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Outpatient Hematopoietic Grafting in Multiple Sclerosis Employing Autologous Peripheral Blood Stem Cells

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ClinicalTrials.gov Identifier: NCT02674217
Recruitment Status : Enrolling by invitation
First Posted : February 4, 2016
Last Update Posted : January 31, 2019
Sponsor:
Information provided by (Responsible Party):
Guillermo J. RUIZ-ARGÜELLES, Centro de Hematología y Medicina Interna

Brief Summary:
Multiple sclerosis (MS) is a chronic, inflammatory, debilitating disease that causes destruction of central nervous system (CNS) myelin, with varying degrees of axonal damage. It mainly affects young adults and is twice as common in women as in men (1). Studies published from the 1990s brought animal models and theoretical considerations of hematopoietic stem cell transplantation (HSCT) being useful in the prevention and treatment of autoimmune diseases, with clinical responses in some patients, suggesting that high-dose chemotherapy followed by HSCT rescue could "reset" the immunological changes through the control of autoreactive clones, followed by immunological tolerance after immune reconstitution (2); this led to the conclusion that HSCT may be a viable therapeutic option for MS (1-6). Autologous HSCT have been done in patients with MS since 1996 and more than 700 HSCTs have been performed around the world (1-6). Most patients have been treated in small trials or in multicenter studies. In retrospective analyzes, a progression-free survival of more than five years after transplant has been observed, the neurological outcomes being considerably more favorable in patients with the relapsing-remitting type and/or those who showed an inflammatory pattern in magnetic resonance imaging (MRI) during the pre-transplant screening. Reports of good results, particularly in the aggressive forms of MS reinforce the effectiveness HSCT in MS patients with prominent inflammatory activity. The risk of transplant related mortality in HSCT for MS was conventionally considered very high but has declined since 2001 to 1.3% (2-6), this probably being the result of the changes in the conditioning regimens, thus reducing toxicity. Recent data, with more than 700 autologous transplants for MS in Europe, showed an overall survival of 92% in five years and a progression-free survival of 46%, the main cause of mortality and morbidity being the recurrence of the autoimmune disease (2-6). The consensus provides an indication of HSCT in patients with progressive MS unresponsive to conventional therapy and Expanded Disability Status Scale (EDSS) (1) between 3.0 and 6.0. The forms of the disease that might benefit from transplantation are: relapsing remitting, primary or secondary progressive, and the "malignant" form, provided there is evidence of inflammatory activity at the time of transplant indication.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Procedure: Hematopoietic stem cell transplantation Not Applicable

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 1000 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Outpatient Hematopoietic Grafting in Patients With Multiple Sclerosis Employing Autologous Non-cryopreserved Peripheral Blood Stem Cells: A Feasibility Study
Study Start Date : May 2015
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Multiple sclerosis autografted patients
Individuals with a relapsing-remitting (RRMS) course, secondary progressive (SPMS) or primary progressive (PPMS) were included. Patients should have a Karnofsky performance status (18) above 70% and a EDSS score (1) of 6 or below. The study is approved by the Etichs Committee of the Clinica RUIZ and all patients signed a consent form after being fully informed about procedure an possible complications. Patients included will de treated with Hematopoietic stem cell transplantation
Procedure: Hematopoietic stem cell transplantation
autologous transplant using non-frozen peripheral blood stem cells
Other Name: autologous transplant




Primary Outcome Measures :
  1. Expanded Disability Status Scale [ Time Frame: 4 years ]
    EDSS


Secondary Outcome Measures :
  1. Overall survival [ Time Frame: 4 years ]
    OS



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with MS having a relapsing-remitting course, even though other forms could also qualify: Secondary progressive (SPMS) or primary progressive (PPMS)
  • Patients must have a Karnofsky performance status above 70% and a EDSS score of 8 or below.
  • In cases of patients with score between 6 and 8 patients will be accepted provided a carer accompanies the patient.
  • A recent central nervous system (CNS) MRI study (less than two months).
  • Patient has to be able to travel to and remain in Puebla, México during an 4-week period, accompanied by a caregiver.
  • Discontinue Immune Modulation or suppression medications 3 months before.

Exclusion criteria:

  • EDSS score higher than 8
  • Karnofsky performance status lower than 70%
  • Been exposed to chemotherapy in the past

Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Guillermo J. RUIZ-ARGÜELLES, Director, Centro de Hematología y Medicina Interna
ClinicalTrials.gov Identifier: NCT02674217     History of Changes
Other Study ID Numbers: CentroHMI
U1111-1195-1632 ( Registry Identifier: Universal Trial Number )
First Posted: February 4, 2016    Key Record Dates
Last Update Posted: January 31, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Additional relevant MeSH terms:
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Multiple Sclerosis
Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases