ClinicalTrials.gov
ClinicalTrials.gov Menu

Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis

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: NCT00282438
Recruitment Status : Terminated (No plan to continue enrollment)
First Posted : January 26, 2006
Results First Posted : February 24, 2017
Last Update Posted : August 31, 2018
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sarcoidosis
Interventions Biological: Autologous hematopoietic stem cell transplantation
Biological: Allogeneic stem cell transplantation
Enrollment 2
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Hide Arm/Group Description

Autologous stem cells will be injected after conditioning

Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning

Allogeneic stem cells will be injected after conditioning

Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning

Period Title: Overall Study
Started 1 1
Completed 0 0
Not Completed 1 1
Reason Not Completed
Lack of Efficacy             1             0
Death             0             1
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation Total
Hide Arm/Group Description

Autologous stem cells will be injected after conditioning

Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning

Allogeneic stem cells will be injected after conditioning

Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning

Total of all reporting groups
Overall Number of Baseline Participants 1 1 2
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 1 participants 2 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
1
 100.0%
1
 100.0%
2
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 1 participants 1 participants 2 participants
54 [1]   (NA) 49 [2]   (NA) 51.5  (2.5)
[1]
Only one participant was analyzed and then the standard deviation could only be provided for the total column.
[2]
Only one participant was analyzed and then the standard deviation could only be provided for the total column
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 1 participants 2 participants
Female
0
   0.0%
1
 100.0%
1
  50.0%
Male
1
 100.0%
0
   0.0%
1
  50.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 1 participants 1 participants 2 participants
1 1 2
1.Primary Outcome
Title Presence of Toxicity
Hide Description Daily assessment will be made with regards to toxicity by one of the protocol investigators.National Cancer Institute Common Toxicity Criteria will be used to grade all non-hematologic toxicities. Toxicity grades as follows: 1 = Mild; 2 = Moderate; 3 = Severe and undesirable; 4 = life threatening or disabling ; 5 = Death
Time Frame For length of hospital stay (until discharge).
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Hide Arm/Group Description:

Autologous stem cells will be injected after conditioning

Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning

Allogeneic stem cells will be injected after conditioning

Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning

Overall Number of Participants Analyzed 1 1
Measure Type: Number
Unit of Measure: participants
1 1
2.Primary Outcome
Title Survival
Hide Description Patient has not died.
Time Frame Participants are to be followed at 6 months and then yearly until 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Hide Arm/Group Description:

Autologous stem cells will be injected after conditioning

Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning

Allogeneic stem cells will be injected after conditioning

Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning

Overall Number of Participants Analyzed 1 1
Measure Type: Count of Participants
Unit of Measure: Participants
1
 100.0%
0
   0.0%
3.Primary Outcome
Title Time to Disease Progression
Hide Description Worsening symptoms, pulmonary function studies, cardiac function and arrhythmia including EKG assessments, and neurological symptoms.
Time Frame Participants are to be followed at 6 months and then yearly until 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Hide Arm/Group Description:

Autologous stem cells will be injected after conditioning

Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning

Allogeneic stem cells will be injected after conditioning

Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning

Overall Number of Participants Analyzed 1 1
Measure Type: Number
Unit of Measure: months
4 0
Time Frame Adverse event data was collected from time of first transplant 4/25/2012 until patient death 6/27/2015. Study closure effective 8/17/2015.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Hide Arm/Group Description

Autologous stem cells will be injected after conditioning

Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning

Allogeneic stem cells will be injected after conditioning

Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning

All-Cause Mortality
Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/1 (0.00%)      1/1 (100.00%)    
Infections and infestations     
Death  [1]  0/1 (0.00%)  0 1/1 (100.00%)  1
Sepsis * [1]  0/1 (0.00%)  0 1/1 (100.00%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
[1]
70 days after the transplant developed staphylococcus aureus sepsis, complicated by ARDS and respiratory failure
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Autologous Hematopoietic Stem Cell Transplantation Allogeneic Stem Cell Transplantation
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/1 (0.00%)      0/1 (0.00%)    
Two patients had been enrolled on trial. One underwent autologous HSCT and relapsed early after the transplant. The second participant underwent allogeneic HSCT and died 70 days after the transplant and the study has been terminated.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr Richard Burt, Chief, Division of Immunotherapy, Department of Medicine
Organization: Northwestern University
Phone: 312-695-4960
Responsible Party: Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00282438     History of Changes
Other Study ID Numbers: NU FDA SARC.2003
First Submitted: January 24, 2006
First Posted: January 26, 2006
Results First Submitted: September 12, 2016
Results First Posted: February 24, 2017
Last Update Posted: August 31, 2018