Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa

• ClinicalTrials.gov Identifier: NCT02793960
• Recruitment Status: Completed
• First Posted: June 8, 2016
• Last Update Posted: June 16, 2020
• Sponsor: Shasa Hu
• Collaborator: Berg, LLC
• Information provided by (Responsible Party): Shasa Hu, University of Miami

Study Description

Brief 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.

Condition or disease	Intervention/treatment	Phase
Epidermolysis Bullosa	Drug: topical BPM31510 3.0% Cream	Phase 1

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

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 11 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: August 10, 2016
• Actual Primary Completion Date: August 26, 2019
• Actual Study Completion Date: August 26, 2019

Arms and Interventions

Arm	Intervention/treatment
Experimental: Topical BPM31510 3.0% Cream; 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.	Drug: topical BPM31510 3.0% Cream; Application of drug to lesions of epidermolysis bullosa

Outcome Measures

Primary Outcome Measures :

1. 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

Secondary Outcome Measures :

1. Peak Plasma Concentration of BPM31510 [ Time Frame: Day 1, Day 3, week 8. ]
   Blood will be drawn pre-study drug application on Day 1 and post study drug application on Day 3 and Week 8
2. Decrease in VAS Pain Scale questionnaire [ Time Frame: Baseline, 16 weeks ]
   Subject completed Questionnaire will assess the .pain at each visit.
3. Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) [ Time Frame: Baseline, 16 weeks ]
   The (EBDASI) be utilized by investigators to quantify wound healing and scarring at each visit.
4. Change in the Lansky Performance Scale and the Children's Dermatology Life Quality Index [ Time Frame: Baseline, 16 weeks ]
   Subject completed questionnaires will be completed at each visit.

Eligibility Criteria

• Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Male or female at least 12 years of age at the time of screening.
2. Have primary, histologically confirmed EB verified by immunofluorescent antigenic mapping (EM) prior to starting application of study drug
3. Have no other dermatological disease that may adversely impact wound healing.
4. 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.
5. Willing to refrain from exposure to excessive direct sunlight or ultraviolet light for the duration of the study.
6. 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.
7. Caregiver/guardian or patient is able to follow study required instructions and likely to complete all study visit requirements.
8. 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.
9. 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.
10. 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).
11. Have an INR value of 0.8-1.2 as well as normal PT/APTT.
12. With at least 1 active EB wound between 2.5 and 50 cm2 in size on nonflexual surfaces

Exclusion Criteria:

1. Received systemic therapy of living skin equivalent grafting in the past 30 days prior to baseline visit.
2. Known or suspected systemic cancer such as lymphoma or leukemia.
3. 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.
4. Concurrent disease or treatment that suppresses the immune system.
5. 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.
6. Known sensitivity to any of the ingredients in the trial medication (BPM31510 3.0% Cream
7. Treatment with any systemic immunomodulators or immunosuppressants within the 2 months prior to enrollment, for a duration longer than 2 weeks.
8. Use of any topical immunomodulators such as topical tacrolimus.
9. Use of systemic or topical steroids within 30 days prior to enrollment is excluded (inhaled steroids and ophthalmic drops containing steroids are allowed).
10. 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.
11. Current enrollment in an investigational drug or device study or participation in such a study within 4 weeks of enrollment.
12. In the investigator's opinion, evidence of unwillingness or inability of the patient or caregiver to follow the restrictions and complete the study.
13. Has any clotting disorder, or is being treated with any anticoagulant.
14. Any abnormal laboratory value or concurrent illness that, in the opinion of the investigator, should preclude the patient's participation in the study.

Contacts and Locations

Locations

United States, Florida

University of Miami Department of Dermatology

Miami, Florida, United States, 33136

Sponsors and Collaborators

Shasa Hu

Berg, LLC

More Information

• Responsible Party: Shasa Hu, Professor, University of Miami
• ClinicalTrials.gov Identifier: NCT02793960
• Other Study ID Numbers: 20151103
• First Posted: June 8, 2016
• Last Update Posted: June 16, 2020
• Last Verified: June 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

Additional relevant MeSH terms:

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin Diseases, Vesiculobullous