Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
A Phase 1 Study to Evaluate the Safety and Tolerability of Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years to 90 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Mentioned in the study description
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Visits mentioned: 15 visits, 3 visits
- Time periods mentioned: 14 weeks, 2 days, 6 weeks, 2 weeks
- Procedures mentioned: Blood draws, Physical exams
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "Design: People who want to join the study will be screened with physical exam with blood tests to see if they are eligible."
- "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."
- "Some visits will include blood tests and other exams."
- "On three visits, a test called near infrared spectroscopy (NIRS) will be done."
- Compensation
- Mentioned in the study description
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests VAS-101, a skin gel made with curcumin, a substance found in turmeric. Researchers want to learn whether the gel helps the body absorb curcumin better. It is for adults ages 18 to 70 with stable sickle cell disease, an inherited blood disorder.
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....
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be 18 to 70 years old and have sickle cell disease confirmed by a blood test.
- Sign a consent form and agree to follow all study steps.
- Have had no blood transfusion in the past 60 days, or have no hemoglobin A found in your blood test.
- Have a hemoglobin level of at least 7 g/dL. Hemoglobin is the protein in red blood cells that carries oxygen.
- Meet the study’s limits for liver and kidney test results.
- If taking hydroxyurea, L-glutamine, or crizanlizumab, have been on a stable dose for at least 90 days.
- Avoid all other products containing turmeric or curcumin until all study visits are done.
- If able to become pregnant, have a negative pregnancy test. Use two effective forms of birth control, or continue your usual practice of not having sex, during the study and for 28 days after the last dose.
Who can't join
- A sickle cell pain crisis needing treatment by injection or IV in the past 14 days, or three or more crises needing care at a medical facility in the past 12 months.
- A health condition that makes the study unsafe or affects its results. Examples include poorly controlled blood pressure or diabetes, recent serious heart or blood vessel problems, certain liver or gallbladder problems, active infections, certain cancers, or mental health problems that prevent study participation.
- A past bone marrow or stem cell transplant.
- Taking part in another treatment study, or exposure to an experimental drug, device, or invasive procedure in the past 12 weeks. Other recent invasive procedures may also rule you out.
- Taking nitroglycerin or another drug that raises nitrate levels.
- Being pregnant or breastfeeding.
- Using drugs that help the body make blood cells, unless stopped at least 90 days before signing consent.
- An allergy to turmeric, curcumin-related substances, or products containing them.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
* 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 study due to an increased risk of more severe disease and higher prevalence of multiple comobodities. * Unequivocal diagnosis of SCD (HbSS, HbSC, HbSbeta+ or HbSbeta\^0) confirmed by hemoglobin electrophoresis performed on patients at least 60 days after a blood transfusion if previously transfused. * No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC) * Serum aspartate aminotransferase (AST) \<= 1.5 x Upper Limit of Normal (ULN) (unless the increased AST is assessed by the Investigator as due to hemolysis) and alanine aminotransferase (ALT) \<=1.5 x ULN. * Hemoglobin \>= 7 g/dL * Serum creatinine \<=1.25 x ULN. If serum creatinine is \>1.25 x ULN, then glomerular filtration rate (based on creatinine) must be \>=60 mL/min. * If on hydroxyurea, participant must have been on stable dose of hydroxyurea (defined as a stable dose for at least 90 days and inclusive of dose modifications for hematological toxicity per PI discretion) prior to signing consent. * If on L-glutamine and/or crizanlizumab, participant must have been on a stable dose for ast least 90 days prior to signing consent. * Agree to abstain from taking any other products/supplements containing turmeric or curcumin until all study visits have been completed (oral, topical, etc.) * For women of reproductive potential, have a negative serum pregnancy test during the screening period. Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy, or tubal occlusion; or who have not been naturally postmenopausal (i.e., who have not menstruated at all for at least the preceding 1 year prior to signing informed consent unrelated to hormonal contraception). * Women of reproductive potential be abstinent as part of their usual lifestyle, or agree to use 2 effective forms of contraception from the time of giving informed consent, during the study, and for 28 days following the last dose of study treatment. An effective form of contraception is defined as hormonal oral contraceptives, injectables, patches, intrauterine or subdermal contraceptive implants, and barrier methods. * Be willing to comply with all study procedures for the duration of the study. EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study: * Vaso-occlusive crisis requiring parenteral treatment within 14 days of signing consent. * Three or more vaso-occlusive crises in the 12 months prior to screening that resulted in receiving treatment in an urgent care, outpatient infusion center/day-clinic, emergency department or inpatient setting. * Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following: * Poorly controlled hypertension (defined as systolic blood pressure \[BP\] \>150 mmHg or diastolic BP \>90 mmHg) refractory to medical management. * History of recent (within 24 weeks prior to signing consent) decompensated congestive heart failure; myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism. * Clinically symptomatic cholelithiasis or cholecystitis. Prior cholecystectomy is not exclusionary. Subjects with symptomatic cholelithiasis or cholecystitis may be rescreened once the disorder has been treated and clinical symptoms have resolved. * History of drug-induced cholestatic hepatitis. * Iron overload sufficiently severe to result in a clinical diagnosis by the Investigator of cardiac (e.g., clinically significant impaired left ventricular ejection fraction), hepatic (e.g., fibrosis, cirrhosis), or pancreatic (e.g., diabetes) dysfunction. * Positive test for hepatitis B surface antigen or hepatitis C virus (HCV) antibody (Ab) with signs of active hepatitis B or C virus infection. If the subject is positive for HCVAb, a reverse transcriptase-polymerase chain reaction test will be conducted. Subjects with hepatitis C may be rescreened after receiving appropriate hepatitis C treatment. * Active infection requiring any use of systemic antimicrobial agents (parenteral or oral) or Grade \>=3 in severity (per National Cancer Institute Common Terminology Criteria for Adverse Events) within 4 weeks prior to signing consent. * Diabetes mellitus judged to be under poor control by the Investigator or requiring \>3 antidiabetic agents, including insulin (all insulins are considered 1 agent); use of insulin per se is not exclusionary. * History of any primary malignancy, with the exception of: curatively treated nonmelanomatous skin cancer; curatively treated cervical or breast carcinoma in situ; or other primary tumor treated with curative intent, no known active disease present, and no treatment administered during the last 3 years. * Current or recent history of psychiatric disorder that, in the opinion of the Investigator or Medical Monitor, could compromise the ability of the subject to cooperate with study visits and procedures. * Have had a prior bone marrow or stem cell transplant. * Are currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo. * Taking nitroglycerin, or any other nitrate-enhancing drug. * Have exposure to any investigational drug, device, or invasive procedure within 12 weeks prior to signing consent. All non-investigational invasive procedures within 12 weeks of signing consent may be considered as a potential exclusion criteria per the PI's discretion. * Currently pregnant or breastfeeding. * Currently receiving hematopoietic stimulating agents (e.g., erythropoietins, granulocyte colony stimulating factors, thrombopoietins) that have not been stopped for a duration of at least 90 days prior to signing consent. * Have a history of allergy to turmeric, curcuminoids, or formulations thereof.
Study sites (1)
MD
- National Institutes of Health Clinical Center · Bethesda, MD 20892NIH Clinical Center Office of Patient Recruitment (OPR) · Study contact · 800-411-1222 ext. TTY dial 711 · ccopr@nih.govMore trials in Bethesda