Stem Cell Transplant for Autoimmune Neurological Diseases

Palo Alto (17 mi)

Overseen byLeona Holmberg

Age: Any Age

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Fred Hutchinson Cancer Research Center

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial tests a combination of strong chemotherapy and an immune-suppressing medicine followed by a stem cell transplant. It targets patients with autoimmune neurological diseases that haven't improved with other treatments. The treatment aims to weaken the immune system to stop it from attacking the nervous system and then helps the body recover by making new blood cells. High-dose chemotherapy followed by a stem cell transplant has been shown to improve various autoimmune diseases.

Eligibility Criteria

This trial is for patients under 70 with severe neurological autoimmune disorders like Multiple Sclerosis, Myasthenia Gravis, and others who haven't responded to at least two standard treatments. Donors must be a syngeneic sibling (like an identical twin) willing to undergo procedures for stem cell collection.

Inclusion Criteria

I have an autoimmune disorder affecting my nervous system.

I have been diagnosed with a severe neurological autoimmune disorder.

I have tried at least 2 standard treatments for my condition without success.

I have an autoimmune disorder affecting my nervous system.

I have inflammation of blood vessels in my brain.

I have been diagnosed with Rasmussen's encephalitis.

I have been diagnosed with GALOP.

I have been diagnosed with Chronic Inflammatory Demyelinating Polyneuropathy.

I have been diagnosed with a severe neurological autoimmune disorder.

I am 70 years old or younger.

My scans show active disease or my condition is getting worse.

I have tried at least 2 standard treatments for my condition without success.

I am the identical twin of a patient in this study, proven by blood and genetic tests.

I am willing to undergo several apheresis procedures or bone marrow harvests if I'm under 12.

Exclusion Criteria

I am 71 years old or older.

I have lung, heart, liver, or kidney problems that could affect my treatment.

I currently have an infection that isn't under control.

I have been diagnosed with myelodysplasia.

I have an active cancer other than skin cancer.

Treatment Details

The study tests high-dose chemotherapy (carmustine, etoposide, cytarabine, melphalan) plus antithymocyte globulin followed by autologous stem cell transplant in treating autoimmune neurologic diseases that are resistant to other therapies.

1Treatment groups

Experimental Treatment

Group I: Treatment (immunosuppressive therapy followed by transplant)Experimental Treatment10 Interventions

Patients receive carmustine IV on day -6, etoposide IV and cytarabine IV BID on days -5 to -2, melphalan IV on day -1 and antithymocyte globulin IV on days -2 and -1. Patients then undergo autologous or syngeneic stem cell transplant on day 0. Patients also receive prednisone PO QD on days 7-21, followed by 2 week taper.

Anti-Thymocyte Globulin is already approved in European Union, United States for the following indications:

🇪🇺 Approved in European Union as Thymoglobulin for: