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Safety and Efficacy of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa (EB)

This study has been terminated.
(Insufficient patient enrollment)
Sponsor:
Information provided by:
Organogenesis
ClinicalTrials.gov Identifier:
NCT00587223
First received: December 21, 2007
Last updated: June 25, 2010
Last verified: June 2010
  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 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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:


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:
  • Time Until Complete Closure [ Time Frame: through 12 weeks ] [ Designated as safety issue: No ]
  • Rate of Complete Wound Closure Over Time [ Time Frame: through 12 weeks ] [ Designated as safety issue: No ]
  • Recurrence of Epidermolysis Bullosa (EB) Lesions [ Time Frame: through 12 months ] [ Designated as safety issue: No ]
  • Reduction of Intensity of Pain [ Time Frame: through 12 weeks ] [ Designated as safety issue: No ]
  • 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
Experimental: 1
Apligraf (a living bilayered cell therapy product)
Device: Apligraf
Up to 3 applications: Day 0, Month 1, Month 2.
Active Comparator: 2
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
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: 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
Study Director: Damien Bates, MD, PhD, FRACS (Plast.) Organogenesis Inc.
  More Information

No publications provided

Responsible Party: Katherine B. Giovino, Director of Clinical Operations, Organogenesis Inc.
ClinicalTrials.gov Identifier: NCT00587223     History of Changes
Other Study ID Numbers: 06-EB-001-AG
Study First Received: December 21, 2007
Results First Received: March 29, 2010
Last Updated: June 25, 2010
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa, Junctional
Collagen Diseases
Congenital Abnormalities
Connective Tissue Diseases
Genetic Diseases, Inborn
Skin Abnormalities
Skin Diseases
Skin Diseases, Genetic
Skin Diseases, Vesiculobullous

ClinicalTrials.gov processed this record on November 20, 2014