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This study is testing a stem cell (bone marrow) transplant for people with certain blood disorders. The goal is to find a treatment approach that is effective while causing fewer side effects. Participants will receive treatment to prepare their body, followed by a transplant using stem cells from a donor. After the transplant, they will receive medications to help prevent complications.

This research is being done to help us better understand the different DNA repair disorders. We will collect data and samples that we will use to develop new therapies and medicine to help treat the disease. We expect that participants will be in this research study for 3 years. Visits will occur every six months and alternate between in-person and remote. Remote visits should be expected to last 1-2 hours, and in-person visits should be expected to last 3-4 hours.

The purpose of the study is to establish and maintain a biorepository of tissue and biospecimen samples relevant to Amyotrophic Lateral Sclerosis (ALS) research. We will obtain, store, and catalogue peripheral blood mononuclear cells (PBMCs), blood and blood components, skin punch biopsy samples, and cerebral spinal fluid (CSF) from people living with ALS, linked to clinical datasets, to advance ALS research.

This research is being completed to examine the cells, brain imaging, and speech in individuals with REM Sleep Behavior Disorder who are taking serotonergic medications such as Citalopram, Escitalopram, Fluoxetine, Fluvoxamine, Paroxetine and Sertraline. The purpose of examining these is to try and see if we can predict signs of Dementia with Lewy bodies (a progressive form of dementia with an increase in decline of thinking, reasoning, and other functions). This may benefit others by enabling us to diagnose Dementia with Lewy Bodies sooner rather than later.

The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatments in ALS. A regimen is a specific course of treatment, each with a different study drug. We are doing this research to find out if different treatments have an effect on Amyotrophic Lateral Sclerosis (ALS). We also want to find out if these treatments are safe to take without causing too many side effects.

We are recruiting only healthy participants who do not have REM sleep behavior disorder. The information gained from this study will help the investigators understand more about REM sleep behavior disorder (RBD), and the possible underlying neurologic disorders that can cause RBD.

The goal of this study is to find out whether combining different study drugs makes tumors shrink in people with advanced head and neck cancer. This type of cancer is in the mouth, nose, throat, and sinuses. The study drugs are antibodies that act in different ways against cancer. BI 770371 and pembrolizumab may help the immune system fight cancer. Cetuximab blocks growth signals and may prevent the tumor from growing. Participants are put into 1 out of 3 groups, by chance (like drawing names from a hat), and which of the study treatments participants will receive.

The purpose of this study is to assess the safety of administering BAFFR-CAR T cells in participants with relapsed or refractory (r/r) B- cell non-Hodgkin lymphoma (B-NHL). We also will determine the maximum tolerated dose (MTD)/RP2D of BAFFR-CART cells.

This research study is for participants who have an inherited neuromuscular disorder or neurogenetic disorder, or family members who are unaffected by such disorders

The purpose of this study is to understand if a new immune treatment (pasritamig) added to best supportive care (including radiation, steroids, pain medication, or medications to strengthen your bones) for metastatic castration-resistant prostate cancer will work better than the best supportive care alone for patients without other treatment options.

The purpose of this research study is to obtain information on the safety and effectiveness of PBCAR0191 to treat certain types of cancers, such as Non-Hodgkin Lymphoma and B-cell Acute Lymphoblastic Leukemia. It is made from a type of blood cells known as T cells. The T cells in PBCAR0191 came from people who have donated their blood. The donated T cells have been genetically changed, so that they may be able to kill specific cancer cells commonly present in Non-Hodgkin Lymphoma and B-cell Acute Lymphoblastic Leukemia.

This is a small, Phase 0, window of opportunity study to provide human experience to support our pre-clinical data and gain preliminary information regarding the safety and tolerability of mirdametinib and vorinostat when given in combination.

This phase Ib/II trial evaluates the safety, optimal dose, and efficacy of the combination of epcoritamab and ibrutinib in treating patients with aggressive B-cell non-Hodgkin lymphoma that has come back (relapsed) or responded to previous treatment (refractory).

The purpose of this study is to find out if a study drug called BI 765423 can improve lung function in people with idiopathic pulmonary fibrosis (IPF). This study compares BI 765423 with a placebo to see if there is a difference in lung function or blood test results related to lung health after 3 month. The placebo looks like BI 765423 but does not contain any active drug.

This research trial studies the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.

