Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa

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Brief Title

Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa

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

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.
    

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 Phase

Phase 1

Study Type

Interventional


Primary Outcome

Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment

Secondary Outcome

 Peak Plasma Concentration of BPM31510

Condition

Epidermolysis Bullosa

Intervention

topical BPM31510 3.0% Cream

Study Arms / Comparison Groups

 Topical BPM31510 3.0% Cream
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

11

Start Date

August 10, 2016

Completion Date

August 26, 2019

Primary Completion Date

August 26, 2019

Eligibility 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.
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02793960

Organization ID

20151103


Responsible Party

Sponsor-Investigator

Study Sponsor

Shasa Hu

Collaborators

 Berg, LLC

Study Sponsor

, , 


Verification Date

June 2020