 to -1; allogeneic T-cell depleted bone marrow or umbilical cord blood transplantation on day 0; cyclosporine IV or orally on days -3 to 180, and then tapering in the absence of graft versus host disease; and filgrastim (G-CSF) IV daily starting day 1 and continuing until blood counts recover. Patients are followed at days 100 and 180, and then at 1, 2, and 3 years.
Eligibility:
Study Type: Interventional, Treatment
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- - Patients with either of the following inborn errors of metabolism (and developmental quotients within two standard deviations of normal): Mucopolysaccharidosis I (MPS I) (Hurler disease) Mucopolysaccharidosis VI (MPS VI) (Maroteaux-Lamy syndrome) OR - Patients with any of the following inborn errors of metabolism (and a performance IQ greater than 45): Adrenoleukodystrophy (co-A ligase deficiency) Metachromatic leukodystrophy (arylsulfatase A deficiency) Presymptomatic late infantile, juvenile, or adult form No symptomatic late infantile form Globoid cell leukodystrophy (Krabbe's disease) (galactocerebrosidase deficiency) No symptomatic infantile form Acid lipase deficiency (Wolman disease) No evidence of hepatic cirrhosis Alpha mannosidase deficiency (mannosidosis) Fucosidase deficiency (fucosidosis) Glucocerebrosidase deficiency (Gaucher's Disease, type III) I cell disease Niemann-Pick disease - Must have an HLA identical, haplotype mismatched (at 1-3 antigens) related bone marrow or umbilical cord blood donor OR HLA 1 antigen mismatched unrelated bone marrow or umbilical cord blood donor --Patient Characteristics-- - Hepatic: Bilirubin and AST less than 2 times normal - Renal: Creatinine less than 2 times normal OR Creatinine clearance at least 60 mL/min - Cardiac: Ejection fraction greater than 30% - Other: Negative pregnancy test
Total Enrollment: 70
Location and Contact Information:
Overall Study Official:
CharlesPeters, Study Chair, Fairview University Medical Center
Fairview University Medical Center *Recruiting*
Minneapolis, Minnesota, 55455
United States
Recruiting Charles Peters 612-624-5407
Additional Information:
Study ID Numbers: 199/15102; UMN-MT-1995-01,UMN-MT-9501
Study Start Date: March 2000
Record last reviewed: October 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00005894
Other Lysosomal Storage Diseases Studies:
1. Study of Oral Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis
2. Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism
3. Study of Protein Translocation in Patients With Beta-Oxidation Disorders
Related Studies:
Other Lysosomal Storage Diseases Clinical Trials
Other Minnesota Clinical Trials
Other Minneapolis Clinical Trials
Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism
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