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Trial record 10 of 38 for:    "epidermolysis bullosa simplex" OR "Epidermolysis Bullosa"

A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

This study has been withdrawn prior to enrollment.
Sponsor:
Information provided by (Responsible Party):
Healthpoint
ClinicalTrials.gov Identifier:
NCT01528306
First received: January 31, 2012
Last updated: May 15, 2012
Last verified: May 2012
  Purpose

This is a pilot study designed to see if HP802-247, an investigational treatment with living human skin cells, helps to heal blisters or wounds in subjects with Dystrophic Epidermolysis Bullosa (DEB). The durability of the skin in healed wounds treated with HP802-247 will also be assessed.


Condition Intervention Phase
Dystrophic Epidermolysis Bullosa
Biological: HP802-247
Other: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: An Exploratory, Cross-Over Study of the Safety of HP802-247 Applied to Open Wounds of Subjects With Dystrophic Epidermolysis Bullosa

Resource links provided by NLM:


Further study details as provided by Healthpoint:

Primary Outcome Measures:
  • Time to healing [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
    Time to healing, defined as the number of days between wound onset (caregiver reported) and complete epithelialization (Investigator assessed).


Secondary Outcome Measures:
  • Wound pain as measured by faces pain scale [ Time Frame: 4 weeks for each of 3 treatment periods ] [ Designated as safety issue: No ]
    Change from baseline (i.e., Week 1 of each period) in wound pain for each visit (Weeks 2-5 of each treatment period)as assessed by the Faces Pain Scale (FPS)

  • Percent of change in wound area [ Time Frame: 4 weeks for each of 3 treatment periods ] [ Designated as safety issue: No ]
    Percent of change from baseline (i.e., Week 1 of each period) in the target wound area for each visit and Weeks 2-5 of each treatment period.

  • Persistence of healing [ Time Frame: 4 weeks for each of 3 treatment periods ] [ Designated as safety issue: No ]
    Persistence of healing as assessed by lack of blister/wound recurrence for healed wounds of each period (up to 40 weeks following enrollment)

  • Burning and stinging upon application [ Time Frame: 4 weeks for each of 3 treatment periods ] [ Designated as safety issue: No ]

    Burning and stinging upon application of HP802-247 or its Vehicle will be assessed at each visit of each period with the exception of the end of treatment visit (Weeks 1-4 of treatment period).

    Burning/stinging scale is a four point scale (i.e., none, mild, moderate, severe).



Enrollment: 0
Study Start Date: March 2012
Estimated Study Completion Date: July 2013
Estimated Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HP802-247 Biological: HP802-247

Wounds up to 12 cm2: 1 spray each component (0.5 x 106 cells per mL allogeneic human keratinocytes and fibroblasts) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods

Larger wounds:

(>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each

Placebo Comparator: Placebo (Vehicle) Other: Placebo

Wounds up to 12 cm2: 1 spray each component (thrombin solution and fibrinogen solution) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods

Larger wounds:

(>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each


  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written consent must be obtained from a parent or legally authorized representative. Assent will be obtained according to local requirements.
  • Subjects with a clinical diagnosis of DEB with recurring blisters, ≥ 2 years of age at Screening. Subjects may be of either sex and of any race or skin type provided that their skin color, in the opinion of the Investigator, will not interfere with the study assessments.
  • A parent or legally authorized representative must be willing and able to ensure subject is present for all required study visits.
  • A parent or legally authorized representative must be able to follow instructions.
  • Have an open wound for ≤ 72 hours that:

    • is still open and has not scabbed or crusted over
    • has no dermatologic disease and/or condition in the treatment area (other than DEB with recurring blisters), including active or recent infection (within 7 days) that may be exacerbated by treatment, require treatment with antibiotics / antifungals /antivirals, require a surgical intervention, or cause difficulty with examination
    • is ≥ 4 cm² and ≤ 48 cm2 in total area (open portion)
  • Females of childbearing potential (defined as post-menarcheal as documented in the medical history) may participate in the study if they meet all of the following conditions:

    • they are not breast feeding;
    • they have a negative urine pregnancy test at Week 1 Period 1;
    • they agree to undertake scheduled urine pregnancy tests at Week 1 for Periods 1, 2, & 3 and at study exit
    • they do not intend to become pregnant during the study;
    • they are using adequate birth control methods and they agree to continue using those methods for the duration of the study
  • A parent or legally authorized representative must be willing and able to ensure subject is present for all required study visits (for minor subjects)
  • A parent or legally authorized representative must be able to follow instructions (for minor subjects)

Exclusion Criteria:

  • Contraindications or hypersensitivity to the use of the test article, their components (e.g., aprotinin, fibrinogen), or substances used in the manufacture of the test article (e.g., penicillin, streptomycin, amphotericin B, bovine serum albumin).
  • Therapy with another investigational agent within thirty (30) days of the Screening Visit, or during the study.
  • Have uncontrolled intercurrent or chronic illness that, in the opinion of the Investigator, would limit compliance with study requirements, represents a potential safety risk, or require treatment with an excluded drug/treatment.
  • The Investigator or Medical Monitor may declare any subject ineligible for a valid medical reason.
  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: NCT01528306

Sponsors and Collaborators
Healthpoint
Investigators
Study Chair: Herbert B Slade, MD Healthpoint
Study Director: Jaime E Dickerson, PhD Healthpoint
Principal Investigator: Herbert B Slade, MD Healthpoint
  More Information

No publications provided

Responsible Party: Healthpoint
ClinicalTrials.gov Identifier: NCT01528306     History of Changes
Other Study ID Numbers: 802-247-09-026
Study First Received: January 31, 2012
Last Updated: May 15, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Healthpoint:
HP802-247
EB (epidermolysis bullosa)

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
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