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ESSENCE Study: Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02384460
First Posted: March 10, 2015
Last Update Posted: August 4, 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.
Collaborator:
Amicus Therapeutics, Inc.
Information provided by (Responsible Party):
Scioderm, Inc.
February 13, 2015
March 10, 2015
August 4, 2017
March 2015
July 2017   (Final data collection date for primary outcome measure)
  • Time to complete target wound closure [ Time Frame: Within 3 months ]
  • The proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 3 months ]
Complete closure of the target wound [ Time Frame: Within 2 months ]
Complete target wound closure is defined as skin re-epithelialization without drainage. The primary efficacy analysis will compare the proportion of patients achieving this endpoint within 2 months.
Complete list of historical versions of study NCT02384460 on ClinicalTrials.gov Archive Site
  • Proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 2 months ]
  • Proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 1 month ]
  • Change in lesional skin [ Time Frame: Month 3 ]
    Based on BSAI at month 3, compared to baseline
  • Change of Total Body Wound Burden based on BSAI [ Time Frame: Month 3 ]
    Assessed at month 3; compared to baseline
  • Change in itching [ Time Frame: Day 7 ]
    Assessed at Day 7, compared to baseline
  • Change in pain [ Time Frame: Day 7 ]
    Assessed at Day 7, compared to baseline
  • Median time to complete target wound closure [ Time Frame: Within 3 months ]
  • Change in lesional skin [ Time Frame: Month 3 ]
    Based on BSA estimates at month 3, compared to baseline
  • Change in itching using Itch Man Pruritus Assessment Tool [ Time Frame: Day 7 ]
    Assessed at Day 7 using Itch Man Pruritus Assessment Tool, compared to baseline
  • Change in pain using "FLACC scale" for patients up to 3 years of age and using "Wrong Faces Pain Scale" for patients 4 years of age and older [ Time Frame: Day 7 ]
    Assessed at Day 7, compared to baseline using the "FLACC scale" for patients up to 3 years of age and for patients 4 years of age and older the "Wong Faces Pain Scale" will be utilized.
  • Change of Total Body Wound Burden based on BSAI [ Time Frame: 2 weeks; months 1 and 2 ]
    Assessed at Week 2 and Months 1 and 2; compared to baseline
  • Percent change from baseline of Total Body Wound Burden based on BSAI [ Time Frame: 2 weeks; Months 1, 2, and 3 ]
    Assessed at week 2 and Months 1, 2, and 3
  • Change in lesional skin [ Time Frame: week 2; months 1 and 2 ]
    based on BSAI at Week 2 and Months 1 and 2, compared to baseline
  • Percent change in lesional skin based on BSAI [ Time Frame: 2 weeks; Months 1, 2, and 3 ]
    Assessed at week 2 and Months 1, 2, and 3 compared to baseline
  • Presence of scarring of healed target wound [ Time Frame: At visit where target wound has healed ]
  • Change in target wound characteristics (i.e. Inflammation, blistering, granulation tissue, erythema, exudate) at week 2, Months 1, 2, and 3, compared to baseline [ Time Frame: 2 weeks; months 1, 2, and 3 ]
  • Change in itching and pain [ Time Frame: Days 1 to 6; week 2; months 1, 2 and 3 ]
    Assessed at Days 1 to 6, Week 2 and Months 1, 2 and 3; compared to baseline
  • Proportion of patients experiencing target wound closure within week 2 [ Time Frame: Week 2 ]
  • Estimation of Total Body Wound Burden [ Time Frame: 2 weeks; months 1, 2 and 3 ]
    Assessed at 2 weeks and Months 1, 2 and 3; compared to baseline
  • Change in lesional skin [ Time Frame: week 2; months 1 and 2 ]
    based on BSA estimates, compared to baseline
  • Presence of scarring of healed target wound [ Time Frame: once target wound has healed assessed at day 7 ]
  • Change in itching and pain [ Time Frame: Days 1 to 6; week 2; months 1, 2 and 3 ]
    Assessed at Days 1 to 6, Week 2 and Months 1, 2 and 3; compared to baseline
  • Proportion of patients experiencing target wound closure [ Time Frame: Week 2; Months 1 and 3 ]
 
