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A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

Primary Purpose

Dystrophic Epidermolysis Bullosa

Status

Withdrawn

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

HP802-247

Placebo

About this trial

This is an interventional treatment trial for Dystrophic Epidermolysis Bullosa focused on measuring HP802-247, EB (epidermolysis bullosa)

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

HP802-247

Placebo (Vehicle)

Arm Description

Outcomes

Primary Outcome Measures

Time to healing

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

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

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

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

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).

Full Information

NCT ID

NCT01528306

First Posted

January 31, 2012

Last Updated

May 15, 2012

Sponsor

Healthpoint

1. Study Identification

Unique Protocol Identification Number

NCT01528306

Brief Title

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

2. Study Status

Record Verification Date

May 2012

Overall Recruitment Status

Withdrawn

Study Start Date

March 2012 (undefined)

Primary Completion Date

March 2013 (Anticipated)

Study Completion Date

July 2013 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Healthpoint

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dystrophic Epidermolysis Bullosa

Keywords

HP802-247, EB (epidermolysis bullosa)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

HP802-247

Arm Type

Experimental

Arm Title

Placebo (Vehicle)

Arm Type

Placebo Comparator

Intervention Type

Biological

Intervention Name(s)

HP802-247

Intervention Description

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

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

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

Primary Outcome Measure Information:

Title

Time to healing

Description

Time to healing, defined as the number of days between wound onset (caregiver reported) and complete epithelialization (Investigator assessed).

Time Frame

4 weeks

Secondary Outcome Measure Information:

Title

Wound pain as measured by faces pain scale

Description

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)

Time Frame

4 weeks for each of 3 treatment periods

Title

Percent of change in wound area

Description

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.

Time Frame

4 weeks for each of 3 treatment periods

Title

Persistence of healing

Description

Persistence of healing as assessed by lack of blister/wound recurrence for healed wounds of each period (up to 40 weeks following enrollment)

Time Frame

4 weeks for each of 3 treatment periods

Title

Burning and stinging upon application

Description

Time Frame

4 weeks for each of 3 treatment periods

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Herbert B Slade, MD

Organizational Affiliation

Healthpoint

Official's Role

Study Chair

First Name & Middle Initial & Last Name & Degree

Jaime E Dickerson, PhD

Organizational Affiliation

Healthpoint

Official's Role

Study Director

First Name & Middle Initial & Last Name & Degree

Herbert B Slade, MD

Organizational Affiliation

Healthpoint

Official's Role

Principal Investigator

12. IPD Sharing Statement

Learn more about this trial

A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

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