A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

An Exploratory, Cross-Over Study of the Safety of HP802-247 Applied to Open Wounds of Subjects With Dystrophic Epidermolysis Bullosa

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.

Study Overview

• Status: Withdrawn
• Conditions: Dystrophic Epidermolysis Bullosa
• Intervention / Treatment: Biological: HP802-247; Other: Placebo

• Study Type: Interventional
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to healing	Time to healing, defined as the number of days between wound onset (caregiver reported) and complete epithelialization (Investigator assessed).	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Wound pain as measured by faces pain scale	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)	4 weeks for each of 3 treatment periods
Percent of change in wound area	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.	4 weeks for each of 3 treatment periods
Persistence of healing	Persistence of healing as assessed by lack of blister/wound recurrence for healed wounds of each period (up to 40 weeks following enrollment)	4 weeks for each of 3 treatment periods
Burning and stinging upon application	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).	4 weeks for each of 3 treatment periods

Collaborators and Investigators

• Sponsor: Healthpoint
• Investigators: Study Director: Jaime E Dickerson, PhD, Healthpoint; Principal Investigator: Herbert B Slade, MD, Healthpoint

Study record dates

Study Major Dates

• Study Start: March 1, 2012
• Primary Completion (Anticipated): March 1, 2013
• Study Completion (Anticipated): July 1, 2013

Study Registration Dates

• First Submitted: January 31, 2012
• First Submitted That Met QC Criteria: February 3, 2012
• First Posted (Estimate): February 8, 2012

Study Record Updates

• Last Update Posted (Estimate): May 16, 2012
• Last Update Submitted That Met QC Criteria: May 15, 2012
• Last Verified: May 1, 2012

More Information

Terms related to this study

• Keywords: HP802-247; EB (epidermolysis bullosa)
• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Collagen Diseases; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica
• Other Study ID Numbers: 802-247-09-026