Overview

Background

HTLV-1 associated myelopathy/tropical spastic paraparesis (HAM/TSP) is a rare, progressive disease. It occurs in some people infected with the HTLV-1 virus. It leads to weakness in the lower limbs and other serious problems. It has no treatment. Teriflunomide is a drug used to treat multiple sclerosis. It reduces immune cells that make the disease worse. Researchers want to learn if this drug can help people with HAM/TSP.

Objective

To learn the effects, immune response, safety, and tolerability of teriflunomide in people with HAM/TSP.

Eligibility

Adults ages 18 and older with HAM/TSP.

Design

Participants will be screened under protocol 98-N-0047.

Participants will have a medical history. They will have physical and neurological exams. They will have blood and urine tests.

Participants will take 1 tablet of the study drug once a day for 9 months. They will keep a drug diary.

Participants will have lymphapheresis. For this, blood is drawn from a needle in one arm. A machine divides the blood into red cells, plasma, and white cells. The white cells are removed. The plasma and red cells are returned to the participant through a needle in the other arm.

Participants will have lumbar punctures ( spinal taps ). For this, a thin needle is inserted into the spinal canal in the lower back. Spinal fluid is removed.

Participants will have magnetic resonance imaging (MRI) of the brain and spine. The MRI scanner is a metal cylinder surrounded by a strong magnetic field. During the MRI, participants will lie on a table that can slide in and out of the scanner.

Participation will last for 15 months.

Eligibility

• INCLUSION CRITERIA:

        In order to be eligible to participate in this study, an individual must meet all of the
        following criteria:
          -  18 years or older
          -  Diagnosis of HAM/TSP as defined by WHO criteria, including a positive HTLV-1 EIA and
             confirmatory Western Blot.
          -  Enrolled in 98-N-0047
          -  Patient must be willing and able to comply with all the aspects of trial design and
             follow-up.
          -  Negative QuantiFERON-TB gold, or completion of latent tuberculosis infection
             treatment, per CDC and National TB Controllers Association recommendations in the
             event of a positive test result
             --In the event of an indeterminant result, the test will be repeated. Should two
             consecutive tests yield indeterminant results, a chest x-ray will be performed to rule
             out radiographic evidence of a latent TB infection. Negative imaging will enable the
             subject to qualify for participation in the study.
          -  Ability to take oral medication and be willing to adhere to the protocol regimen
          -  Patients must be able to provide informed consent
          -  If able to become pregnant or to father a child, patient must agree to commit to the
             use of a reliable/accepted method of birth control (i.e. hormonal contraception (birth
             control pills, injected hormones, vaginal ring), intrauterine device, barrier methods
             with spermicide (diaphragm with spermicide, condom with spermicide) or surgical
             sterilization (hysterectomy, tubal ligation, or vasectomy) for the duration of the
             treatment arm of the study and for two years following cessation of treatment with
             teriflunomide.
        EXCLUSION CRITERIA:
        An individual who meets any of the following criteria will be excluded from participation
        in this study:
          -  Alternative diagnoses that can explain neurological disability
          -  History or current evidence of any condition, therapy, or laboratory abnormality that
             might confound the results of the trial or interfere with participation for the full
             duration of the trial; or not in the best interest of the subject to participate, in
             the opinion of the treating investigator
          -  Severe immunodeficiency, bone marrow disease, or severe, uncontrolled infections.
          -  Liver dysfunction, as indicated by baseline aspartate aminotransferase (AST) or
             alanine or alanine aminotransferase (ALT) greater than 2 times the upper limit of
             normal
          -  Positive serological evidence of HIV, HTLV-II, Hepatitis B or C
          -  Treatment with immunomodulatory/immunosuppressive therapy is exclusionary except in
             the cases below:
               -  current use of topical steroids
               -  <= 10 mg of prednisone usage within three months prior to administration of study
                  drug.
          -  Pregnant or lactating women.
          -  Treatment with other investigational drugs within 6 months before enrollment
          -  Known hypersensitivity to teriflunomide or leflunomide
          -  Concomitant treatment with leflunomide