Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa

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.

Study Overview

• Status: Completed
• Conditions: Epidermolysis Bullosa
• Intervention / Treatment: Drug: topical BPM31510 3.0% Cream

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 Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • University Of Miami Department of Dermatology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment	Analysis of laboratory values and/or Adverse events	16 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Day 1, Day 3, week 8.
Decrease in VAS Pain Scale questionnaire	Subject completed Questionnaire will assess the .pain at each visit.	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.	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.	Baseline, 16 weeks

Collaborators and Investigators

• Sponsor: Shasa Hu
• Collaborators: Berg, LLC

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 10, 2016
• Primary Completion (ACTUAL): August 26, 2019
• Study Completion (ACTUAL): August 26, 2019

Study Registration Dates

• First Submitted: May 18, 2016
• First Submitted That Met QC Criteria: June 3, 2016
• First Posted (ESTIMATE): June 8, 2016

Study Record Updates

• Last Update Posted (ACTUAL): June 16, 2020
• Last Update Submitted That Met QC Criteria: June 14, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa
• Other Study ID Numbers: 20151103

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO