236 Studies Now Enrolling
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- Autoimmune Disorders (3)
- (-) Blood Disorders (6)
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- Neurological Disorders (14)
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Displaying 1 - 11 of 11
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…
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…
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…
Condition: Cancer / Leukemia
Investigator: Ran Reshef, MD
Status: Closed
The main purpose of this research study is to investigate if a drug called Vedolizumab is safe and effective in preventing acute graft-versus-host disease (attack of donor cells on recipient healthy organs after transplant) of the gut in comparison to placebo (dummy drug). This will be studied in patients undergoing…
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.
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to determine the optimal dose of oral CGT9486 in patients with Advanced Systemic Mastocytosis (AdvSM) and to determine the efficacy of CGT9486 at the selected optimal dose in patients with Advanced Systemic Mastocytosis (AdvSM). This includes Aggressive Sy…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to test the safety and effectiveness of avapritinib (also known as BLU-285) in individuals who have been diagnosed with aggressive systemic mastocytosis (ASM), Systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell…
Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study will enroll with children, adolescents and young adults that are less than 22 years old that have returned or 'relapsed' acute myeloid leukemia (AML) diagnoses. Within this group, the study is also targeting those are also unable to receive additional chemotherapy treatments containing anthracycline, which is a type of antibiotic that is used to…
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…
Condition: Cancer / Leukemia
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem …
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…