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Sirolimus for Autoimmune Disease of Blood Cells

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00392951
First Posted: October 26, 2006
Last Update Posted: December 6, 2017
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.
Information provided by (Responsible Party):
Children's Hospital of Philadelphia
Results First Submitted: June 1, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Autoimmune Pancytopenia
Autoimmune Lymphoproliferative Syndrome (ALPS)
Evans Syndrome
Idiopathic Thrombocytopenic Purpura
Anemia, Hemolytic, Autoimmune
Autoimmune Neutropenia
Lupus Erythematosus, Systemic
Inflammatory Bowel Disease
Rheumatoid Arthritis
Intervention: Drug: sirolimus

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Sirolimus Treatment

Sirolimus treatment

sirolimus: Tablet or liquid; taken once or twice daily; dosage is based on establishing a serum trough of 5-15 ng/ml by high-performance liquid chromatography (initial loading dose of 3 mg/m2 then 2.5 mg/m2 with adjustment based on serum trough)


Participant Flow:   Overall Study
    Sirolimus Treatment
STARTED   30 
COMPLETED   30 
NOT COMPLETED   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Sirolimus Treatment

Sirolimus treatment

sirolimus: Tablet or liquid; taken once or twice daily; dosage is based on establishing a serum trough of 5-15 ng/ml by high-performance liquid chromatography (initial loading dose of 3 mg/m2 then 2.5 mg/m2 with adjustment based on serum trough)


Baseline Measures
   Sirolimus Treatment 
Overall Participants Analyzed 
[Units: Participants]
 30 
Age 
[Units: Participants]
Count of Participants
 
Participants Analyzed 
[Units: Participants]
 30 
<=18 years      12  40.0% 
Between 18 and 65 years      18  60.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Median (Inter-Quartile Range)
 
Participants Analyzed 
[Units: Participants]
 30 
   11 
 (1.8 to 21) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Participants Analyzed 
[Units: Participants]
 30 
Female      10  33.3% 
Male      20  66.7% 
Race and Ethnicity Not Collected [1] 
[Units: Participants]
Count of Participants
  
[1] Race and Ethnicity were not collected from any participant.
Region of Enrollment 
[Units: Participants]
Count of Participants
 
United States   
Participants Analyzed 
[Units: Participants]
 30 
United States   30 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Participants With Grade 3 and 4 Toxicities of Administration of Oral Sirolimus   [ Time Frame: 6 months ]

2.  Secondary:   Number of Participants With Autoimmune Disease Response to Oral Sirolimus   [ Time Frame: 6 months ]

3.  Secondary:   Trough Levels Produced by Administration of Oral Sirolimus   [ Time Frame: Within first 5 days of starting sirolimus ]

4.  Secondary:   Effect of Sirolimus on Intracellular Targets   [ Time Frame: 6 months ]

5.  Secondary:   Number of Participants With Lymphoproliferation Response to Oral Sirolimus   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: David T. Teachey, MD
Organization: Children's Hospital of Philadelphia
phone: 267-426-5802
e-mail: teacheyd@email.chop.edu


Publications of Results:
Other Publications:

Responsible Party: Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00392951     History of Changes
Other Study ID Numbers: 2006-7-4873
First Submitted: October 24, 2006
First Posted: October 26, 2006
Results First Submitted: June 1, 2017
Results First Posted: December 6, 2017
Last Update Posted: December 6, 2017