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New treatment option for Sickle Cell Disease, Hemolytic Anemia
Official title Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
ClinicalTrials.gov ID: NCT07566494
What this study is testing
What is VAS-101?
VAS-101 is an investigational medicine, given as an once-weekly pill taken by mouth, being studied as a potential treatment for sickle cell disease, hemolytic anemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.
- Phase 1: an early, usually small safety study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 90
You may be able to join if
- people will enroll onto the study and undergo screening. people who do not meet any of the following criteria during screening will not receive the...
- 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...
- Unequivocal diagnosis of SCD (HbSS, HbSC, HbSbeta+ or HbSbeta\^0) confirmed by hemoglobin electrophoresis performed on patients at least 60 days...
- No transfusion in the 60 days prior to signing consent, or absence of Hb A on hemoglobin analysis (by high-performance liquid chromatography; HPLC)
You likely can't join if
- 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...
- Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound the interpretation...
- 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...
See the full eligibility criteria
Who can join
- people will enroll onto the study and undergo screening. people 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...
- 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, 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...
- 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...
- 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...
- Be willing to comply with all study procedures for the duration of the study.
What rules you out
- 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...
- 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...
- Clinically symptomatic cholelithiasis or cholecystitis. Prior cholecystectomy is not exclusionary. people with symptomatic cholelithiasis or cholecystitis may be rescreened once the disorder has been treated and...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- Have a history of allergy to turmeric, curcuminoids, or formulations thereof.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Compensation & support
A stipend or compensation may be offered.
Compensation likely.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 90 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.