Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02793960
• Date of registration: 18/05/2016
• Prospective Registration: Yes
• Primary sponsor: Shasa Hu
• Public title: Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
• Scientific title: A Phase 1 Study to Evaluate the Safety, Pharmacokinetics and Therapeutic Effect of Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
• Date of first enrolment: August 10, 2016
• Target sample size: 11
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02793960
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion 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.



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa

Intervention(s)

Drug: topical BPM31510 3.0% Cream

Primary Outcome(s)

Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [Time Frame: 16 weeks]

Secondary Outcome(s)

Decrease in VAS Pain Scale questionnaire [Time Frame: Baseline, 16 weeks]

Change in the Lansky Performance Scale and the Children's Dermatology Life Quality Index [Time Frame: Baseline, 16 weeks]

Peak Plasma Concentration of BPM31510 [Time Frame: Day 1, Day 3, week 8.]

Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) [Time Frame: Baseline, 16 weeks]

Secondary ID(s)

20151103

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Berg, LLC