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CT Open to Enrollment

TARGET–NASH: A 5-year Longitudinal Observational Study of Patients with Non-Alcoholic Fatty Liver or Non-Alcoholic Steatohepatitis

This is an observational study. This study is for men and women who have nonalcoholic fatty liver (NAFL), which is storage of fat in the liver, or NASH (nonalcoholic steatohepatitis) which is a form of NAFL liver disease that includes irritation or inflammation of the liver due to fat. 

A Gene-Transfer, Open-Label, Dose-Escalation Study of SPK-8011 [Recombinant Adeno-Associated Viral Vector with B-Domain Deleted Human Factor VIII Gene] in Individuals with Hemophilia A

Hemophilia A is a condition in which blood is unable to clot effectively. It is caused by a mutation or deletion in the gene that is responsible for producing blood-clotting factor VIII protein. Individuals with hemophilia A suffer from repeated bleeding episodes, often into the joints, which can cause chronic joint disease and sometimes results in death due to the inability of the blood to clot efficiently. This chronic joint disease can have significant physical, psychosocial, and quality-of-life effects, including financial burden.

IRONMAN: International Registry for Men with Advanced Prostate Cancer

This clinical trial is for patients with Advanced Prostate Cancer. The purpose of this study is to learn more about prostate cancer and:

Fecal Microbiota Transplantation National Registry

This registry study is being done to collect information on fecal microbiota transplant (FMT). Eligible participants are men or women who are being seen in the clinic as an FMT recipient or FMT donor or receiving other gut-related microbiota product. 

(CLONEVO): Cell cycLe inhbitiON to target the EVolution of urOthelial cancer

This is a clinical trial for men and women age 18 and older diagnosed with advanced bladder cancer who are not eligible or choose not to receive chemotherapy before standard-of-care surgery for bladder cancer

The purpose of this study is to test the safety of the study drug, abemaciclib, and see what effects (good and bad) it has in muscle-invasive bladder cancer. 

ROMA Trial

The purpose of this study is to determine the effect of single vs. multiple arterial revascularization (this is the restoration of blood flow to an organ) for patients undergoing coronary artery bypass surgery. 

Comprehensive Care in Patients with Thalassemia and Severe Congenital Anemias

 The purpose of this study is to continue to learn about impact of comprehensive care on the natural history of patients with thalassemia (Cooley’s anemia) and other severe anemias requiring periodic or regular red blood cell transfusions. We will collect data on guideline recommended measures obtained from the standard of care: routine tests (EKG, Echocardiogram, Holter Monitor, MRI, eye exam, hearing test, x-ray, bone studies, pulmonary function study) done once a year and monthly blood draw and urinalysis for complete blood count, chemistries, and iron studies.

Safety of Immunosuppression in A Prospective Cohort of Inflammatory Bowel Disease Patients With a History of Cancer (SAPPHIRE)

This is a prospective, multi-center, observational study designed to determine if specific agents used for treatment of inflammatory bowel disease (IBD) are associated with the development of new or recurrent cancer in patients with IBD and a history of cancer.

 Commitments for enrollment include:

BP1001 in Combination with Low-dose Cytarabine (LDAC) in Patients with Previously Untreated (AML) Who Are Ineligible for Induction Therapy

The goal of this clinical research study is to assess whether the combination of BP1001 and cytarabine provides greater efficacy (Complete Remission [CR], Complete Remission with incomplete hematologic recovery [CRi], Complete Remission with incomplete platelet recovery [CRp]) than cytarabine alone (by historical comparison) in participants with AML that cannot or elect not to be treated with more intensive chemotherapy.