~2 spots leftby Aug 2025

Teriflunomide for Tropical Spastic Paraparesis

Recruiting in Palo Alto (17 mi)
DS
Overseen byDaniel S Reich, M.D.
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)
Must not be taking: Immunosuppressives, Leflunomide
Disqualifiers: HIV, Hepatitis B, Liver dysfunction, others
No Placebo Group
Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that participants stop taking certain medications. If you are taking prednisone or other oral steroids, you must stop them at least 3 months before joining the trial. Other immunomodulatory or immunosuppressive therapies are also not allowed.

Is Teriflunomide generally safe for humans?

Teriflunomide, also known as Aubagio, is generally well tolerated in patients with multiple sclerosis, but it can cause increased liver enzyme levels and is not safe for pregnant women due to potential risks to the unborn baby. Long-term safety data is limited, but information from its parent drug, leflunomide, suggests it is safe for long-term use.12345

How does the drug Teriflunomide differ from other treatments for tropical spastic paraparesis?

Teriflunomide is unique because it is primarily used for multiple sclerosis and works by reducing the activity of immune cells, which may help in conditions like tropical spastic paraparesis where the immune system is involved. Unlike other treatments like prednisone or methylprednisolone, which are steroids, Teriflunomide is not a steroid and has a different mechanism of action.678910

Research Team

DS

Daniel S Reich, M.D.

Principal Investigator

National Institute of Neurological Disorders and Stroke (NINDS)

Eligibility Criteria

Adults over 18 with HAM/TSP, a rare disease linked to HTLV-1 virus causing lower limb weakness. Participants must be able to take oral meds, follow the trial design, and use reliable birth control if necessary. Excluded are those with severe immune or liver issues, other conditions that could affect results, or recent immunomodulatory drugs usage.

Inclusion Criteria

Enrolled in 98-N-0047
I can take pills and will follow the treatment plan.
Patient must be willing and able to comply with all aspects of trial design and follow-up
See 5 more

Exclusion Criteria

Pregnant or lactating women
I haven't taken immune-suppressing drugs recently, except for low-dose prednisone or topical steroids.
My liver tests are more than twice the normal limit.
See 7 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Treatment

Participants receive teriflunomide 14 mg daily for 9 months

9 months
Visits every 3 months (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months
Visits every 3 months (in-person)

Treatment Details

Interventions

  • Teriflunomide (Immunomodulatory Agent)
Trial OverviewThe study tests teriflunomide's effectiveness on HAM/TSP by monitoring effects and immune response over 9 months of daily tablet intake. It includes blood/urine tests, lymphapheresis (white cell removal), spinal taps for fluid analysis, and MRI scans of brain/spine.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: TeriflunomideExperimental Treatment1 Intervention
Teriflunomide 14 mg daily

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Institute of Neurological Disorders and Stroke (NINDS)

Lead Sponsor

Trials
1,403
Recruited
655,000+

Jordan Gladman

National Institute of Neurological Disorders and Stroke (NINDS)

Chief Medical Officer

MD from Harvard Medical School

Walter J. Koroshetz profile image

Walter J. Koroshetz

National Institute of Neurological Disorders and Stroke (NINDS)

Chief Executive Officer since 2007

MD from the University of Chicago

Findings from Research

Teriflunomide (Aubagio®) is an effective once-daily oral treatment for relapsing forms of multiple sclerosis (RMS), approved in multiple regions including Europe and North America.
The review discusses teriflunomide's safety and efficacy based on extensive clinical trials, while also exploring its therapeutic mechanism of action, which is still being researched through various studies.
Teriflunomide (Aubagio®) for the treatment of multiple sclerosis.Bar-Or, A.[2021]
In a real-world study involving 200 patients with relapsing multiple sclerosis, teriflunomide demonstrated stable quality of life over 24 months, with significant reductions in relapse activity compared to the pre-baseline period.
The study indicated high treatment adherence and acceptability of teriflunomide, as reflected in patient-reported outcomes, with no substantial changes in fatigue and a marginal increase in reported disability.
Real-world study of relapsing-remitting multiple sclerosis patients treated with Teriflunomide in Nordic countries: Quality-Of-Life, efficacy, safety and adherence outcomes.Hestvik, ALK., Frederiksen, JL., Nielsen, HH., et al.[2022]
Teriflunomide (Aubagio®) is an oral medication that works by targeting the mitochondrial enzyme dihydroorotate dehydrogenase, helping to modulate the immune response in patients with relapsing-remitting multiple sclerosis (MS).
On June 18, 2021, teriflunomide was approved for use in pediatric patients aged 10 years and older in the EU, marking a significant milestone in its development for treating MS in younger populations.
Teriflunomide: Pediatric First Approval.Paik, J.[2021]

References

Teriflunomide (Aubagio®) for the treatment of multiple sclerosis. [2021]
Real-world study of relapsing-remitting multiple sclerosis patients treated with Teriflunomide in Nordic countries: Quality-Of-Life, efficacy, safety and adherence outcomes. [2022]
Teriflunomide: Pediatric First Approval. [2021]
Real-life outcomes of teriflunomide treatment in patients with relapsing multiple sclerosis: TAURUS-MS observational study. [2022]
Teriflunomide: a review of its use in relapsing multiple sclerosis. [2021]
[Tropical spastic paraparesis/HTLV-I associated myelopathy. Report of 2 cases diagnosed in Cuiabá, Mato Grosso, Brazil]. [2019]
[HTLV-I-associated myelopathy/tropical spastic paraparesis: report of 2 cases diagnosed in Florianópolis, Santa Catarina, Brazil]. [2019]
Tropical spastic paraparesis/HTLV-I-associated myelopathy in Europe and in Africa: clinical and epidemiologic aspects. [2019]
Tropical spastic paraparesis treated with Combivir (lamivudine-zidovudine). [2013]
HTLV-1 and tropical spastic paraparesis. 1. Clinical features, pathology and epidemiology. [2019]