A Phase 1 Study to Evaluate the Safety and Tolerability of Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
National Heart, Lung, and Blood Institute (NHLBI)
Summary
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen. Research has shown that curcumin, a natural compound found in turmeric, can improve the health of red blood cells in people with SCD. But the body cannot absorb curcumin well when it is taken by mouth. Researchers want to know if a skin gel (VAS-101) can help the body better absorb curcumin. VAS-101 contains curcumin, which comes from turmeric. Objective: To test VAS-101 in people with stable SCD. Eligibility: People aged 18 to 70 years with stable SCD. Design: People who want to join the study will be screened with physical exam with blood tests to see if they are eligible. If they qualify, they can enroll in the study. Participants will have up to 15 clinic visits over about 14 weeks. Some may need to stay overnight in the hospital for up to 2 days to make it easier to collect blood samples after the gel is applied. For 6 weeks, a special gel called VAS-101 will be put on the forearms in the clinic two times a week. Staff will rub the gel into the skin for at least 30 seconds using a soft toothbrush. The area stays uncovered for at least 10 minutes, then is covered with a bandage or sleeve. After 24 hours, the dressing can be removed and the skin can be washed. Some visits will include blood tests and other exams. On three visits, a test called near infrared spectroscopy (NIRS) will be done. For this test, probes are placed on the skin to measure blood flow, oxygen levels, and the makeup of skin and muscle. A blood pressure cuff is used to squeeze the arm for up to 5 minutes. The last clinic visit will happen about 4 weeks after the final gel application.
Description
Study Description: The overall objective of this study is to assess the clinical safety and tolerability of a patented, bioavailability-enhanced, transdermal application of curcuminoids, VAS-101 / Vasceptor(R) (8.5 percent curcuminoids transdermal gel), in subjects with stable sickle cell disease (SCD). VAS-101 contains Curcugen(R) as the active ingredient, with concentrations of 2mg in VAS-101 0.05 mL, 4mg in VAS-101 0.1 mL, and 12mg in VAS-101 0.3 mL. Subjects enrolled will receive 0.2 mL of VAS-101 topically twice weekly for two weeks, followed by 0.4 mL twice weekly for two weeks, then 0.…
Eligibility
- Age range
- 18–90 years
- Sex
- All
- Healthy volunteers
- No
* INCLUSION CRITERIA: Subjects will enroll onto the study and undergo screening. Subjects who do not meet any of the following criteria during screening will not receive the study intervention but will be counted toward study accrual. Screen failures may be rescreened at a later time. In order to be eligible to participate in this study, an individual must meet all of the following criteria: * Have provided signed written informed consent prior to performing any study procedure, including screening procedures. * Age between 18-70 years. Adults over 70 years of age will be excluded from this…
Interventions
- DrugVAS-101
VAS-101 contains Curcugen (Registered Trademark) as the active ingredient, with concentrations of 2mg in VAS-101 0.05 mL, 4mg in VAS-101 0.1 mL, and 12mg in VAS-101 0.3 mL
Location
- National Institutes of Health Clinical CenterBethesda, Maryland