Stem Cell Transplantation for Stiff Person Syndrome (SPS)

Summary

Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment.

When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. While not necessary for recovery, stem cell infusion may shorten the interval of neutropenia and attendant complications. Thus in reality there is no transplant only an autologous supportive blood product.

Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate.

Conditions

• Stiff-Person Syndrome

Interventions

BIOLOGICAL

Autologous Hematopoietic Stem Cells

The stem cells will be collected from patient's blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous Hematopoietic Stem Cell Transplantation is to re-infuse immature cells that can re-establish blood production and patient's immune system.

DRUG

Cyclophosphamide

An alkylating agent which causes prevention of cell division by forming adducts with DNA

DRUG

Mesna

Medication used to decrease the risk of hemorrhagic cystitis prophylaxis

DRUG

rATG

A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells

DRUG

Methylprednisolone

Steroid

DRUG

G-CSF

Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream

DRUG

Rituxan

A chimeric monoclonal antibody used in the treatment of B cell non-Hodgkin's lymphoma, B cell leukemia, and some autoimmune disorders

Sponsors & Collaborators

• Northwestern University

  lead OTHER

Principal Investigators

• Richard Burt, MD · Northwestern University

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Max Age

60 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2014-10-31

Primary Completion

2019-08-19

Completion

2019-08-30

FDA Drug

Yes

Countries

• United States

Study Locations