Clinical Trial: Stem Cell Transplantation for Stiff Person Syndrome (SPS)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Non-myeloablative Hematopoietic Stem Cell Transplantation for Stiff Person Syndrome (SPS) and Anti-GAD Antibody Variants: Progressive Encephalomyelitis With Rigidity and M

Brief 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.


Detailed Summary:

Pre-study Testing

1. History and physical; 2. Electrocardiogram (EKG); 3. Dobutamine stress echocardiogram; 4. High-resolution computed tomography of the chest (HRCT); 5. Blood draw for laboratory tests- these tests will include a complete blood count, evaluating liver and kidney function, assessing immune system, tissue typing, and checking for certain germs that can cause infections, including a pregnancy test for females and prostate-specific antigen (PSA) for male as well as testing for HIV; 6. Pulmonary Function Test (PFT); 7. Electromyography (EMG); 8. Magnetic Resonance Imaging (MRI) of the Abdomen and Pelvis; 9. Magnetic Resonance Imaging (MRI) of the Spinal Cord; 10. Magnetic Resonance Imaging (MRI) of the Brain with Gadolinium (only if PERM of cerebellar ataxia); 11. Colonoscopy; 12. Mammogram (if female; 13. Timed ambulation; 14. Quality of Life Questionnaires [ Short Form (36) Health Survey (SF36) and Barthel Index]; 15. Chronic Pain Acceptance Questionnaire (CPAQ);16. Rankin Functional Scale; 17. Modified Ashworth Scale;18. PCA-1, PCA-2 antibody ( only if cerebellar ataxia); 19. Spinocerebellar ataxia (SCA) 1, 2, 3, 4, 5, 6, 7, 8 genes (only if ataxia)

Study Treatment

Stem Cell Collection-Cyclophosphamide 2.0 gm/m2 will be given on day 0, G-CSF 5-10 mcg/kg/day SQwill start on day +5 and will continue until apheresis is discontinued. Apheresis will begin when the ANC > 1.0 x 109/L and continue until >2.0 x 106 CD34+ cells/kg patient weight are cryopreserved. A 10-15 liter apheresis will be performed unless stopped earlier for clinical judgment of toxicity (e.g., numbness, tetany). A maximum of four apheresis will be performed.

Conditioning regimen Cyclophosphamide 50 mg/kg/day will be given IV over 1 hour i
Sponsor: Northwestern University

Current Primary Outcome: Overall Survival [ Time Frame: Up to 5 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Decrease (50%) and complete discontinuation of muscle relaxation anti-spasmatic medications [ Time Frame: Up to 5 years ]
  • Improvement of Chronic Pain Acceptance Questionnaire (CPAQ) [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in the CPAQ score
  • Timed ambulation [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in the timed ambulation score
  • Activities of Daily Living ((Barthel Index) [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in Barthel Index score
  • Short-form 36 quality of life questionnaire (SF-36 QOL) [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in SF-36 QOL score
  • Rankin Functional Scale [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in Rankin Functional Scale score


Original Secondary Outcome:

  • Decrease (50%) and complete discontinuation of muscle relaxation anti-spasmotic medications [ Time Frame: Up to 5 years ]
  • Improvement of Chronic Pain Acceptance Questionnaire (CPAQ) [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in the CPAQ score
  • Timed ambulation [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in the timed ambulation score
  • Activities of Daily Living ((Barthel Index) [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in Barthel Index score
  • Short-form 36 quality of life questionnaire (SF-36 QOL) [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in SF-36 QOL score
  • Rankin Functional Scale [ Time Frame: Up to 5 years ]
    Improvement is defined as a statistically significant change in Rankin Functional Scale score


Information By: Northwestern University

Dates:
Date Received: October 30, 2014
Date Started: October 2014
Date Completion: October 2021
Last Updated: March 9, 2017
Last Verified: March 2017