Epidermolysis BullosaPhase 1NCT02793960
Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
A Phase 1 study testing topical BPM31510 3.0% Cream in people with Epidermolysis Bullosa, run by Shasa Hu at 1 site in United States.
Trial summary
This is a single-center study to investigate the effects of a topical cream on patients 12 years of age and older that have been diagnosed with epidermolysis bullosa. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- topical BPM31510 3.0% Cream
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Aug 2016to 26 Aug 2019
- Phase
- Phase 1
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female at least 12 years of age at the time of screening.
- Have primary, histologically confirmed EB verified by immunofluorescent antigenic mapping (EM) prior to starting application of study drug
- Have no other dermatological disease that may adversely impact wound healing.
- Willing to refrain from using topical creams or lotions other than the study drug to the designated areas during the treatment period and from washing the designated areas until the next application is done.
- Willing to refrain from exposure to excessive direct sunlight or ultraviolet light for the duration of the study.
- Laboratory values for the tests listed in the Study Schedule are within the local reference ranges as defined by the local laboratory, or "out of range" test results are clinically acceptable to the investigator. Acceptable "out of range" values are generally those within the patient's normal baseline levels due to concurrent medications or disease processes with the exception of INR and PT/APTT.
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- Caregiver/guardian or patient is able to follow study required instructions and likely to complete all study visit requirements.
- Has a provided written informed consent by patient or a legal guardian, including consent for tissue to be examined and stored by the Department of Dermatology and Cutaneous Surgery. If the patient is between 12 and 17 years of age, assent must be given by the patient.
- Guardian has provided written consent to allow photographs of the target EB lesion(s) to be used as part of the study data and documentation.
- Females of childbearing potential must have a negative urine or serum pregnancy test at screening and be using an acceptable form of birth control (oral/implant/injectable/transdermal contraceptives, intrauterine device, condom, diaphragm, abstinence, or a monogamous relationship with a partner who has had a vasectomy).
- Have an INR value of 0.8-1.2 as well as normal PT/APTT.
- With at least 1 active EB wound between 2.5 and 50 cm2 in size on nonflexual surfaces
Who cannot join
- Received systemic therapy of living skin equivalent grafting in the past 30 days prior to baseline visit.
- Known or suspected systemic cancer such as lymphoma or leukemia.
- Evidence of dermatological disease or confounding skin condition in the treatment area, e.g., BCC, actinic keratosis, rosacea, psoriasis, atopic dermatitis, eczema, xerodermapigmentosa, or clinical evidence of infection.
- Concurrent disease or treatment that suppresses the immune system.
- Any chronic medical condition that, in the judgment of the investigator(s), can interfere with the performance of the study or would place the patient at undue risk.
- Known sensitivity to any of the ingredients in the trial medication (BPM31510 3.0% Cream
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- Treatment with any systemic immunomodulators or immunosuppressants within the 2 months prior to enrollment, for a duration longer than 2 weeks.
- Use of any topical immunomodulators such as topical tacrolimus.
- Use of systemic or topical steroids within 30 days prior to enrollment is excluded (inhaled steroids and ophthalmic drops containing steroids are allowed).
- Any elective or non-elective surgery other than biopsy for EB, or surgeries for the treatment of sequelae of EB such as g-tube placement or esophageal dilatation within 4 weeks prior to enrollment during the study. Elective procedures pertaining to skin graft will not be allowed at any time.
- Current enrollment in an investigational drug or device study or participation in such a study within 4 weeks of enrollment.
- In the investigator's opinion, evidence of unwillingness or inability of the patient or caregiver to follow the restrictions and complete the study.
- Has any clotting disorder, or is being treated with any anticoagulant.
- Any abnormal laboratory value or concurrent illness that, in the opinion of the investigator, should preclude the patient's participation in the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Miami Department of Dermatology
Miami, Florida, 33136
About this study
Official title: A Phase 1 Study to Evaluate the Safety, Pharmacokinetics and Therapeutic Effect of Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
This is a single-center study to investigate the effects of a topical cream on patients 12 years of age and older that have been diagnosed with epidermolysis bullosa.
Read the detailed description
The trial will be conducted in patients with any form of Epidermolysis Bullosa (EB) with at least 1 active EB wound between 2.5 and 50 cm2 in size or up to 10% of the Body Surface Area. The investigators will identify an "index lesion" and other lesions for treatment An area of un-involved skin will also be treated with the study cream. This are will be used for testing by producing tiny blisters th so that the underlying tissue can be examined using a special microscope.
