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

NCT00587223 | Terminated Phase 3 Results DMC

• 1 other identifier: 06-EB-001-AG
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Epidermolysis Bullosa, Junctional; Epidermolysis Bullosa Dystrophica

Outcome Measures

Primary Outcomes (1)

• Proportion of Wounds First Achieving 100% Epithelialization of Tissue With the Absence of Drainage (i.e. Complete Wound Closure) Through Study Week 12

  Through 12 weeks

Secondary Outcomes (5)

• Time Until Complete Closure

  through 12 weeks

• Rate of Complete Wound Closure Over Time

  through 12 weeks

• Recurrence of Epidermolysis Bullosa (EB) Lesions

  through 12 months

• Reduction of Intensity of Pain

  through 12 weeks

• Proportion of Wounds Experiencing an Adverse Event

  through 12 months

Study Arms (2)

1

EXPERIMENTAL

Apligraf (a living bilayered cell therapy product)

Device: Apligraf

2

ACTIVE COMPARATOR

Dressing regimen comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer.

Other: Standard dressing regimen

Interventions

Apligraf DEVICE

Up to 3 applications: Day 0, Month 1, Month 2.

1

Standard dressing regimen OTHER

Dressing regimen will be comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer

2

Eligibility Criteria

• Age: 2 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Organogenesis: lead

Study Sites (5)

Stanford University School of Medicine

Stanford, California, 94305-5168, United States

Location

University of Miami - Miller School of Medicine

Miami, Florida, 33125, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Children's Hospital of Cincinnati

Cincinnati, Ohio, 45229, United States

Location

University of Texas

Houston, Texas, 77030-1341, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa, Junctional; Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Collagen Diseases; Connective Tissue Diseases

Limitations and Caveats

Study terminated early, the 1 enrolled subject subsequently discontinued study at Day 7.

Results Point of Contact

• Title: Director of Clinical Operations
• Organization: Organogenesis Inc.

kgiovino@organo.com

Study Officials

• Elizabeth Alvarez- Connelly, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

• Damien Bates, MD, PhD, FRACS (Plast.)

  Organogenesis Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: December 21, 2007
• First Posted: January 7, 2008
• Study Start: December 1, 2007
• Primary Completion: December 1, 2008
• Study Completion: December 1, 2008
• Last Updated: June 29, 2010
• Results First Posted: June 22, 2010

Record last verified: 2010-06

Locations

US (5)