Safety and Efficacy of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa - Full Text View

Sponsor:	Organogenesis
Information provided by:	Organogenesis
ClinicalTrials.gov Identifier:	NCT00587223

Purpose

The purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with dystrophic or junctional epidermolysis bullosa. Apligraf will be evaluated for efficacy and safety compared to a conventional nonadherent dressing. A matched-pair design will be used to evaluate Apligraf treatment versus conventional treatment in 68 study pairs.

Condition	Intervention	Phase
Epidermolysis Bullosa, Junctional Epidermolysis Bullosa Dystrophica	Device: Apligraf Other: Standard dressing regimen	Phase III

Study Type:	Interventional
Study Design:	Treatment, Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	A Prospective, Multicenter, Within Subject Controlled Study to Evaluate the Effect of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa

Resource links provided by NLM:

Genetics Home Reference related topics: dystrophic epidermolysis bullosa epidermolysis bullosa simplex epidermolysis bullosa with pyloric atresia junctional epidermolysis bullosa

U.S. FDA Resources

Further study details as provided by Organogenesis:

Primary Outcome Measures:

Proportion of wounds first achieving 100% epithelialization of tissue with the absence of drainage (i.e. complete wound closure) through Study Week 12 [ Time Frame: Through 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

1. Time until complete wound closure 2. Rate of complete wound closure over time 3. Recurrence of EB lesions 4. Reduction of intensity of pain 5. Proportion of wounds experiencing an adverse event [ Time Frame: through 12 months ] [ Designated as safety issue: Yes ]

Enrollment:	1
Study Start Date:	December 2007
Study Completion Date:	December 2008
Primary Completion Date:	December 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental Apligraf (a living bilayered cell therapy product)	Device: Apligraf Up to 3 applications: Day 0, Month 1, Month 2.
2: Active Comparator Dressing regimen comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer.	Other: Standard dressing regimen Dressing regimen will be comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer

Eligibility

Ages Eligible for Study:	2 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Subject is between 2 and 65 years of age.
Subject with clinical and histological diagnosis of junctional or dystrophic EB with documented electron microscopy or immunofluorescence microscopy.
Subject has at least two non-adjacent EB lesions, at least 4 cm apart. The two selected EB lesions must be relatively matched in terms of diagnosis, location, level of erosion and size. Lesions may be on the same limb.
Subject with dystrophic or junctional EB lesions between 10-44 cm2 present for at least 6 weeks. For the purposes of this study, a lesion is defined as a wound resulting from a post blister erosion.
Subject who is a female of child-bearing potential (females >10 years of age) must have a documented negative urine or serum pregnancy test. Sexually active females must be practicing a medically proven form of contraception during the course of the study period.
Subject or legal guardian must have read, understood and signed an institutional review board (IRB) approved Informed Consent Form or Assent Form.
Subject and/or legal guardian must be able and willing to follow study procedures and instructions.

Exclusion Criteria:

Subject with lesions only on the soles, posterior thigh or gluteus maximus.
Subject whose lesion has healed 20% or greater in area from post debridement (if applicable) Baseline Screen (Visit 1) to post debridement Day 0 (Visit 2) as determined by wound tracings.
Subject with uncontrolled diabetes mellitus (glycosylated HbA1C > 10%), cancer (biopsy confirmed active malignancy), or positive HIV test.
Subject is a child (<18 years of age) who is currently receiving or has received oral steroid therapy exceeding a total daily dose of 0.5mg/kg for more than two weeks, radiation or other immuno-suppressive therapy which would interfere with wound healing within the past four weeks.
Subject is an adult (>18 years of age) who is currently receiving or has received chronic high dose steroid therapy exceeding a total daily dose of 20mg for more than two weeks, radiation or other immuno-suppressive therapy which would interfere with wound healing within the past four weeks.
Subject who is currently on or has received topical steroidal therapy within 30 days before screening. Inhaled steroids are allowed.
Subject with the presence of acute infections in the areas intended for treatment.
Subject with a history of squamous cell carcinoma.
Known hypersensitivity to bovine collagen or to the components of the Apligraf agarose shipping medium.
Subject who is lactating or pregnant (hCG positive as determined by lab testing).
Subject is a child (<18 years of age) with liver (ALT/SGPT, ALP, AST/SGOT, bilirubin, total protein, LDH) and/or renal function (BUN and creatinine) tests > 2 x upper limit of normal (ULN). Albumin < 2.0 mg/dL.
Subject is an adult (>18 years of age) with liver (ALT/SGPT, ALP, AST/SGOT, bilirubin, total protein, LDH) and/or renal function (BUN and creatinine) tests > 2 x upper limit of normal (ULN). Albumin < 2.0 mg/dL.
Subject enrolled in any wound or investigational device study for any disease within the past four weeks.
Subject who has received an investigational drug or biological treatment within three months.
Subject previously treated with Apligraf, Dermagraft or any other cell therapy at the target sites.
Subject with a history of alcohol or substance abuse within the previous year, which could interfere with study compliance such as inability to attend scheduled study visits or compliance with home dressing changes.
Subject who, in the opinion of the investigator, for any reason other than those listed above, will not be able to complete the study per protocol.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00587223

Locations

United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305-5168
United States, Florida
University of Miami - Miller School of Medicine
Miami, Florida, United States, 33125
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
United States, Ohio
Children's Hospital of Cincinnati
Cincinnati, Ohio, United States, 45229
United States, Texas
University of Texas
Houston, Texas, United States, 77030-1341

Sponsors and Collaborators

Organogenesis

Investigators

Principal Investigator:	Elizabeth Alvarez- Connelly, MD	University of Miami, Miller School of Medicine
Study Director:	Damien Bates, MD, PhD, FRACS (Plast.)	Organogenesis Inc.

More Information

No publications provided

Responsible Party:	Organogenesis Inc. ( Katherine B. Giovino, Director of Clinical Operations )
Study ID Numbers:	06-EB-001-AG
Study First Received:	December 21, 2007
Last Updated:	February 13, 2009
ClinicalTrials.gov Identifier:	NCT00587223 History of Changes
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Collagen Diseases
Skin Diseases, Vesiculobullous
Skin Diseases
Genetic Diseases, Inborn
Epidermolysis Bullosa, Junctional
Connective Tissue Diseases

Skin Abnormalities
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa
Congenital Abnormalities
Skin Diseases, Genetic

ClinicalTrials.gov processed this record on February 08, 2010