Trial Outcomes & Findings for Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis (NCT NCT00282438)
NCT ID: NCT00282438
Last Updated: 2018-08-31
Results Overview
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
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
2 participants
Primary outcome timeframe
For length of hospital stay (until discharge).
Results posted on
2018-08-31
Participant Flow
Participant milestones
| Measure |
Autologous Hematopoietic Stem Cell Transplantation
Autologous stem cells will be injected after conditioning
Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning
|
Allogeneic Stem Cell Transplantation
Allogeneic stem cells will be injected after conditioning
Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Autologous Hematopoietic Stem Cell Transplantation
Autologous stem cells will be injected after conditioning
Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning
|
Allogeneic Stem Cell Transplantation
Allogeneic stem cells will be injected after conditioning
Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning
|
|---|---|---|
|
Overall Study
Lack of Efficacy
|
1
|
0
|
|
Overall Study
Death
|
0
|
1
|
Baseline Characteristics
Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis
Baseline characteristics by cohort
| Measure |
Autologous Hematopoietic Stem Cell Transplantation
n=1 Participants
Autologous stem cells will be injected after conditioning
Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning
|
Allogeneic Stem Cell Transplantation
n=1 Participants
Allogeneic stem cells will be injected after conditioning
Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
54 years
STANDARD_DEVIATION NA • n=5 Participants
|
49 years
STANDARD_DEVIATION NA • n=7 Participants
|
51.5 years
STANDARD_DEVIATION 2.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: For length of hospital stay (until discharge).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
Outcome measures
| Measure |
Autologous Hematopoietic Stem Cell Transplantation
n=1 Participants
Autologous stem cells will be injected after conditioning
Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning
|
Allogeneic Stem Cell Transplantation
n=1 Participants
Allogeneic stem cells will be injected after conditioning
Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning
|
|---|---|---|
|
Presence of Toxicity
|
1 participants
|
1 participants
|
PRIMARY outcome
Timeframe: Participants are to be followed at 6 months and then yearly until 5 yearsPatient has not died.
Outcome measures
| Measure |
Autologous Hematopoietic Stem Cell Transplantation
n=1 Participants
Autologous stem cells will be injected after conditioning
Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning
|
Allogeneic Stem Cell Transplantation
n=1 Participants
Allogeneic stem cells will be injected after conditioning
Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning
|
|---|---|---|
|
Survival
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Participants are to be followed at 6 months and then yearly until 5 yearsWorsening symptoms, pulmonary function studies, cardiac function and arrhythmia including EKG assessments, and neurological symptoms.
Outcome measures
| Measure |
Autologous Hematopoietic Stem Cell Transplantation
n=1 Participants
Autologous stem cells will be injected after conditioning
Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning
|
Allogeneic Stem Cell Transplantation
n=1 Participants
Allogeneic stem cells will be injected after conditioning
Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning
|
|---|---|---|
|
Time to Disease Progression
|
4 months
|
0 months
|
Adverse Events
Autologous Hematopoietic Stem Cell Transplantation
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Allogeneic Stem Cell Transplantation
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Autologous Hematopoietic Stem Cell Transplantation
n=1 participants at risk
Autologous stem cells will be injected after conditioning
Autologous hematopoietic stem cell transplantation: Autologous hematopoietic stem cells will be injected after conditioning
|
Allogeneic Stem Cell Transplantation
n=1 participants at risk
Allogeneic stem cells will be injected after conditioning
Allogeneic stem cell transplantation: Allogeneic stem cells will be injected after conditioning
|
|---|---|---|
|
Infections and infestations
Death
|
0.00%
0/1 • 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.
|
100.0%
1/1 • Number of events 1 • 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.
|
|
Infections and infestations
Sepsis
|
0.00%
0/1 • 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.
|
100.0%
1/1 • Number of events 1 • 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.
|
Other adverse events
Adverse event data not reported
Additional Information
Dr Richard Burt, Chief, Division of Immunotherapy, Department of Medicine
Northwestern University
Phone: 312-695-4960
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place