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 : Completed
First Posted : June 8, 2016
Last Update Posted : June 16, 2020
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa||Drug: topical BPM31510 3.0% Cream||Phase 1|
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 (Clinical Trial)|
|Actual Enrollment :||11 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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
- 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
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02793960
|United States, Florida|
|University of Miami Department of Dermatology|
|Miami, Florida, United States, 33136|