Clinical trial NCT01528306: A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa
Official title: An Exploratory, Cross-Over Study of the Safety of HP802-247 Applied to Open Wounds of Subjects With Dystrophic Epidermolysis Bullosa
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Dystrophic Epidermolysis Bullosa
Keywords: HP802-247
EB (epidermolysis bullosa)
Lead sponsor: Healthpoint
Sponsor class: INDUSTRY
First posted: 2012-02-08
Enrollment: 0
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility 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.

Primary outcomes:
measure: Time to healing
timeFrame: 4 weeks
Secondary outcomes:
measure: Wound pain as measured by faces pain scale
timeFrame: 4 weeks for each of 3 treatment periods
measure: Percent of change in wound area
timeFrame: 4 weeks for each of 3 treatment periods
measure: Persistence of healing
timeFrame: 4 weeks for each of 3 treatment periods
measure: Burning and stinging upon application
timeFrame: 4 weeks for each of 3 treatment periods
Other outcomes:
Locations:
Study officials:
affiliation: Healthpoint
name: Herbert B Slade, MD
role: STUDY_CHAIR
affiliation: Healthpoint
name: Jaime E Dickerson, PhD
role: STUDY_DIRECTOR
affiliation: Healthpoint
name: Herbert B Slade, MD
role: PRINCIPAL_INVESTIGATOR
References: