Teriflunomide for Tropical Spastic Paraparesis
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
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
Exclusion Criteria
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive teriflunomide 14 mg daily for 9 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Teriflunomide (Immunomodulatory Agent)
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Institute of Neurological Disorders and Stroke (NINDS)
Lead Sponsor
Jordan Gladman
National Institute of Neurological Disorders and Stroke (NINDS)
Chief Medical Officer
MD from Harvard Medical School
Walter J. Koroshetz
National Institute of Neurological Disorders and Stroke (NINDS)
Chief Executive Officer since 2007
MD from the University of Chicago