Trial Outcomes & Findings for Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa (NCT NCT01538862)

Results Overview

Percent change of active blisters and in total blister/erosion counts from baseline to 7 days

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

7 participants

Primary outcome timeframe

7 days

Results posted on

2017-06-23

Participant Flow

Participant milestones

Participant milestones
Measure	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
Overall Study STARTED	7
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Granulocyte Colony Stimulating Factor (GCSF) n=7 Participants GCSF 10mcg/kg/d SQ for 7 days Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d SQ for 7 days
Age, Categorical <=18 years	6 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants

PRIMARY outcome

Timeframe: 7 days

Population: Time frame was originally entered as 30 days. This was not consistent with the protocol which listed a 7 day time frame for this Outcome

Percent change of active blisters and in total blister/erosion counts from baseline to 7 days

Outcome measures

Outcome measures
Measure	Granulocyte Colony Stimulating Factor (GCSF) n=7 Participants GCSF 10mcg/kg/d subcutaneously for 7 days Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d subcutaneously for 7 days
Percent Change of Active Blisters and in Total Blister/Erosion Counts	-29.6 percent change Standard Deviation 30.3

SECONDARY outcome

Timeframe: 7 days

Population: Time frame was originally entered as 30 days. This was not consistent with the protocol which listed a 7 day time frame for this Outcome

Change in surface area of one or two nonhealing erosions

Outcome measures

Outcome measures
Measure	Granulocyte Colony Stimulating Factor (GCSF) n=7 Participants GCSF 10mcg/kg/d subcutaneously for 7 days Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d subcutaneously for 7 days
Surface Area of Nonhealing Erosions	-35.3 percentage change Standard Deviation 100.8

SECONDARY outcome

Timeframe: 28 days

Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent. This would include decrease in the number and size of blister and erosions, decreased pain, improved comfort of the patient.

Outcome measures

Outcome measures
Measure	Granulocyte Colony Stimulating Factor (GCSF) n=7 Participants GCSF 10mcg/kg/d subcutaneously for 7 days Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d subcutaneously for 7 days
Overall Improved Symptomatology	6 Participants

Adverse Events

Granulocyte Colony Stimulating Factor (GCSF)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Haydar Frangoul, MD, MS

Vanderbilt University

Phone: 615-342-7339

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place