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Immunosuppressant

Stem Cell Transplant for Autoimmune Neurological Diseases

Phase 2
Waitlist Available
Led By Leona Holmberg
Research Sponsored by Fred Hutchinson Cancer Research Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with an autoimmune disorder of the central or peripheral nervous system will be eligible; this will include: Primary Central Nervous System (CNS) vasculitis, Rasmussen's encephalitis, Autoimmune peripheral neuropathy (anti-Hu [Anna-1], anti-GM1 [GD1b], anti-MAG, anti-ganglioside, anti-sulfatide), Autoimmune cerebellar degeneration, Gait Ataxia with Late age Onset Polyneuropathy (GALOP), Stiff Person Syndrome, Chronic Inflammatory Demyelinating Polyneuropathy, Myasthenia Gravis, Lambert-Eaton myasthenic syndrome, Human T-cell lymphotropic virus (HTLV)-1-associated myelopathy (HAM) / tropical spastic paraparesis (TSP), Opsoclonus/myoclonus (anti-Ri), Neuromyelitis optica, Multiple sclerosis, Other central or peripheral nervous system autoimmune diseases as approved by study neurologists and the Fred Hutchinson Cancer Research Center (FHCRC) faculty at Patient Care Conference (PCC)
Patients must satisfy the criteria for a diagnosis of one of the severe neurological autoimmune disorders outlined
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Study Summary

This trial is testing the side effects and effectiveness of a combination of drugs given before a stem cell transplant to treat patients with autoimmune neurological disease.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Potential side effects include weakened immune system leading to increased infection risk; blood count fluctuations; organ inflammation from the drugs used; and complications from the stem cell transplant process.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have an autoimmune disorder affecting my nervous system.
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I have been diagnosed with a severe neurological autoimmune disorder.
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I have tried at least 2 standard treatments for my condition without success.
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I have an autoimmune disorder affecting my nervous system.
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I have inflammation of blood vessels in my brain.
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I have been diagnosed with Rasmussen's encephalitis.
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I have been diagnosed with GALOP.
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I have been diagnosed with Chronic Inflammatory Demyelinating Polyneuropathy.
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I have been diagnosed with a severe neurological autoimmune disorder.
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I have been diagnosed with a severe neurological autoimmune disorder.
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I am 70 years old or younger.
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My scans show active disease or my condition is getting worse.
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I have tried at least 2 standard treatments for my condition without success.
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I am the identical twin of a patient in this study, proven by blood and genetic tests.
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I am willing to undergo several apheresis procedures or bone marrow harvests if I'm under 12.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of grades 4-5 regimen-related toxicity
Secondary outcome measures
Disease responses
Engraftment kinetics
Number of subjects achieving greater than or equal to 4.0 x 10^6 CD34+ cells/kg, after up to two peripheral blood stem cell mobilizations
+2 more

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cytarabine
2016
Completed Phase 3
~3310
Prednisone
2014
Completed Phase 4
~2370
Anti-Thymocyte Globulin
2009
Completed Phase 4
~980
Autologous Hematopoietic Stem Cell Transplantation
2017
Completed Phase 3
~1730
Carmustine
1990
Completed Phase 3
~1790
Peripheral Blood Stem Cell Transplantation
1997
Completed Phase 3
~1330
Melphalan
2008
Completed Phase 3
~1500
Syngeneic Bone Marrow Transplantation
2008
Completed Phase 2
~40
Etoposide
2010
Completed Phase 3
~2440

Find a Location

Who is running the clinical trial?

Fred Hutchinson Cancer Research CenterLead Sponsor
443 Previous Clinical Trials
148,192 Total Patients Enrolled
1 Trials studying Multiple Sclerosis
35 Patients Enrolled for Multiple Sclerosis
Fred Hutchinson Cancer CenterLead Sponsor
556 Previous Clinical Trials
1,343,215 Total Patients Enrolled
1 Trials studying Multiple Sclerosis
35 Patients Enrolled for Multiple Sclerosis
Leona HolmbergPrincipal InvestigatorFred Hutch/University of Washington Cancer Consortium
11 Previous Clinical Trials
326 Total Patients Enrolled

Media Library

Anti-Thymocyte Globulin (Immunosuppressant) Clinical Trial Eligibility Overview. Trial Name: NCT00716066 — Phase 2
Anti-Thymocyte Globulin (Immunosuppressant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT00716066 — Phase 2
Multiple Sclerosis Research Study Groups: Treatment (immunosuppressive therapy followed by transplant)
Multiple Sclerosis Clinical Trial 2023: Anti-Thymocyte Globulin Highlights & Side Effects. Trial Name: NCT00716066 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this study still enrolling new test subjects?

"The trial, which was first posted on 6/1/2008 and last edited on 6/17/2022, is still recruiting patients according to the listing on clinicaltrials.gov."

Answered by AI

What have been some of the most successful treatments using Autologous Hematopoietic Stem Cell Transplantation?

"Autologous Hematopoietic Stem Cell Transplantation is an effective treatment for patients suffering from cancer of the merkel cells, ophthalmia, sympathetic tumors, and scalp structure."

Answered by AI

Does this treatment have a precedent?

"Currently, there are 910 active trials studying Autologous Hematopoietic Stem Cell Transplantation with 229 trials in Phase 3. While most of the studies for Autologous Hematopoietic Stem Cell Transplantation are located in New york, New York, there are 34749 locations operating clinical trials for Autologous Hematopoietic Stem Cell Transplantation."

Answered by AI

What is the stance of the FDA on Autologous Hematopoietic Stem Cell Transplantation?

"Autologous Hematopoietic Stem Cell Transplantation is classified as a Phase 2 trial, meaning that while there is safety data available, there is currently no evidence to support its efficacy. Our team has rated its safety at a 2."

Answered by AI

How many people are being signed up for this experiment?

"That is correct, the online information indicates that this research project is still recruiting individuals. The listing was created on 6/1/2008 and updated as recently as 6/17/2022. They need a total of 80 people at 3 facilities."

Answered by AI

Who else is applying?

What state do they live in?
Texas
Florida
Colorado
What site did they apply to?
Colorado Blood Cancer Institute
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
1
2

Why did patients apply to this trial?

I need to try something new that will help me. I have tried so many different drugs and other procedures. My doctor has diagnosed me with Multifocal acquired demylating sensory and motor syndrome or Mad Sam. He also says I have CIDP. I can barely walk with a walker and use an electric wheelchair for most of the time. I do have many other issues and I'm not sure if they are related to my diagnosis or not. I'm going on 13 years of this horrible disease, but I'm still hoping to find help. It was a gradual progression over the past 13 years and it is asymetrical. Thank you. Bill.
PatientReceived 2+ prior treatments

How responsive is this trial?

Typically responds via
Phone Call
Most responsive sites:
  1. Colorado Blood Cancer Institute: < 48 hours
Average response time
  • < 2 Days
Recent research and studies
clinicaltrials.govStudy
Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases
2008. "Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT00716066.
All > Chronic Inflammatory Demyelinating Polyneuropathy > Washington
~2 spots leftby Jan 2025
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