HTLV-I-Associated Myelopathy
Conditions
Keywords
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.
Interventions
DRUGTamibarotene
Oral administration of tamibarotene 2 mg daily over a period of 12 weeks, then 4mg daily for another 12 weeks.
Sponsors
St. Marianna University School of Medicine
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
30 Years to 75 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Soluble IL-2 Receptor level in peripheral blood | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
| Change in HTLV-I viral load in peripheral blood | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
| Change in T cell population in peripheral blood | 0,12, 24, 28 and 32 weeks |
| Change in cerebrospinal fluid examination | baseline and after the treatment defined as from 24 to 32 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Change in Modified Ashworth Scale | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
| Change in Osame's Motor Disability Score for HAM patients | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
| Change in Urination function and defecation score | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
| Change in The Expanded Disability Status Scale (EDSS) | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
| Change in timed 10m walk | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
| Change in Manual Muscle Testing and vibratory perception of the lower limbs | 0, 4, 8, 12, 16, 20, 24, 28 and 32 weeks |
Countries
Japan
Outcome results
None listed