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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.
  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. Funding Source - FDA OOPD

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: 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

Resource links provided by NLM:


Further study details as provided by Scioderm, Inc.:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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


Other Outcome Measures:
  • 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 ]

Estimated Enrollment: 150
Study Start Date: March 2015
Study Completion Date: July 2017
Primary Completion Date: July 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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02384460


  Hide Study Locations
Locations
United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
United States, California
Stanford University-Dept of Dermatology
Redwood City, California, United States, 94063
United States, Colorado
Children's Hospital Coloardo, University of Colorado School of Medicine
Aurora, Colorado, United States, 80045
United States, District of Columbia
MedStar Georgetown University Hospital
Washington, D.C., District of Columbia, United States, 20016
United States, Florida
University of Miami Miller School of Medicine
Miami, Florida, United States, 33136
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago, Department of Dermatology
Chicago, Illinois, United States, 60611
United States, Minnesota
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55455
United States, Missouri
University of Missouri
Columbia, Missouri, United States, 65212
Washington University-Dermatology
Saint Louis, Missouri, United States, 63110
United States, New Hampshire
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States, 03756
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
United States, New York
Stony Brook Medicine Department of Dermatology
East Setauket, New York, United States, 11733
Columbia University Medical Center Department of Dermatology
New York, New York, United States, 10032
United States, North Carolina
UNC Dermatology and Skin Cancer Center
Chapel Hill, North Carolina, United States, 27516
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425-5780
United States, Texas
'Specially for Children
Austin, Texas, United States, 78723
Texas Dermatology and Laser Specialists
San Antonio, Texas, United States, 78218
United States, Washington
Seattle Children's
Seattle, Washington, United States, 98105
Australia, New South Wales
Premier Specialists Pty Ltd.
Kogarah, New South Wales, Australia, 2217
Australia, Victoria
The Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
The Royal Children's Hospital
Parkville, Victoria, Australia, 3052
Austria
EB House Austria, General Hospital Salzburg/Salzburger Landesklinikum (SALK), Paracelsus Medical University Salzburg (PMU)
Salzburg, Austria, 5020
France
Hopital Archet 2 Service de Dermatologie
Nice, France, 0-06200
University of Paris Sorbonne Descartes, IMAGINE Institute, Necker-Enfants Malades Medical School
Paris, France, 75015
Larrey Hospital Dermatology Department
Toulouse, France
Germany
Medical Center- University of Freiburg, Department of Dermatology, Hauptstrasse 7
Freiburg, Germany
Kinder-und-Jugendkrankenhaus
Hannover, Germany
Israel
Tel Aviv Tourasky Medical Center
Tel Aviv, Israel
Italy
Universita degli Studi di Milano, Fondazione IRCCS Ca' Granda Ospedale Maggiore
Milano, Italy, 20122
Lithuania
Hospital of Lithuanian University of Health Sciences Public Institution
Kaunas, Lithuania
Netherlands
University Medical Center Groningen, Hanzeplein 1 Department Dermatology
Groningen, Netherlands, 9713 GZ
Poland
Department of Immunodermatology, Medical University of Warsaw
Warszawa, Koszykowa, Poland
Serbia
Clinical Center of Serbia, Center for Medical Biochemistry
Belgrade, Serbia
Spain
Hospital La Paz
Madrid, Spain
United Kingdom
Clinical Research Facilities (CRF) unit, Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom, WC1N 3JH
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 )
R01-005095-01 ( Other Identifier: Orphan Product Grant )
First Submitted: February 13, 2015
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