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

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Scioderm, Inc.
Sponsor:
Collaborator:
Amicus Therapeutics, Inc.
Information provided by (Responsible Party):
Scioderm, Inc.
ClinicalTrials.gov Identifier:
NCT02384460
First received: February 13, 2015
Last updated: February 7, 2017
Last verified: February 2017
  Purpose
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.

Condition Intervention Phase
Epidermolysis Bullosa
Drug: SD-101-6.0 cream
Drug: SD-101-0.0 cream
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Scioderm, Inc.:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • Median time to complete target wound closure [ Time Frame: Within 3 months ]
  • Change in lesional skin [ Time Frame: Month 2 ]
    Based on BSA estimates at month 2, 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.


Other Outcome Measures:
  • 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 3 ]
    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 ]

Estimated Enrollment: 130
Study Start Date: March 2015
Estimated Study Completion Date: September 2017
Estimated Primary Completion Date: June 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02384460

  Show 35 Study Locations
Sponsors and Collaborators
Scioderm, Inc.
Amicus Therapeutics, Inc.
  More Information

Responsible Party: Scioderm, Inc.
ClinicalTrials.gov Identifier: NCT02384460     History of Changes
Other Study ID Numbers: SD-005
2014-002288-14 ( EudraCT Number )
Study First Received: February 13, 2015
Last Updated: February 7, 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

ClinicalTrials.gov processed this record on March 29, 2017