Dystrophic Epidermolysis BullosaPhase 2NCT01528306
A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa
A Phase 2 study testing HP802-247 in people with Dystrophic Epidermolysis Bullosa, run by Healthpoint.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- What is being tested?
- HP802-247
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Mar 2012to Jul 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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)
Who cannot join
- 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.
About this study
Official title: 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.
- Conditions
- Dystrophic Epidermolysis Bullosa
- Being tested
- HP802-247, Placebo
- Keywords
- HP802-247, EB (epidermolysis bullosa)
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HP802-247Biological
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
PlaceboOther
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
| Study group | Role | What they receive |
|---|---|---|
| HP802-247 | Experimental | Not described Receives: Biological: HP802-247 |
| Placebo (Vehicle) | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to healing
Time to healing, defined as the number of days between wound onset (caregiver reported) and complete epithelialization (Investigator assessed).
Measured: 4 weeks
Secondary outcomes (4)
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)
Measured: 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.
Measured: 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)
Measured: 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).
Measured: 4 weeks for each of 3 treatment periods
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2012 (estimated)
- Study startMar 2012
- Primary completionMar 2013 (estimated)
- Study completionJul 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Healthpoint
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Herbert B Slade, MD, Study chair, HealthpointJaime E Dickerson, PhD, Study director, HealthpointHerbert B Slade, MD, Principal investigator, Healthpoint
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01528306
- Sponsor's protocol ID
- 802-247-09-026
- Status last verified
- May 2012
- First submitted
- 31 Jan 2012
- Last updated
- 16 May 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory, Cross-Over Study of the Safety of HP802-247 Applied to Open Wounds of Subjects With Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT01528306
- Acronym / sponsor ID
- 802-247-09-026
- Conditions
- Dystrophic Epidermolysis Bullosa
- Keywords
- HP802-247, EB (epidermolysis bullosa)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- HealthpointIndustry (pharma, biotech, devices)
- Study start
- Mar 2012
- Primary completion
- Mar 2013 (estimated)
- Study completion
- Jul 2013 (estimated)
- First posted
- 8 Feb 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HP802-247 | Experimental | Biological: HP802-247 |
| Placebo (Vehicle) | Placebo comparator | Other: Placebo |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- 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:
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HP802-247Wounds 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 | Biological | HP802-247 |
| PlaceboWounds 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 | Other | Placebo (Vehicle) |
Primary outcome
Time to healing
Time frame: 4 weeks
Time to healing, defined as the number of days between wound onset (caregiver reported) and complete epithelialization (Investigator assessed).
4 secondary outcomes
Wound pain as measured by faces pain scale
Time frame: 4 weeks for each of 3 treatment periods
Percent of change in wound area
Time frame: 4 weeks for each of 3 treatment periods
Persistence of healing
Time frame: 4 weeks for each of 3 treatment periods
Burning and stinging upon application
Time frame: 4 weeks for each of 3 treatment periods
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Herbert B Slade, MD study chair
Healthpoint
Jaime E Dickerson, PhD study director
Healthpoint
Herbert B Slade, MD principal investigator
Healthpoint
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HP802-247 in people with Dystrophic Epidermolysis Bullosa, run by Healthpoint.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 16 May 2012. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to healing (time frame: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2012.