Patients will apply the study cream
- Conditions
- Epidermolysis Bullosa
- Being tested
- topical BPM31510 3.0% Cream
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
topical BPM31510 3.0% CreamDrug
Application of drug to lesions of epidermolysis bullosa
| Study group | Role | What they receive |
|---|---|---|
| Topical BPM31510 3.0% Cream | Experimental | Patients/ caregiver will apply topical BPM31510 3.0% cream from every other day to twice per week to wounded skin, and every day to a section of intact skin for up to 12 weeks.The area to be covered may not exceed 10% BSA inclusive of intact skin area, and other lesions. Receives: Drug: topical BPM31510 3.0% Cream |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment
Analysis of laboratory values and/or Adverse events
Measured: 16 weeks
Secondary outcomes (4)
Peak Plasma Concentration of BPM31510
Blood will be drawn pre-study drug application on Day 1 and post study drug application on Day 3 and Week 8
Measured: Day 1, Day 3, week 8.
Decrease in VAS Pain Scale questionnaire
Subject completed Questionnaire will assess the .pain at each visit.
Measured: Baseline, 16 weeks
Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI)
The (EBDASI) be utilized by investigators to quantify wound healing and scarring at each visit.
Measured: Baseline, 16 weeks
Change in the Lansky Performance Scale and the Children's Dermatology Life Quality Index
Subject completed questionnaires will be completed at each visit.
Measured: Baseline, 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2016 (estimated)
- Study start10 Aug 2016
- Primary completion26 Aug 2019
- Study completion26 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shasa Hu
- Sponsor type
- University, hospital or other
- Collaborators
- BPGbio
- Responsible party
- Sponsor-investigator, Shasa Hu, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02793960
- Sponsor's protocol ID
- 20151103
- Status last verified
- Jun 2020
- First submitted
- 18 May 2016
- Last updated
- 16 Jun 2020
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Evaluate the Safety, Pharmacokinetics and Therapeutic Effect of Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT02793960
- Acronym / sponsor ID
- 20151103
- Conditions
- Epidermolysis Bullosa
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Shasa HuUniversity, hospital or other
- Collaborators
- BPGbio
- Study start
- 10 Aug 2016 (actual)
- Primary completion
- 26 Aug 2019 (actual)
- Study completion
- 26 Aug 2019 (actual)
- First posted
- 8 Jun 2016 (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 single-center study to investigate the effects of a topical cream on patients 12 years of age and older that have been diagnosed with epidermolysis bullosa.
Detailed description
The trial will be conducted in patients with any form of Epidermolysis Bullosa (EB) with at least 1 active EB wound between 2.5 and 50 cm2 in size or up to 10% of the Body Surface Area. The investigators will identify an "index lesion" and other lesions for treatment An area of un-involved skin will also be treated with the study cream. This are will be used for testing by producing tiny blisters th so that the underlying tissue can be examined using a special microscope.
Patients will apply the study cream
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Topical BPM31510 3.0% Cream | Experimental | Drug: topical BPM31510 3.0% CreamPatients/ caregiver will apply topical BPM31510 3.0% cream from every other day to twice per week to wounded skin, and every day to a section of intact skin for up to 12 weeks.The area to be covered may not exceed 10% BSA inclusive of intact skin area, and other lesions. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Male or female at least 12 years of age at the time of screening.
- Have primary, histologically confirmed EB verified by immunofluorescent antigenic mapping (EM) prior to starting application of study drug
- Have no other dermatological disease that may adversely impact wound healing.
- Willing to refrain from using topical creams or lotions other than the study drug to the designated areas during the treatment period and from washing the designated areas until the next application is done.
- Willing to refrain from exposure to excessive direct sunlight or ultraviolet light for the duration of the study.
Key exclusion criteria (14 listed)
- Received systemic therapy of living skin equivalent grafting in the past 30 days prior to baseline visit.
- Known or suspected systemic cancer such as lymphoma or leukemia.
- Evidence of dermatological disease or confounding skin condition in the treatment area, e.g., BCC, actinic keratosis, rosacea, psoriasis, atopic dermatitis, eczema, xerodermapigmentosa, or clinical evidence of infection.
- Concurrent disease or treatment that suppresses the immune system.
- Any chronic medical condition that, in the judgment of the investigator(s), can interfere with the performance of the study or would place the patient at undue risk.
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 |
|---|---|---|
| topical BPM31510 3.0% CreamApplication of drug to lesions of epidermolysis bullosa | Drug | Topical BPM31510 3.0% Cream |
Primary outcome
Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment
Time frame: 16 weeks
Analysis of laboratory values and/or Adverse events
4 secondary outcomes
Peak Plasma Concentration of BPM31510
Time frame: Day 1, Day 3, week 8.
Decrease in VAS Pain Scale questionnaire
Time frame: Baseline, 16 weeks
Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI)
Time frame: Baseline, 16 weeks
Change in the Lansky Performance Scale and the Children's Dermatology Life Quality Index
Time frame: Baseline, 16 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2020.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing topical BPM31510 3.0% Cream in people with Epidermolysis Bullosa, run by Shasa Hu at 1 site in United States.
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 12 inclusion and 14 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: finished, as of the sponsor's last update on 16 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment (time frame: 16 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 Jun 2020.