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa
A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa
The aim is to assess the efficacy and safety of SD-101-6.0 cream versus SD-101-0.0 (placebo) in the treatment of patients with Epidermolysis Bullosa. Funding Source - FDA OOPD

Epidermolysis Bullosa (EB) is a rare group of inherited disorders that typically manifest at birth as blistering and lesion formation on the skin and, in some cases, the epithelial lining of other organs, in response to little or no apparent trauma. In consequence, the skin is extremely fragile which can result in shearing of the skin, causing a high risk of infection. All forms of EB are both debilitating and life threatening. In some EB subtypes, high mortality occurs before the age of 1 (Junctional Herlitz), and others in adolescence to early adulthood, typically due to infection or failure to thrive. In addition, children surviving into their 20's and 30's are also at risk for development of a virulent form of squamous cell carcinoma, which is in many cases fatal.

There are no standard of care products available to treat the dermal manifestations of EB, and there is no approved drug for EB in either Europe or the United States. There have been numerous studies published on potential treatments for skin manifestations associated with EB, including vitamin E therapy, systemic phenytoin, topical nonsteroidal agents, cyproheptadine, tetracycline, and dapsone. No controlled studies showed clinical benefit of any therapy. Newer exploratory treatments including skin grafts, bioengineered skin products, and gene therapy have been unsuccessful to date.

This is a Phase 3, multi-center, randomized, double-blind, placebo-controlled, study to assess the efficacy and safety of SD-101-6.0 cream vs. placebo (SD-101-0.0) on lesions in patients with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa.

SD-101-6.0 cream or placebo (SD-101-0.0) will be applied topically, once a day to the entire body for a period of 90 days. Patients will have 1 target wound selected at baseline by the investigator. Selected target wound must be of at least a certain age. Photographic confirmation of the target wound location will be collected at baseline, and the picture saved from the first visit will be used to confirm location of the target wound at subsequent visits.

The patient will return to the study site for Visit 2 (14 days ±5 days from baseline), Visit 3 (30 days ±7 days from baseline), Visit 4 (60 days ±7 days from baseline), and Visit 5 (90 days ±7 days from baseline) to have the target wound, previously identified at baseline, re-assessed for the level of healing. In addition, itching, pain, body surface area (BSA), target wound closure, and scarring of healed target wound will also be assessed at each visit. The ARANZ SilhouetteStar™ will be used to measure the target wound at all visits.

Interventional
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Epidermolysis Bullosa
  • Drug: SD-101-6.0 cream
    applied topically once a day for 90 days
    Other Name: SD-101
  • Drug: SD-101-0.0 cream
    applied topically once a day for 90 days
    Other Name: placebo
  • Experimental: SD-101-6.0 cream
    SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
    Intervention: Drug: SD-101-6.0 cream
  • Placebo Comparator: SD-101-0.0 cream
    SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
    Intervention: Drug: SD-101-0.0 cream
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
150
July 2017
July 2017   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Informed Consent form signed by the patient or patient's legal representative; if the patient is under the age of 18 but capable of providing assent, signed assent from the patient.
  • Patient (or caretaker) must be willing to comply with all protocol requirements.
  • Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
  • Patient must have 1 target wound within a prespecified size range at study entry
  • Patients 1 month and older.
  • Target wound must of at least a certain age

Exclusion Criteria:

  • Patients who do not meet the entry criteria outlined above.
  • Selected target wound cannot have clinical evidence of local infection.
  • Use of any investigational drug within the 30 days before enrollment.
  • Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.
  • Use of systemic or topical steroidal therapy within the 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids are allowed)
  • Use of systemic antibiotics within the 7 days before enrollment.
  • Current or former malignancy.
  • Arterial or venous disorder resulting in ulcerated lesions.
  • Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed at screening and every 30 days until the final visit for female patients of childbearing potential)
  • Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
Sexes Eligible for Study: All
1 Month and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Austria,   France,   Germany,   Israel,   Italy,   Lithuania,   Netherlands,   Poland,   Serbia,   Spain,   United Kingdom,   United States
 
 
NCT02384460
SD-005
2014-002288-14 ( EudraCT Number )
R01-005095-01 ( Other Identifier: Orphan Product Grant )
No
Not Provided
Not Provided
Scioderm, Inc.
Scioderm, Inc.
Amicus Therapeutics, Inc.
Not Provided
Scioderm, Inc.
August 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP