Clinical Trial: Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Open-Label, Exploratory Study of the Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)

Brief Summary: An open-label, non-randomised, uncontrolled, proof-of-concept study of patients with HTLV-I-associated myelopathy/Tropical Spastic Paraparesis (HAM/TSP). Participants will receive oral administration of tamibarotene in the amount of 2 mg daily over a period of 12 weeks, then 4mg daily for another 12 weeks. The patients will be followed up for further 8 weeks. Efficacy will be monitored by measuring clinical scores including motor and urination function, HTLV-1 proviral load, immunological parameters, and markers in the spinal fluid. Safety will be evaluated at the same time.

Detailed Summary:
Sponsor: St. Marianna University School of Medicine

Current Primary Outcome:

  • Change in Soluble IL-2 Receptor level in peripheral blood [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
  • Change in HTLV-I viral load in peripheral blood [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
  • Change in T cell population in peripheral blood [ Time Frame: 0,12, 24, 28 and 32 weeks ]
  • Change in cerebrospinal fluid examination [ Time Frame: baseline and after the treatment defined as from 24 to 32 weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in Osame's Motor Disability Score for HAM patients [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
  • Change in The Expanded Disability Status Scale (EDSS) [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
  • Change in timed 10m walk [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
  • Change in Manual Muscle Testing and vibratory perception of the lower limbs [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
  • Change in Modified Ashworth Scale [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]
  • Change in Urination function and defecation score [ Time Frame: 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks ]


Original Secondary Outcome: Same as current

Information By: St. Marianna University School of Medicine

Dates:
Date Received: April 25, 2011
Date Started: January 2011
Date Completion: March 2012
Last Updated: July 21, 2011
Last Verified: July 2011