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

St. Marianna University School of Medicine

Status and phase

Unknown

Phase 3

Phase 2

Conditions

HTLV-I-Associated Myelopathy

Treatments

Drug: Tamibarotene

Study type

Interventional

Funder types

Other

Identifiers

NCT01343355

AM80H-01

Details and patient eligibility

About

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.

Enrollment

15 estimated patients

Sex

All

Ages

30 to 75 years old

Volunteers

No Healthy Volunteers

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.