Study of Effectiveness and Safety of SD-101 in Subjects With Epidermolysis Bullosa

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Scioderm, Inc.
ClinicalTrials.gov Identifier:
NCT02014376
First received: December 12, 2013
Last updated: June 26, 2014
Last verified: June 2014
  Purpose

The purpose of this study is to assess whether SD-101 cream (3% or 6%) is effective in treating the lesions in subjects with Simplex, Recessive Dystrophic, or Junctional Epidermolysis Bullosa.


Condition Intervention Phase
Epidermolysis Bullosa
Drug: 3% SD-101 dermal cream
Drug: 6% SD-101 dermal cream
Drug: Vehicle (0% SD-101)
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2B, Multi-center, Randomized, Double-Blind, Vehicle Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa

Resource links provided by NLM:


Further study details as provided by Scioderm, Inc.:

Primary Outcome Measures:
  • Complete Target Wound Closure [ Time Frame: within 1 month ] [ Designated as safety issue: No ]
    The primary endpoint is target wound closure within 1 month of treatment.


Secondary Outcome Measures:
  • Change in Body Surface Area (BSA) coverage of blisters or lesions [ Time Frame: month 3 ] [ Designated as safety issue: No ]
    Change in blisters or lesions based on body surface area (BSA) measurements at month 3, compared to baseline.

  • Change in itching [ Time Frame: 1 week ] [ Designated as safety issue: No ]
    Change in itching assessed at 1 week, compared to baseline.

  • Change in pain [ Time Frame: 1 week ] [ Designated as safety issue: No ]
    Change in pain assessed at 1 week, compared to baseline.


Estimated Enrollment: 48
Study Start Date: January 2014
Study Completion Date: June 2014
Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SD-101 dermal cream (6%)
6% SD-101 dermal cream applied topically once daily for 90 days
Drug: 6% SD-101 dermal cream
applied topically once a day for 90 days
Other Name: SD-101
Experimental: SD-101 dermal cream (3%)
3% SD-101 dermal cream applied topically once daily for 90 days
Drug: 3% SD-101 dermal cream
applied topically once a day for 90 days
Other Name: SD-101
Placebo Comparator: Vehicle
Vehicle dermal cream (0% SD-101) applied topically once daily for 90 days
Drug: Vehicle (0% SD-101)
applied topically once a day for 90 days
Other Name: Placebo Comparator

Detailed Description:

Epidermolysis Bullosa (EB) is a rare group of inherited disorders that manifest as blistering or erosion of the skin and in some cases, the epithelial liming of other organs, in response to little or no apparent trauma. There is a lack of effective agents for skin disorders involving blistering and lesion formation. Current approved therapies are minimally effective and have safety issues. Scioderm has developed SD-101 Dermal Cream [0% (vehicle), 3% and 6%] for the treatment of lesions associated with EB.

The purpose of this study is to assess the safety and efficacy of SD-101 cream (3% or 6%) vs. vehicle in the treatment of lesions in 48 subjects with Simplex, Recessive Dystrophic, or Junctional Epidermolysis Bullosa.

SD-101 cream [containing 3% or 6% or vehicle (0%)] will be applied topically, once a day to the entire body for a period of 90 days. Subjects will come into the study site to have a target wound assessed at baseline. Selected target wound must be at least a certain age and within a prespecified size range at study entry. 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 subject will return to the study site for visits 2,3,4 and 5 (at 14, 30, 60 and 90 days respectively) to have the target wound previously identified at baseline, re-assessed for the level of healing. In addition, changes in itching, pain, Body Surface Area (BSA) coverage of blisters and lesions, and scaring of the healed target wound will also be assessed at each visit.

  Eligibility

Ages Eligible for Study:   6 Months and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Informed Consent form signed by the subject or the subject's legal representative; if the subject is under the age of 18 but capable of providing assent, signed assent from the subject.
  • Subject (or caretaker) must be willing to comply with all protocol requirements.
  • Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
  • Subjects must be 6 months of age and older.
  • Subjects must have 1 target wound within a prespecified size range at study entry
  • Target wound must be of at least a certain age

Exclusion Criteria:

  • Subjects who do not meet the entry criteria outlined in inclusion criteria.
  • 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.
  • Diabetes mellitus.
  • Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed ar screening for female subjects of childbearing potential)
  • Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
  • Known history of cardiac, hepatic, or renal disease.
  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: NCT02014376

Locations
United States, California
Stanford University School of Medicine
Palo Alto, California, United States, 94304
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Missouri
Washington University School of Medicine
St. Louis, Missouri, United States, 63110
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
United States, North Carolina
University of North Carolina School of Medicine
Chapel Hill, North Carolina, United States, 27516
United States, Texas
Texas Dermatology and Laser Specialists
San Antonio, Texas, United States, 78218
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Scioderm, Inc.
  More Information

No publications provided

Responsible Party: Scioderm, Inc.
ClinicalTrials.gov Identifier: NCT02014376     History of Changes
Other Study ID Numbers: SD-003
Study First Received: December 12, 2013
Last Updated: June 26, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Scioderm, Inc.:
Epidermolysis Bullosa
Simplex
Recessive Dystrophic
Junctional non-Herlitz

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 September 22, 2014