351 Studies Now Enrolling
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Displaying 1 - 4 of 4
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: Cancer / Brain Cancer
Investigator: Fabio Iwamoto, MD
Status: Currently Recruiting
The purpose of this study is to learn the effects of the investigational new drug, tirabrutinib, when given for the treatment of PCNSL. Investigational new drug means a drug that has not been approved as a marketed product (that is, available to be prescribed or sold) by the United States (US) Food and Drug Administration (FDA). Tirabrutinib acts by…
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 / Brain Cancer
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this study is to evaluate and determine the efficacy of pemigatinib in participants with recurrent glioblastoma or other CNS tumors with changes in the FGFR1, FGFR2 or FGFR3 genes. Pemigatinib (also referred as INCB054828) is an investigational drug that is being studied by Incyte Corporation (Sponsor) for use in the treatment of previously…
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