Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

53 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-06-30

Study Completion Date

2030-01-31

Brief Summary

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This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a stem cell transplant works in treating patients with autoimmune neurologic disease that did not respond to previous therapy. In autoimmune neurological diseases, the patient's own immune system 'attacks' the nervous system which might include the brain/spinal cord and/or the peripheral nerves. Giving high-dose chemotherapy, including carmustine, etoposide, cytarabine, melphalan, and antithymocyte globulin, before a stem cell transplant weakens the immune system and may help stop the immune system from 'attacking' a patient's nervous system. When the patient's own (autologous) stem cells are infused into the patient they help the bone marrow make red blood cells, white blood cells, and platelets so the blood counts can improve.

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Detailed Description

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OUTLINE:

Patients receive carmustine intravenously (IV) on day -6, etoposide IV and cytarabine IV twice daily (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 orally (PO) once daily (QD) on days 7-21, followed by 2 week taper.

After completion of study treatment, patients are followed up at 3 months, 1 year, and then annually thereafter for up to 5 years.

Conditions

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Autoimmune Disease Neurologic Autoimmune Disease Autologous Transplant Autoimmune Multiple Sclerosis Transplant MS Stem Cell Transplant Multiple Sclerosis Stem Cell Transplant Stiff Person Syndrome HCT for Neurologic Autoimmune Disorders CIDP Transplant Myasthenia Gravis Transplant Autoimmune Nervous System Disorder Central Nervous System Vasculitis Cerebellar Degeneration Chronic Inflammatory Demyelinating Polyneuropathy Lambert Eaton Myasthenic Syndrome Myasthenia Gravis Neuromyelitis Optica Opsoclonus Myoclonus Syndrome Rasmussen Subacute Encephalitis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment (immunosuppressive therapy followed by transplant)

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.