We have determined that the microbes (bacteria) in the colon can play a role in causing and preventing complications of colon surgery. While the surgical bowel prep before surgery eliminates the harmful bacteria, it also eliminates the beneficial bacteria that aid wound healing. The purpose of this study is to determine if we can restore the presence of good bacteria (also known as ‘intestinal microbiota’) in the colon by transplanting them from a healthy donor.

This trial will evaluate whether azirkitug (ABBV-514) as monotherapy and in combination with budigalimab (ABBV-181) or bevacizumab is safe and tolerable for people with locally advanced or metastatic solid tumors.

The study evaluates the safety and effects of a novel regulatory CARTreg cell-based autoimmune and inflammatory disease therapy for the treatment of rheumatoid arthritis. The therapy is an autologous (using the patient's own cells) Treg cell therapy that targets proteins in the inflamed, disease-associated tissue, with the aim to dampen inflammation and restore balance to the immune system.

The main purpose of this study is to look at how safe and well tolerated the study drug is in combination with ribociclib (Group 1) or alpelisib (Group 2), the levels of the study drug and ribociclib or alpelisib in your blood, and how your body and your cancer respond.

The purpose of this study is to compare two treatment approaches for people with amyloidosis: daratumumab, cyclophosphamide, bortezomib, and dexamethasone (Dara-VCD) chemotherapy followed by an autologous stem cell transplant versus Dara-VCD followed by daratumumab maintenance therapy without a stem cell transplant.

The purpose of this study is to compare effects of the study drug fosmanogepix with already-approved drugs caspofungin and fluconazole to find out if fosmanogepix is safe and effective in treating patients with candidemia and/or invasive candidiasis.

This study will test GTB-5550, a B7H3-targeted natural killer (NK) cell engager, for the treatment of select solid tumor cancers. The study will determine the optimal dose level and evaluate preliminary safety and efficacy.

The purpose of this project is to ask adults with cystic fibrosis (CF) about their opinions and experiences with working with a primary care provider (PCP). It is also to assess the utilization of primary care providers in a complex medical care patient population whose primary providers have historically had a medical home in the pulmonary subspecialty.

The purpose of this study is to test a new CAR T-cell therapy called AZD0120 in adults with multiple myeloma that has returned or has not responded to previous treatments. Researchers want to learn whether this therapy is safe, determine the best dose to use, and see how well it works against the cancer. The study will also look at how the treatment affects the immune system and participants’ quality of life.

The purpose of the study is to determine the safety and effectiveness of a new procedure to treat Mucopolysaccharidosis Type I Hurler-Scheie and Scheie (MPS I). This procedure involves collecting some white blood cells (termed “B cells”) and growing them outside of the body in a laboratory. While the cells are in the lab, the B cells will be changed to produce more of the IDUA that is missing. This process is called “genetic modification.” The newly modified B cells are then infused back into the participant.

The purpose of this research study is to evaluate the safety and effectiveness of a new investigational drug called atumelnant for adults with classic Congenital Adrenal Hyperplasia (CAH) caused by 21-hydroxylase deficiency (21-OHD). Researchers want to learn how well atumelnant helps control CAH symptoms, how the body processes the drug (pharmacokinetics or PK), and whether it produces the expected effects in the body (pharmacodynamics or PD). The study will also evaluate whether participants may be able to reduce their steroid (glucocorticoid) dose while taking atumelnant.

The upfront treatment for astrocytoma is surgery, radiation therapy, and chemotherapy. There is currently no standard therapy for when the astrocytoma has returned (recurs) or re-grows (progresses). This study uses the investigational drug FT536, a cell therapy that stimulates the immune system into action to treat astrocytoma that has returned or regrown. FT536 is a type of cell therapy made up of “natural killer” or NK cells, a type of immune blood cell that are known to attack cancer cells. The primary purpose of this study is to identify a safe dose of FT536 cells when given as an intratumoral injection and to identify the side effects of treatment with FT536. Another purpose of this study is to understand how FT536 alters the immune system in the brain and body.

To evaluate the safety of the EN-374 treatment regimen (HSC mobilization, immune prophylaxis, EN-374 dose and administration, and enrichment of HSCs with O6BG/TMZ).

This study is testing an compound called TP-3654, which is an investigational product being developed for Myelofibrosis.

This study aims to evaluate the ROCA2 algorithm, which calculates a woman's risk of having ovarian cancer based on age and biomarkers, for the early detection of ovarian cancer in a low risk cohort of women.