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Trial record 18 of 1098 for:    stem cells | Studies With Results

Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00278629
Recruitment Status : Completed
First Posted : January 18, 2006
Results First Posted : July 23, 2020
Last Update Posted : July 23, 2020
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Chronic Inflammatory Demyelinating Polyneuropathy
Intervention Biological: hematopoietic stem cell transplantation
Enrollment 80
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Period Title: Overall Study
Started 80
Completed 66
Not Completed 14
Reason Not Completed
Non-CIDP Immune-Mediated Peripheral Neur             14
Arm/Group Title Hematopoietic Stem Cell Transplantation for CIDP
Hide Arm/Group Description Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Overall Number of Baseline Participants 66
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 60 participants
<=18 years
0
   0.0%
Between 18 and 65 years
60
 100.0%
>=65 years
0
   0.0%
[1]
Measure Analysis Population Description: The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Age, Continuous   [1] 
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 60 participants
43
(20 to 63)
[1]
Measure Analysis Population Description: The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 60 participants
Female
37
  61.7%
Male
23
  38.3%
[1]
Measure Analysis Population Description: The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Region of Enrollment   [1] 
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 60 participants
60
[1]
Measure Analysis Population Description: The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
1.Primary Outcome
Title Number of Participants With Survival
Hide Description Survival of participants who survived the treatment and up to 5 years post treatment.
Time Frame up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
The reason that the number analyzed is different from the overall number of participants anaylyzed is because 2 patients died from non treatment related conditions that were present before the transplant. Both deaths occurred more than 100 days after the hospital discharge from treatment.
Arm/Group Title Hematopoietic Stem Cell Transplantation for CIDP
Hide Arm/Group Description:
Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV
Overall Number of Participants Analyzed 66
Measure Type: Count of Participants
Unit of Measure: Participants
Treatment Survival
66
 100.0%
Post Treament Survival
64
  97.0%
2.Secondary Outcome
Title Disease Improvement - Medication Free Remission
Hide Description Post transplant immune medication-free remission with stable or improving neurological exam
Time Frame 6 months, 1, 2, 3, 4 and 5 years post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed in one or more rows differs from the over all number because not all patients returned for follow up.
Arm/Group Title Hematopoietic Stem Cell Transplantation for CIDP
Hide Arm/Group Description:
Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Overall Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: participants
6 months post transplant Number Analyzed 60 participants
48
1 year post transplant Number Analyzed 60 participants
48
2 years after transplant Number Analyzed 60 participants
47
3 years post transplant Number Analyzed 59 participants
45
4 years post transplant Number Analyzed 50 participants
39
5 years post transplant Number Analyzed 42 participants
35
3.Secondary Outcome
Title Disease Improvement - Ambulatory Assistance
Hide Description Disease Improvement - Requires No Assistance to Ambulate
Time Frame pre transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed in one or more rows differs from the overall number analyzed because not all patients returned for followed up testing.
Arm/Group Title Hematopoietic Stem Cell Transplantation for CIDP
Hide Arm/Group Description:
Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Overall Number of Participants Analyzed 60
Measure Type: Count of Participants
Unit of Measure: Participants
pre transplant Number Analyzed 60 participants
19
  31.7%
6 months post transplant Number Analyzed 60 participants
41
  68.3%
1 year post transplant Number Analyzed 60 participants
49
  81.7%
2 years post transplant Number Analyzed 59 participants
48
  81.4%
3 years post transplant Number Analyzed 59 participants
48
  81.4%
4 years post transplant Number Analyzed 50 participants
43
  86.0%
5 years post transplant Number Analyzed 42 participants
35
  83.3%
4.Secondary Outcome
Title Change in Disability and Strength Functional Scales
Hide Description Change in the Medical Research Council (MRC) Scale: range is 0 (quadriplegic) - 60 (normal).
Time Frame Pre Transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
The reason the number analyzed in one or more rows differs from the overall number analyzed is because not all patients returned for followed up testing.
Arm/Group Title Hematopoietic Stem Cell Transplantation for CIDP
Hide Arm/Group Description:
Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Overall Number of Participants Analyzed 60
Mean (Standard Deviation)
Unit of Measure: score on a scale
Pre Transplant Number Analyzed 60 participants
51.8  (6.3)
6 Months Post Transplant Number Analyzed 50 participants
54.8  (5.6)
1 year post transplant Number Analyzed 45 participants
57  (4.18)
2 years post transplant Number Analyzed 44 participants
57  (4.3)
3 years post transplant Number Analyzed 39 participants
57  (3.9)
4 years post transplant Number Analyzed 33 participants
57  (3.9)
5 years post transplant Number Analyzed 26 participants
57  (4.3)
Time Frame Up to 5 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Hematopoietic Stem Cell Transplantation for CIDP
Hide Arm/Group Description Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
All-Cause Mortality
Hematopoietic Stem Cell Transplantation for CIDP
Affected / at Risk (%)
Total   2/66 (3.03%)    
Hide Serious Adverse Events
Hematopoietic Stem Cell Transplantation for CIDP
Affected / at Risk (%) # Events
Total   2/66 (3.03%)    
Cardiac disorders   
Non Transplant Related Death   1/66 (1.52%)  1
Gastrointestinal disorders   
Total Parenteral Nutrition - due to vomiting   1/66 (1.52%) 
Metabolism and nutrition disorders   
Hypokalemia   1/66 (1.52%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Non Transplant Related Death  [1]  1/66 (1.52%)  1
Respiratory, thoracic and mediastinal disorders   
Transient Dyspnea   1/66 (1.52%)  1
Indicates events were collected by systematic assessment
[1]
Gastric Cancer
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Hematopoietic Stem Cell Transplantation for CIDP
Affected / at Risk (%) # Events
Total   29/66 (43.94%)    
General disorders   
Febrile Neutropenia   26/66 (39.39%)  26
Infections and infestations   
Infections   8/66 (12.12%)  8
Clostridium Difficile Diarrhea   4/66 (6.06%)  4
Metabolism and nutrition disorders   
Hypophosphatemia   29/66 (43.94%)  29
Hypokalemia   19/66 (28.79%)  19
Hypocalcemia   5/66 (7.58%)  5
Hyponatremia   5/66 (7.58%)  5
Respiratory, thoracic and mediastinal disorders   
Dyspnea   8/66 (12.12%)  8
Indicates events were collected by systematic assessment
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: Kathleen Quigley
Organization: Northwestern University
Phone: 312-695-8192
EMail: k-quigley@northwestern.edu
Layout table for additonal information
Responsible Party: Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00278629    
Other Study ID Numbers: NU FDA CIDP.AUTO2003
First Submitted: January 16, 2006
First Posted: January 18, 2006
Results First Submitted: June 29, 2020
Results First Posted: July 23, 2020
Last Update Posted: July 23, 2020