Overview

This trial is active, not recruiting.

Condition htlv-i-associated myelopathy
Treatment tamibarotene
Phase phase 2/phase 3
Sponsor St. Marianna University School of Medicine
Start date January 2011
End date March 2012
Trial size 15 participants
Trial identifier NCT01343355, AM80H-01

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment

Primary Outcomes

Measure
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

Secondary Outcomes

Measure
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

Eligibility Criteria

Male or female participants from 30 years up to 75 years old.

Inclusion Criteria: - Patients who have been diagnosed as HAM according to the WHO criteria - Patients who are positive for HTLV-I antibody in the spinal fluid - Patients, if female, who are not pregnant or breastfeeding, either agreed to take contraceptive measures during and two years after the treatment, or sterile - Patients, if male, who agreed to take contraceptive measures during and six months after the treatment - Patients who have been informed and understood the contents of the study and consented to participate in the signed form. Exclusion Criteria: - Patients who has a rapid progress in the symptoms defined as an increase of two or more in Osame's Motor Disability Score for HAM patients in the past one year. - Patients of hyperlipidemia (serum triglyceride higher than 400 mg/dL) - Patients who were administered new or increased dose of corticosteroid in the past 8 weeks before the intervention - Patients who received steroid pulse therapy in the past 8 weeks before the intervention - Patients who were administered new or increased dose of immunosuppressant in the past 8 weeks before the intervention - Patients with a history of serious drug allergy - Patients with significant complication such as malignancy, severe heart failure, and other serious diseases. - Patients who were in the past administered etretinate.

Additional Information

Official title Open-Label, Exploratory Study of the Efficacy and Safety of Tamibarotene(AM80H) for HTLV-1 Associated Myelopathy/ Tropical Spastic Paraparesis (HAM/TSP)
Principal investigator Yoshihisa Yamano, MD
Trial information was received from ClinicalTrials.gov and was last updated in July 2011.
Information provided to ClinicalTrials.gov by St. Marianna University School of Medicine.