A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

NCT01528306 | Withdrawn Phase 2

• 1 other identifier: 802-247-09-026
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Dystrophic Epidermolysis Bullosa

Keywords

HP802-247; EB (epidermolysis bullosa)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Wound pain as measured by faces pain scale

  4 weeks for each of 3 treatment periods

• Percent of change in wound area

  4 weeks for each of 3 treatment periods

• Persistence of healing

  4 weeks for each of 3 treatment periods

• Burning and stinging upon application

  4 weeks for each of 3 treatment periods

Study Arms (2)

HP802-247

EXPERIMENTAL

Biological: HP802-247

Placebo (Vehicle)

PLACEBO COMPARATOR

Other: Placebo

Interventions

HP802-247 BIOLOGICAL

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

HP802-247

Placebo OTHER

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

Placebo (Vehicle)

Eligibility Criteria

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

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Healthpoint: lead

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa

Condition Hierarchy (Ancestors)

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

Study Officials

• Herbert B Slade, MD

  Healthpoint

  STUDY CHAIR

• Jaime E Dickerson, PhD

  Healthpoint

  STUDY DIRECTOR

• Herbert B Slade, MD

  Healthpoint

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2012
• First Posted: February 8, 2012
• Study Start: March 1, 2012
• Primary Completion: March 1, 2013
• Study Completion: July 1, 2013
• Last Updated: May 16, 2012

Record last verified: 2012-05