Using Teriflunomide to treat HTLV-1 associated myelopathy

Phase I/II Study of Teriflunomide in HTLV-1 Associated Myelopathy/Tropical Spastic Paraparesis

PHASE1; PHASE2 · National Institutes of Health Clinical Center (CC) · NCT04799288

This study is testing if the medication teriflunomide can help people with HTLV-1 associated myelopathy feel better over nine months.

Quick facts

PhasePHASE1; PHASE2
Study typeInterventional
Enrollment24 (estimated)
Ages18 Years to 120 Years
SexAll
SponsorNational Institutes of Health Clinical Center (CC) (nih)
Drugs / interventionsprednisone
Locations1 site (Bethesda, Maryland)
Trial IDNCT04799288 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effects of teriflunomide, a medication typically used for multiple sclerosis, on patients with HTLV-1 associated myelopathy/tropical spastic paraparesis (HAM/TSP). The study involves a single center, open-label design where participants will take the drug daily for nine months, followed by a three-month follow-up. Researchers will assess immune responses, safety, and tolerability through various clinical measures and laboratory tests. The primary focus is on immune activation markers and their correlation with disease burden in HAM/TSP patients.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with HAM/TSP.

Not a fit: Patients without a confirmed diagnosis of HAM/TSP or those unable to comply with the study protocol may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from HAM/TSP, potentially improving their quality of life.

How similar studies have performed: While this approach is novel for HAM/TSP, teriflunomide has shown efficacy in treating other autoimmune conditions, suggesting potential for success in this context.

Eligibility criteria

Show full inclusion / exclusion criteria
* 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
  * Study participants who have been prescribed \<= 10 mg prednisone (PO) per day will be required to discontinue this medication for a period of at least 3 months (90 days) prior to enrolling in 21-N-0016. Additionally, participants who have been prescribed burst therapy with an oral steroid as an outpatient over the course of \<= 10 days (e.g., Medrol Dosepak \[methylprednisolone\]), can enroll after discontinuing burst dose oral steroids for at least 3 months.
* Pregnant or lactating women.
* Treatment with other investigational drugs within 6 months before enrollment
* Known hypersensitivity to teriflunomide or leflunomide
* Concomitant treatment with leflunomide

Where this trial is running

Bethesda, Maryland

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: HAM/TSP, Aubagio, Single center, Open Label

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.