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Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT01538862
Recruitment Status : Completed
First Posted : February 24, 2012
Results First Posted : April 25, 2017
Last Update Posted : June 23, 2017
Sponsor:
Information provided by (Responsible Party):
Haydar Frangoul, Vanderbilt University Medical Center

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Dystrophic Epidermolysis Bullosa
Intervention: Drug: Granulocyte Colony Stimulating Factor (GCSF)

  Participant Flow

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
Granulocyte Colony Stimulating Factor (GCSF)

GCSF 10mcg/kg/d SQ for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d SQ for 7 days


Participant Flow:   Overall Study
    Granulocyte Colony Stimulating Factor (GCSF)
STARTED   7 
COMPLETED   7 
NOT COMPLETED   0 



  Baseline Characteristics

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
Granulocyte Colony Stimulating Factor (GCSF)

GCSF 10mcg/kg/d SQ for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d SQ for 7 days


Baseline Measures
   Granulocyte Colony Stimulating Factor (GCSF) 
Overall Participants Analyzed 
[Units: Participants]
 7 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      6  85.7% 
Between 18 and 65 years      1  14.3% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      4  57.1% 
Male      3  42.9% 
Region of Enrollment 
[Units: Participants]
 
United States   7 


  Outcome Measures

1.  Primary:   Percent Change of Active Blisters and in Total Blister/Erosion Counts   [ Time Frame: 7 days ]

2.  Secondary:   Surface Area of Nonhealing Erosions   [ Time Frame: 7 days ]

3.  Secondary:   Overall Improved Symptomatology   [ Time Frame: 28 days ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Haydar Frangoul, MD, MS
Organization: Vanderbilt University
phone: 615-342-7339
e-mail: Haydar.Frangoul@hcahealthcare.com



Responsible Party: Haydar Frangoul, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT01538862     History of Changes
Other Study ID Numbers: VICCNCPED1210
First Submitted: February 20, 2012
First Posted: February 24, 2012
Results First Submitted: April 1, 2016
Results First Posted: April 25, 2017
Last Update Posted: June 23, 2017