Clinical Trial: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/-

Brief Summary: This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

Detailed Summary:
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: percentage of subjects who achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) [ Time Frame: neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • percentage of subjects who develop graft-versus-host disease [ Time Frame: by Day +100 post-transplant ]
  • number of subjects who die from transplant complications [ Time Frame: Day +100 days post-transplant ]
  • incidence of regimen-related toxicity (such as infection, acute renal failure, respiratory failure, cardiac failure, and veno-occlusive disease) [ Time Frame: Day +100 post-transplant ]
  • incidence of radiographic, physiologic, neuro-psychologic, and/or biochemical changes [ Time Frame: at 6 months, 1 year and yearly for a total of 5 years ]


Original Secondary Outcome:

  • percentage of subjects who develop graft-versus-host disease [ Time Frame: by Day +100 post-transplant ]
  • number of subjects who die from transplant complications [ Time Frame: Day +100 days post-transplant ]
  • incidence of regimen-related toxicity (such as infection, acute renal failure, respiratory failure, cardiac failure, and veno-occlusive disease) [ Time Frame: Day +100 post-transplant ]


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: June 20, 2014
Date Started: July 10, 2014
Date Completion: September 2019
Last Updated: March 22, 2017
Last Verified: March 2017