Toxic Epidermal NecrolysisPhase 1/2NCT02037347
Study to Evaluate the Use of Palifermin to Treat Toxic Epidermal Necrolysis
A Phase 1/2 study testing Palifermin in people with Toxic Epidermal Necrolysis and Stevens-Johnson Syndrome, run by Brett King at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Toxic Epidermal Necrolysis, Stevens-Johnson Syndrome
- What is being tested?
- Palifermin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 2 sitesUnited States
- Study dates
- Oct 2010to Dec 2014
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Bridgeport Hospital
Bridgeport, Connecticut, 06610
Yale-New Haven Hospital
New Haven, Connecticut, 06510
About this study
Official title: Palifermin Treatment of Toxic Epidermal Necrolysis
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.
- Being tested
- Palifermin
- Medical subject headings
- Stevens-Johnson Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PaliferminDrug
Also called Kepivance
| Study group | Role | What they receive |
|---|---|---|
| Palifermin | Experimental | Palifermin 60 micrograms/kg/day IV for 3 consecutive days Receives: Drug: Palifermin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time-to-cutaneous Re-epithelialization
Measured: The number of days between the start of palifermin administration and complete re-epithelialization of skin up to 14 days
Secondary outcomes (2)
Time-to-mucosal Re-epithelialization
Measured: The number of days between the start of palifermin administration and complete re-epithelialization of oral mucosa up to 14 days
Time-to-cessation of Epidermal Necrosis
Measured: The number of days between the start of palifermin administration and cessation of further epidermal necrosis up to 14 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2014 (estimated)
- Study startOct 2010
- Primary completionDec 2014
- Study completionDec 2014
Why it stopped: Low enrollment numbers precluded completion of the study during a reasonable amount of time.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Toxic Epidermal NecrolysisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brett King
- Sponsor type
- University, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Responsible party
- Sponsor-investigator, Brett King, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02037347
- Sponsor's protocol ID
- 0901004673
- Other IDs
- 020901
- Status last verified
- Oct 2016
- First submitted
- 13 Jan 2014
- Last updated
- 30 Nov 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Palifermin Treatment of Toxic Epidermal Necrolysis
- ClinicalTrials.gov ID
- NCT02037347
- Acronym / sponsor ID
- 0901004673
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low enrollment numbers precluded completion of the study during a reasonable amount of time.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Brett KingUniversity, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Study start
- Oct 2010
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 15 Jan 2014 (estimated)
- Results
- Posted 30 Nov 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Palifermin | Experimental | Drug: PaliferminPalifermin 60 micrograms/kg/day IV for 3 consecutive days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PaliferminAlso: Kepivance | Drug | Palifermin |
Primary outcome
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
2 secondary outcomes
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
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Toxic Epidermal Necrolysis
Not yet recruitingPhase 1/2
New Therapeutic Target for Toxic Epidermal Necrolysis (TEN) Using Anti-CD38+ Monoclonal Antibodies.
Toxic Epidermal Necrolysis, Immunotherapy
RecruitingPhase 1/2
LYell SYndrome MEsenchymal Stromal Cells Treatment
Epidermal Necrolysis, Lyell Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Palifermin in people with Toxic Epidermal Necrolysis and Stevens-Johnson Syndrome, run by Brett King at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Nov 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Nov 2016; AzTrials last refreshed its copy on 8 Nov 2026.