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AUTOIMMUNE DISEASES (DAIT SACCC)

Study: High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT MS) 

Sponsor: NIH-National Institute of Allergy and Infectious Diseases (NIAID)

Collaborator: Immune Tolerance Network (ITN)

Prime: Rho

Project Status: Completed

Project Start and End Dates: July 2006–November 2015

Disease: Multiple sclerosis (MS) is a progressive disease in which a person’s own immune system attacks the brain and spinal cord. Symptoms of MS can range from mild to severe, and may include numbness and tingling, a loss of vision, chronic fatigue, coordination problems, a decline in memory and thinking skills, or paralysis. MS has no cure and affects about 250,000 to 300,000 people in the United States and about 2.3 million people worldwide. 

Objective: The HALT MS study evaluated the safety, efficacy, and durability of disease stabilization in patients with relapsing-remitting MS who were given an experimental treatment. 

Treatment: For the experimental treatment, patients received high-dose immunosuppressive therapy (HDIT) with autologous hematopoietic cell transplantation (HCT)—that is, transplantation of their own blood-forming stem cells.  A comprehensive assessment of MS disease activity—including death, progression, clinical relapse, or new lesions documented on MRI—was used as a composite primary endpoint.

Results: Twenty-four participants were followed for 5 years after receiving the experimental treatment that aims to suppress active disease and prevent further disability by removing disease-causing cells and resetting the immune system. After 5 years, nearly 70% of the patients who received the treatment sustained full neurologic function, more than 90% did not experience disease progression, and almost 87% did not have any periods of relapse. Few serious early complications or unexpected adverse events were associated with this treatment.
Public Health Impact: HDIT/HCT without maintenance therapy effectively induced long-term sustained remissions of active relapsing-remitting MS at 5 years. These results are now paving the way for making stem cell transplant a mainstream treatment option for individuals with MS. 

For full details, please refer to the HALT MS study published 3-year results in JAMA Neurology and final 5-year results published in Neurology:

Study Highlights: 136 people from Rho spent a total of 19,098 hours working on this study.

The HALT MS study began years before electronic data capture (EDC) systems became available. When EDC systems became available years after study initiation, the team came up with a comprehensive plan for transitioning the data collection from paper forms to electronic format in EDC. This massive overhaul was executed with ease and drastically reduced the time burden on the study sites as well the coordinating center. This transition allowed the database to be locked in a more timely fashion and ultimately sped up the process of publishing the results of this study. 

Services:

  • Biostatistics and Statistical Programming
  • Data Management
  • Clinical Operations And Monitoring
  • Clinical Project Management
  • Product Safety and Pharmacovigilance Services
  • Protocol Design/Development
  • Regulatory and Medical Writing Services
  • Study Document Management
  • Study Website Content Development and Maintenance