Why are we doing this research?
Cincinnati Children’s is conducting a research
study, sometimes known as a clinical trial or clinical study, to evaluate the
safety and effectiveness of an investigational skin cream, SD-101, for the
treatment of people with Epidermolysis Bullosa, or EB.
The investigational skin cream is being developed to address the most urgent issues of patients with EB (including wounds and blisters) and can be used with a participant’s current care plan.
Who can participate?
children, teens and adults who are at least 1 month old and have Recessive
Dystrophic EB, Junctional EB, or EB Simplex may be eligible for participation.
Only one member of a family may
What will happen in the study?
If you (as a participant who is 18 or older) or your child qualifies, and you decide you want to take part in the study, you will come to Cincinnati Children’s 5 times over the next 3 months. Visits may last from 1 to 4 hours.
The following tests and procedures will occur during one or more of the study visits:
- Review of demographic information (such as age or race)
- Review of medical and medication/supplement history
- Review of how you or your child is feeling
- Physical exam (including height and weight measurements)
- Urine sample (for pregnancy testing, if you are female and of childbearing age)
- Complete skin examination
- Evaluation of itching, pain, body surface area (BSA) and scarring
- Identification, photographs and assessments of 1 target wound
- Completion of a patient diary
- Completion of itching and skin pain scores
During the first visit, or the screening, study staff will make sure that you or your child is eligible to participate in this research study. If you or your child are eligible, and you agree to participate, the doctor will identify 1 target wound and a photograph will be taken of the wound area.
You or your child will then be randomly (like flipping a coin) placed into 1 of 2 study groups to receive the active study cream, SD-101, or a placebo cream. A placebo looks like the study cream but does not contain any active ingredient. You or your child will apply the cream to your skin over your entire body once a day for the next 90 days
While in the study, neither you, your child, nor the study staff will know which cream (study cream or placebo) you are using.
You or your child will come back for 4 more study visits. At every visit, you or your child will return all of the study cream as well as the participant diary.
Participants or parents interested in having their child participate will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.
What are the good things that can happen from this research?
in this study may
not help you
or your child right now. Symptoms may improve, worsen or stay the same. However,
you or your child’s participation will provide information about the study
cream, which might help you or others in the future.
What are the bad things that can happen from this research?
list of possible side effects will be provided to those participants, parents
or guardians interested in knowing more about this study.
Will you/your child be paid to be in this research study?
Participants will not receive payment for being
in this study; however, travel reimbursement will be available. Those who
complete this study may also be eligible to receive the active study cream in a
follow-up, long-term safety study.