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Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02793960
Recruitment Status : Active, not recruiting
First Posted : June 8, 2016
Last Update Posted : July 9, 2019
Sponsor:
Collaborator:
Berg, LLC
Information provided by (Responsible Party):
Shasa Hu, University of Miami

Tracking Information
First Submitted Date  ICMJE May 18, 2016
First Posted Date  ICMJE June 8, 2016
Last Update Posted Date July 9, 2019
Actual Study Start Date  ICMJE August 10, 2016
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 9, 2017)
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
Original Primary Outcome Measures  ICMJE
 (submitted: June 3, 2016)
Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [ Time Frame: 16 weeks ]
Change History Complete list of historical versions of study NCT02793960 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 3, 2016)
  • 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
  • Decrease in VAS Pain Scale questionnaire [ Time Frame: Baseline, 16 weeks ]
    Subject completed Questionnaire will assess the .pain at each visit.
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Epidermolysis Bullosa
Intervention  ICMJE Drug: topical BPM31510 3.0% Cream
Application of drug to lesions of epidermolysis bullosa
Study Arms  ICMJE 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.
Intervention: Drug: topical BPM31510 3.0% Cream
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: July 8, 2019)
11
Original Estimated Enrollment  ICMJE
 (submitted: June 3, 2016)
10
Estimated Study Completion Date  ICMJE January 2020
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02793960
Other Study ID Numbers  ICMJE 20151103
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Shasa Hu, University of Miami
Study Sponsor  ICMJE Shasa Hu
Collaborators  ICMJE Berg, LLC
Investigators  ICMJE Not Provided
PRS Account University of Miami
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP