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Hematopoietic Stem Cell Therapy for Patients With Multiple Sclerosis

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ClinicalTrials.gov Identifier: NCT00278655
Recruitment Status : Terminated
First Posted : January 18, 2006
Results First Posted : May 1, 2014
Last Update Posted : May 1, 2014
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Multiple Sclerosis
Intervention Biological: Hematopoietic stem cell transplantation
Enrollment 21
Recruitment Details Between January 2003 and February 2005 in Northwestern Memorial Hospital 21 patients with relapsing-remitting multiple sclerosis (MS) underwent autologous hematopoietic stem cell transplantation in order to evaluate the safety and clinical outcome of autologous non-myeloablative hematopoetic stem cell transplantation in MS.
Pre-assignment Details 21 eligible patients had relapsing remitting MS not responding to interferon and had had two corticosteroid -treated relapses within the previous 12 months or one relapse and gadolinium-enhancing lesions seen on MRI and separate from the relapse.
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description Patient's own blood stem cells collected after mobilization with cyclophosphamide (2.0 gm/m²)and granulocyte colony-stimulating factor (G-CSF) (5-10mcg/kg/day.Collected stem cells given back to the patient after receiving conditioning regimen (Cyclophosphamide 50 mg/kg/day for four days and either 20 mg alemtuzumab or 6mg/kg rabbit antithymocyte globulin.
Period Title: Overall Study
Started 21
Completed 21
Not Completed 0
Arm/Group Title Stem Cell Transplantation
Hide Arm/Group Description

All participants will undergo stem cell transplantation after receiving conditioning regimen.

hematopoietic stem cell transplantation : Autologous hematopoietic stem cell transplantation

Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
<=18 years
0
   0.0%
Between 18 and 65 years
21
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 21 participants
21  (2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
11
  52.4%
Male
10
  47.6%
1.Primary Outcome
Title Disease Progression
Hide Description Data are reporting number of participants with disease progression. Disease progression is defined as a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 3 months apart.
Time Frame 3 years after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Stem Cell Transplantation
Hide Arm/Group Description:
Autologous hematopoietic stem cell transplantation Peripheral blood stem cells were mobilised with 2g per square m intravenous (IV) cyclophosphamide (CY) followed by 10 µg per kg subcutaneous filgrastim daily from day 5. After neutrophil recovery, the mobilised cells were collected by apheresis. The recovered cells were unselected and cryopreserved. There was at least three weeks between mobilisation and the conditioning regimen. The conditioning regimen used was 200 mg per kg intravenous CY , given in four equal fractions between day -5 and day -2 with IV mesna, and one 20mg dose of IV alemtuzumab (CAMPATH-1H) given on day -2 with 250 mg intravenous methyl-prednisolone as premedication. Alemtuzumab was changed to rabbit antithymocyte globulin rATG) in the last 4 patients, who received 6 mg per kg rATG over 5 days instead of alemtuzumab . Stem cells wer reinfused 36 h after completion of CY. 5 µg/kg/day filgrastim was given from day 5 until neutrophil recovery.
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: participants
4
2.Secondary Outcome
Title Survival
Hide Description

Data are reporting the number of participants who survived three years after the transplant

Survival of 21 participants was evaluated at three years after the transplant

Time Frame three years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Patient's own blood stem cells collected after mobilization with cyclophosphamide (2.0 gm/m²)and G-CSF (5-10 mcg/kg/day.Collected stem cells given back to the patient after receiving conditioning regimen (Cyclophosphamide 50 mg/kg/day for four days and either 20 mg alemtuzumab or 6mg/kg rabbit antithymocyte globulin.
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: participants
21
Time Frame three years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Stem Cell Transplantation
Hide Arm/Group Description

All participants will undergo stem cell transplantation after receiving conditioning regimen.

hematopoietic stem cell transplantation : Autologous hematopoietic stem cell transplantation

All-Cause Mortality
Stem Cell Transplantation
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   0/21 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   7/21 (33.33%)    
Blood and lymphatic system disorders   
Grade IV thrombocytopenia  [1]  2/21 (9.52%)  2
Gastrointestinal disorders   
Diarrhoea due to Clostridium difficile  [2]  1/21 (4.76%)  1
Infections and infestations   
Blood culture with coagulase-negative staphylococcus  [3]  1/21 (4.76%)  1
Dermatomal zoster  [4]  2/21 (9.52%)  2
Nervous system disorders   
Transient neurological symptoms  [5]  1/21 (4.76%)  1
Indicates events were collected by systematic assessment
[1]
Two patients treated with alemtuzumab developed grade IV thrombocytopenia 7 and 14 months after transplantation. The thrombocytopenia presented as easy bruising and petechiae, resolved with intravenous immunoglobulin, prednisone and rituximab
[2]
One month after hospital discharge after the transplantation, one patient developed diarrhoea due to Clostridium difficile that resolved with oral metronidazole.
[3]
One patient had a blood culture with coagulase-negative staphylococcus; this was isolated in one specimen of four sent concurrently, which suggests a probable contaminant.
[4]
Two patients developed dermatomal zoster at 20 and 22 months post-transplantation and both resolved with oral antiviral therapy.
[5]
One patient had Transient neurological symptoms that manifested as leftsided hypoaesthesia, which was attributed to a filgrastim-related flair. The neurological symptoms resolved when the drug was discontinued.
The study had following limitations: small number of enrolled participants and no randomization.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr Richard Burt
Organization: Northwestern University
Phone: 312-908-0059
EMail: rburt@northwestern.edu
Layout table for additonal information
Responsible Party: Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00278655     History of Changes
Other Study ID Numbers: DIAD MS.Auto2002
First Submitted: January 16, 2006
First Posted: January 18, 2006
Results First Submitted: April 9, 2013
Results First Posted: May 1, 2014
Last Update Posted: May 1, 2014