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  • Currently Recruiting (2)
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  • Autoimmune Disorders (3)
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Displaying 1 - 8 of 8

Stem Cell Treatment for Patients with Perianal Fistula due to Crohn's Disease

Condition: Gastrointestinal and Digestive Diseases
Investigator: Marco Bertucci Zoccali, MD
Status: Currently Recruiting
The purpose of this study is to research and evaluate safety and effectiveness of the administration of bone marrow mesenchymal stem cell (bmMSC) derived extracellular vesicles product, DB-3Q, the study drug for perianal fistulizing Crohns disease. The study will…
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Study of stem cell transplant effects in patients with sickle cell disease

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle…
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A Study to Evaluate the Safety of a Dose of Autologous CRISPR-Cas9 Human Stem Cells in Subjects With Severe Sickle Cell Disease

Condition: Blood Disorders
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stem cells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent…
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Study of EDIT-301 in Patients with Severe Sickle Cell Disease (SCD)

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem…
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Study of Patients Receiving a Stem Cell Transplant for Non-Malignant Disease

Condition: Blood Disorders
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant condition.
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Crohn's Disease treatment with Vedolizumab (ENTYVIO) With and Without Upadacitinib (Rinvoq)

Condition: Gastrointestinal and Digestive Diseases
Investigator: Bo Shen, MD
Status: Currently Recruiting
The Takeda study drug used in this study is vedolizumab, a monoclonal antibody. Monoclonal antibodies are proteins made in laboratories that act like proteins in our bodies called antibodies. Vedolizumab acts specifically in the gut and works by preventing certain immune cells from going to the gut and helps reduce…
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Study For Previously Treated Patients Of Light-Chain Amyloidosis Using Study Drug Carfilzomib

Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is a research study for subjects that have been previously treated for systemic light chain (AL)amyloidosis (a disease in which abnormal protein deposits can damage organs and tissues in your body) and nowrequire further treatment. The main purpose of this study is to evaluate the safety and determine the maximum…
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A study for patients with myelofibrosis using study drug PF-04449913

Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
This is a Phase 2 study for patients with myelofibrosis previously treated with one or more Janus Kinase(JAK) Inhibitor. A JAK inhibitor targets a certain pathway that may be causing cancer to grow, bystopping or inhibiting the cycle of development of the pathway. There is one JAK inhibitor that has beenapproved for the treatment of patients with…
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