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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT00716066
Date of registration: 15/07/2008
Prospective Registration: Yes
Primary sponsor: Fred Hutchinson Cancer Research Center
Public title: Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune Diseases
Scientific title: High-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic Diseases
Date of first enrolment: June 2008
Target sample size: 40
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT00716066
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Bernie McLaughlin
Address: 
Telephone: 206.667-4916
Email: bmclaugh@fredhutch.org
Affiliation: 
Name:     George Georges
Address: 
Telephone:
Email:
Affiliation:  Fred Hutch/University of Washington Cancer Consortium
Key inclusion & exclusion criteria

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

- 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:

- 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



Age minimum: N/A
Age maximum: 71 Years
Gender: All
Health Condition(s) or Problem(s) studied
Autoimmune Disease
Autoimmune Nervous System Disorder
Autologous Transplant Autoimmune
Central Nervous System Vasculitis
Cerebellar Degeneration
Chronic Inflammatory Demyelinating Polyneuropathy
CIDP Transplant
HCT for Neurologic Autoimmune Disorders
Lambert Eaton Myasthenic Syndrome
MS Stem Cell Transplant
Multiple Sclerosis Stem Cell Transplant
Multiple Sclerosis Transplant
Myasthenia Gravis
Myasthenia Gravis Transplant
Neurologic Autoimmune Disease
Neuromyelitis Optica
Opsoclonus Myoclonus Syndrome
Rasmussen Subacute Encephalitis
Stiff Person Syndrome
Intervention(s)
Biological: Anti-Thymocyte Globulin
Drug: Carmustine
Drug: Cytarabine
Drug: Etoposide
Drug: Melphalan
Drug: Prednisone
Other: Laboratory Biomarker Analysis
Procedure: Autologous Hematopoietic Stem Cell Transplantation
Procedure: Peripheral Blood Stem Cell Transplantation
Procedure: Syngeneic Bone Marrow Transplantation
Primary Outcome(s)
Incidence of grades 4-5 regimen-related toxicity [Time Frame: Within 28 days post-transplant]
Secondary Outcome(s)
Disease responses [Time Frame: Up to 5 years]
Engraftment kinetics [Time Frame: Over first 60 days post-transplant]
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 [Time Frame: Baseline to post mobilization, assessed up to 20 days after starting final mobilization (up to two mobilizations)]
Number of subjects with an exacerbation of autoimmune disease symptoms secondary to G-CSF (filgrastim) during peripheral blood stem cell mobilization [Time Frame: Baseline to post mobilization, assessed up to 20 days after starting final mobilization (up to two mobilizations)]
Transplant-related mortality [Time Frame: Within 100 days post-transplant]
Secondary ID(s)
2260.00
NCI-2010-00403
P30CA015704
RG9213030
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
National Cancer Institute (NCI)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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