Group Type EXPERIMENTAL

Anti-Thymocyte Globulin

Intervention Type BIOLOGICAL

Given IV

Autologous Hematopoietic Stem Cell Transplantation

Intervention Type PROCEDURE

Undergo autologous or syngeneic stem cell transplantation

Carmustine

Intervention Type DRUG

Given IV

Cytarabine

Intervention Type DRUG

Given IV

Etoposide

Intervention Type DRUG

Given IV

Laboratory Biomarker Analysis

Intervention Type OTHER

Correlative studies

Melphalan

Intervention Type DRUG

Given IV

Peripheral Blood Stem Cell Transplantation

Intervention Type PROCEDURE

Undergo autologous or syngeneic stem cell transplantation

Prednisone

Intervention Type DRUG

Given PO

Syngeneic Bone Marrow Transplantation

Intervention Type PROCEDURE

Undergo syngeneic bone marrow transplantation

Interventions

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Anti-Thymocyte Globulin

Given IV

Intervention Type BIOLOGICAL

Autologous Hematopoietic Stem Cell Transplantation

Undergo autologous or syngeneic stem cell transplantation

Intervention Type PROCEDURE

Carmustine

Given IV

Intervention Type DRUG

Cytarabine

Given IV

Intervention Type DRUG

Etoposide

Given IV

Intervention Type DRUG

Laboratory Biomarker Analysis

Correlative studies

Intervention Type OTHER

Melphalan

Given IV

Intervention Type DRUG

Peripheral Blood Stem Cell Transplantation

Undergo autologous or syngeneic stem cell transplantation

Intervention Type PROCEDURE

Prednisone

Given PO

Intervention Type DRUG

Syngeneic Bone Marrow Transplantation

Undergo syngeneic bone marrow transplantation

Intervention Type PROCEDURE

Other Intervention Names

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Antithymocyte Globulin Antithymocyte Serum ATG ATGAM ATS Thymoglobulin Autologous Stem Cell Transplantation BCNU Becenum Becenun BiCNU Bis(chloroethyl) Nitrosourea Bis-Chloronitrosourea Carmubris Carmustin Carmustinum FDA 0345 Gliadel N,N'-Bis(2-chloroethyl)-N-nitrosourea Nitrourean Nitrumon SK 27702 SRI 1720 WR-139021 154-93-8 .beta.-Cytosine arabinoside 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone 1-.beta.-D-Arabinofuranosylcytosine 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone 1-Beta-D-arabinofuranosylcytosine 1.beta.-D-Arabinofuranosylcytosine 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl- 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl- Alexan Ara-C ARA-cell Arabine Arabinofuranosylcytosine Arabinosylcytosine Aracytidine Aracytin Aracytine Beta-Cytosine Arabinoside CHX-3311 Cytarabinum Cytarbel Cytosar Cytosar-U Cytosine Arabinoside Cytosine-.beta.-arabinoside Cytosine-beta-arabinoside Erpalfa Starasid Tarabine PFS U 19920 U-19920 Udicil WR-28453 147-94-4 Demethyl Epipodophyllotoxin Ethylidine Glucoside EPEG Lastet Toposar Vepesid VP 16-213 VP-16 VP-16-213 33419-42-0 Alanine Nitrogen Mustard CB-3025 L-PAM L-Phenylalanine Mustard L-Sarcolysin L-Sarcolysin Phenylalanine mustard L-Sarcolysine Melphalanum Phenylalanine Mustard Phenylalanine Nitrogen Mustard Sarcoclorin Sarkolysin WR-19813 148-82-3 PBPC transplantation Peripheral Blood Progenitor Cell Transplantation Peripheral Stem Cell Support Peripheral Stem Cell Transplantation .delta.1-Cortisone 1, 2-Dehydrocortisone Adasone Cortancyl Dacortin DeCortin Decortisyl Decorton Delta 1-Cortisone Delta-Dome Deltacortene Deltacortisone Deltadehydrocortisone Deltasone Deltison Deltra Econosone Lisacort Meprosona-F Metacortandracin Meticorten Ofisolona Orasone Panafcort Panasol-S Paracort PRED Predicor Predicorten Prednicen-M Prednicort Prednidib Prednilonga Predniment Prednisonum Prednitone Promifen Servisone SK-Prednisone 53-03-2

Eligibility Criteria

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Inclusion Criteria

* 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
* Patients age =\< 70 years
* Evidence of disease activity as outlined (e.g. gadolinium enhancement on magnetic resonance imaging of the brain or clinical progression)
* Patients must have failed at least 2 lines of standard therapy as outlined for the specific diseases
* DONOR: Sibling of any patient enrolled on this protocol proven by ABO typing, human leukocyte antigen (HLA) typing and variable number tandem repeat (VNTR) analysis to be syngeneic with the patient (e.g. identical twin)
* DONOR: Willing to undergo multiple apheresis procedures (except donors \< 12 years who will undergo bone marrow harvests)

Exclusion Criteria

* Age \>= 71 years
* Pregnancy or expressed plans to become pregnant within 1 year of the procedure
* Patients who are serologically positive for human immunodeficiency virus (HIV)
* Patients with pulmonary, cardiac, hepatic or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival; this should include patients with any of the following:

* Severe pulmonary dysfunction associated with a carbon monoxide diffusing capacity (DLCO) (corrected for hemoglobin) \< 60%, or requires supplemental oxygen; patients who are unable to perform pulmonary function test (because of underlying disease) will be excluded if the oxygen saturation is \< 92% on room air
* Uncontrolled malignant arrhythmias, or clinical evidence of congestive heart failure (New York class III-IV) or ejection fraction \< 50%
* Renal disease with estimated glomerular filtration rate (GFR) by creatinine clearance or iothalamate clearance \< 50 ml/min/1.73 m\^2 body surface area
* Serum glutamate pyruvate transaminase (SGPT)/aspartate aminotransferase (AST) \> 3 times normal or direct bilirubin greater than 2.5 mg/dL on two repeated tests
* Active uncontrolled infection
* Demonstrated lack of compliance with prior medical care
* Patients whose life expectancy is limited by illness other than their neurological condition
* Patients with evidence of myelodysplasia
* Active malignancy (excluding localized squamous cell or basal cell carcinoma of the skin)
* DONOR: Inadequate documentation that donor and recipient are syngeneic
* DONOR: Donors who do not fulfill criteria as apheresis donors as established by institutional guidelines
* DONOR: Concordant for autoimmune neurological disease(s) as determined by neurological evaluation

Maximum Eligible Age

71 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No