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

Study Description

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Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Epidermolysis Bullosa	Drug: SD-101-6.0 cream; Drug: SD-101-0.0 cream	Phase 3

Detailed Description:

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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	150 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:	Treatment
Official Title:	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
Study Start Date :	March 2015
Actual Primary Completion Date :	July 2017
Actual Study Completion Date :	July 2017

Arms and Interventions

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Arm	Intervention/treatment
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	Drug: SD-101-6.0 cream; applied topically once a day for 90 days; Other Name: SD-101
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	Drug: SD-101-0.0 cream; applied topically once a day for 90 days; Other Name: placebo

Outcome Measures

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Primary Outcome Measures :

1. Time to complete target wound closure [ Time Frame: Within 3 months ]
2. The proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 3 months ]

Secondary Outcome Measures :

1. Proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 2 months ]
2. Proportion of patients experiencing complete closure of their target wound. [ Time Frame: Within 1 month ]
3. Change in lesional skin [ Time Frame: Month 3 ]
   Based on BSAI at month 3, compared to baseline
4. Change of Total Body Wound Burden based on BSAI [ Time Frame: Month 3 ]
   Assessed at month 3; compared to baseline
5. Change in itching [ Time Frame: Day 7 ]
   Assessed at Day 7, compared to baseline
6. Change in pain [ Time Frame: Day 7 ]
   Assessed at Day 7, compared to baseline

Other Outcome Measures:

1. 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
2. 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
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
4. 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
5. Presence of scarring of healed target wound [ Time Frame: At visit where target wound has healed ]
6. 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 ]
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
8. Proportion of patients experiencing target wound closure within week 2 [ Time Frame: Week 2 ]

Eligibility Criteria

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Ages Eligible for Study:	1 Month and older (Child, Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

  Show 38 Study Locations

Sponsors and Collaborators

Scioderm, Inc.

Amicus Therapeutics, Inc.

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Scioderm, Inc.
ClinicalTrials.gov Identifier:	NCT02384460 History of Changes
Other Study ID Numbers:	SD-005; 2014-002288-14 ( EudraCT Number ); R01-005095-01 ( Other Identifier: Orphan Product Grant )
First Posted:	March 10, 2015
Last Update Posted:	August 4, 2017
Last Verified:	August 2017

Keywords provided by Scioderm, Inc.:

Simplex; Recessive Dystrophic; Junctional non-Herlitz; Epidermolysis Bullosa

Additional relevant MeSH terms:

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Skin Diseases, Genetic	Genetic Diseases, Inborn; Skin Diseases; Skin Diseases, Vesiculobullous