Study to Evaluate the Use of Palifermin to Treat Toxic Epidermal Necrolysis

• Sponsor: Brett King (Other)
• Overall Status: Terminated
• CT.gov ID: NCT02037347
• Collaborator: Swedish Orphan Biovitrum (Industry)
• Enrollment: 1
• Locations: 2
• Arm: 1
• Duration (Months): 50
• Patients Per Site: 0.5
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

To test the ability of palifermin (a recombinant human keratinocyte growth factor) to decrease mucocutaneous injury and to promote epithelial repair in Toxic Epidermal Necrolysis and Stevens-Johnson Syndrome-Toxic Epidermal Necrolysis Overlap, diseases in which there is extensive sloughing of the skin and mucosa, including that of the eyes, gastrointestinal tract, respiratory and genitourinary systems.

Condition or Disease	Intervention/Treatment	Phase
Toxic Epidermal Necrolysis; Stevens-Johnson Syndrome	Drug: Palifermin	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 1 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Palifermin Treatment of Toxic Epidermal Necrolysis
• Study Start Date: Oct 1, 2010
• Actual Primary Completion Date: Dec 1, 2014
• Actual Study Completion Date: Dec 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Palifermin Palifermin 60 micrograms/kg/day IV for 3 consecutive days	Drug: Palifermin; Other Names: Kepivance

Outcome Measures

Primary Outcome Measures

1. Time-to-cutaneous Re-epithelialization [The number of days between the start of palifermin administration and complete re-epithelialization of skin up to 14 days]

Secondary Outcome Measures

1. Time-to-mucosal Re-epithelialization [The number of days between the start of palifermin administration and complete re-epithelialization of oral mucosa up to 14 days]

2. Time-to-cessation of Epidermal Necrosis [The number of days between the start of palifermin administration and cessation of further epidermal necrosis up to 14 days]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Skin biopsy in early disease showing apoptotic keratinocytes in the epidermis and in more advanced disease necrosis of the entire epidermis

• Epidermal detachment or erythematous to purpuric macules involving more than 10% of body surface area in addition to involvement of the oropharynx; there may be further involvement of other mucosal surfaces, ie. conjunctivae, gastrointestinal tract, respiratory and genitourinary epithelia

• Age of 18 years old or older

• The patient is expected to survive longer than 48 hours

Exclusion Criteria:

• Skin detachment above 90% of the body surface area

• Skin detachment has not progressed during the previous 48 hours

• A positive serum pregnancy test

• Age < 18 years old

• Known hematologic or solid organ malignancy

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Bridgeport Hospital	Bridgeport	Connecticut	United States	06610
2	Yale-New Haven Hospital	New Haven	Connecticut	United States	06510

Sponsors and Collaborators

• Brett King
• Swedish Orphan Biovitrum

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Brett King, Assistant Professor of Dermatology, Yale University
• ClinicalTrials.gov Identifier: NCT02037347
• Other Study ID Numbers: 0901004673; 020901
• First Posted: Jan 15, 2014
• Last Update Posted: Nov 30, 2016
• Last Verified: Oct 1, 2016

• Additional relevant MeSH terms: Stevens-Johnson Syndrome

Study Results

Participant Flow

Arm/Group Title	Palifermin
Arm/Group Description	Palifermin 60 micrograms/kg/day IV for 3 consecutive days
Period Title: Overall Study
STARTED	1
COMPLETED	1
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Palifermin
Arm/Group Description	Palifermin 60 micrograms/kg/day IV for 3 consecutive days
Overall Participants	1
Age, Customized (years) [Number]
Age	73
Sex/Gender, Customized (participants) [Number]
Male	1 100%

Outcome Measures

1. Primary Outcome

Title	Time-to-cutaneous Re-epithelialization
Time Frame	The number of days between the start of palifermin administration and complete re-epithelialization of skin up to 14 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Palifermin
Arm/Group Description	Palifermin 60 micrograms/kg/day IV for 3 consecutive days
Measure Participants	1
Number [days]	6

2. Secondary Outcome

Title	Time-to-mucosal Re-epithelialization
Time Frame	The number of days between the start of palifermin administration and complete re-epithelialization of oral mucosa up to 14 days

Outcome Measure Data

Analysis Population Description

Adverse event experienced by participant precluded measuring this outcome.

Arm/Group Title	Palifermin
Arm/Group Description	Palifermin 60 micrograms/kg/day IV for 3 consecutive days
Measure Participants	0

3. Secondary Outcome

Title	Time-to-cessation of Epidermal Necrosis
Time Frame	The number of days between the start of palifermin administration and cessation of further epidermal necrosis up to 14 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Palifermin
Arm/Group Description	Palifermin 60 micrograms/kg/day IV for 3 consecutive days
Measure Participants	1
Number [days]	3

Adverse Events

Arm/Group Title	Palifermin
Arm/Group Description	Palifermin 60 micrograms/kg/day IV for 3 consecutive days
All Cause Mortality
	Palifermin
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Palifermin
	Affected / at Risk (%)	# Events
Total	1/1 (100%)
Gastrointestinal disorders
ischemic necrosis	1/1 (100%)
Other (Not Including Serious) Adverse Events
	Palifermin
	Affected / at Risk (%)	# Events
Total	0/1 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.