Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica (SCT-NMO)

University of Calgary

Status and phase

Terminated

Phase 2

Phase 1

Conditions

Neuromyelitis Optica

Treatments

Procedure: AHSCT

Study type

Interventional

Funder types

Other

Identifiers

NCT01339455

CHREB ID# 23282

Details and patient eligibility

About

Neuromyelitis Optica (NMO) is a demyelinating and degenerative disorder of the CNS affecting vision and spinal cord function. This disease is rare compared to Multiple Sclerosis (MS), but it is devastating and often leads to accumulating disability with a 5 year-mortality of approximately 30%. Survivors are typically left with severe morbidity secondary to blindness, quadriparesis and respiratory failure. No agent has been found to be highly effective in halting disease activity. Based on recent outcomes of stem cell transplant trials and reports in autoimmune diseases including MS, and based on the mechanisms of NMO, we anticipate that stem cell transplantation may provide lasting disease stability for NMO patients. The hypothesis of the present trial is that autologous hematopoetic stem cell transplantation in patients with NMO will provide lasting benefit in relapse prevention. Specifically, we anticipate a 50% reduction in the proportion of patients experiencing relapse over a three year period. We will be following patients for a total of five years after transplantation.

Full description

Patients who are deemed eligible will be enrolled and undergo a two stage transplant process followed by neurological assessments every 6 months for the following 5 years assessing EDSS, visual metrics, MRI, AQP-4 antibodies, MSFC and SF36.

Enrollment

3 patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Age between 18-65, inclusive
Diagnosis of NMO using Wingerchuk 2006 NMO Criteria
EDSS 0-6.5
Treatment with a minimum of one NMO therapy in past 12 months
One objective and documented relapse in the past 12 months and two relapse events in the past 24 months despite medical therapy
ECOG performance status 0-3
Platelets ≥100 x 109/L
ALT ≤3 x ULN
Total bilirubin ≤2.0 x ULN, except in patients with Gilbert syndrome or in patients in whom the bilirubin rise is of non-hepatic origin
Serum creatinine <1.5 x ULN or creatinine clearance ≥50 cc/min
Patients must reside in Alberta, Canada for the duration of the transplant period of the trial

Exclusion criteria

Any illness that would jeopardize the ability of the patient to complete study protocol
Prior malignancy unless non-melanoma skin cancer, carcinoma in-situ of the cervix (CIN) or breast, or malignancy treated more than 5 years previously with no evidence of recurrent disease since initial treatment
Pregnant or lactating females. Women of childbearing potential must have a negative serum or urine β-hCG pregnancy test at screening
Inability or unwillingness to pursue effective means of birth control
FEV1/FVC < 50% of predicted
DLCO < 50% of predicted
Resting LVEF < 50 %
Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins, or to iron compounds/medications
Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams
Unable or unwilling to provide written informed consent for participation
Active infection except asymptomatic bacteriuria
Any use of investigational therapies within 4 weeks prior to initiation of study treatment
Patients dependent on prednisone who cannot be successfully tapered to a maximum of 0.5mg/kg/d prior to mobilization therapy