Active clinical trials for "Syndrome"

The purpose of this study is to determine the activity of SY-1425 in relapsed/refractory acute myeloid leukemia (AML) patients (SY-1425 administered as a monotherapy or in combination with azacitidine), relapsed/refractory higher-risk myelodysplastic syndrome (MDS) patients (SY-1425 administered as a monotherapy or in combination with daratumumab), newly diagnosed treatment naïve AML patients who are unlikely to tolerate standard intensive chemotherapy (SY-1425 administered as a monotherapy or in combination with azacitidine), or lower-risk myelodysplastic syndrome (MDS) patients (SY-1425 administered as a monotherapy).

This phase IIa trial studies how well guadecitabine works in treating patients with acute myelogenous leukemia and myelodysplastic syndrome that has returned after a period of improvement after allogeneic stem cell transplant. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving guadecitabine before the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them. Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.

This phase II trial studies how well guadecitabine works in treating patients with myelodysplastic syndromes that are at higher risk for becoming acute myeloid leukemia. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

This study is evaluating the safety and efficacy of a new BTK inhibitor, acalabrutinib, for the treatment of chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL).

EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.

This is a Randomized Controlled Trial (RCT) regarding conservative treatment of Patellofemoral Pain Syndrome (PFPS). Patellofemoral Pain Syndrome (PFPS) is a very common cause of knee pain in young active adults with a high rate of recurrent and/or chronic occurrence. PFPS is notoriously difficult to treat and has been referred to as "one of the most vexatious clinical challenges in rehabilitative medicine". Its etiology is unclear but is commonly thought to be related to pathomechanics in the patellofemoral joint (PFJ). There are many factors that can influence PFJ mechanics. Among these, quadriceps strength and timing has been shown to be important. As such, treatment of PFPS has traditionally been based on correction of pathomechanics through influencing quadriceps strength and timing. However, a growing body of evidence is revealing the importance of strength and control of hip abduction and external rotation in PFPS. Hip strength in ab/adduction and rotation is thought to influence femoral positioning in the patellofemoral joint, thereby affecting PFJ mechanics. Several cohort and smaller RCT studies within the last 7 years have shown that additional exercises for hip strength and control give an improved effect in pain and function compared with quadriceps based training alone. A smaller RCT from 2012 compared isolated hip strengthening exercises to a control group and found surprisingly good results on pain in function in the hip strengthening group. The investigators plan a RCT in which isolated hip strengthening will be compared to traditional quadriceps training and a control group which will receive no structured training. Primary outcomes will be pain and function. This high-quality study will include 40-50 patients in each group, making it one of the largest of its kind on conservative treatment for PFPS. In contrast to the vast majority of studies of this type, this study will also include men, which will potentially help to fill a significant gap in the literature on this subject. The investigators study will therefore be an important contribution to elucidating the etiology of PFPS and improving treatment options for both men and women in the future. As well, the role of psychometric parameters will be examined and a standardized clinical test for hip abduction endurance will be developed. Follow-up at 3 months and 12 months is completed and published. A 5-year follow-up of the same patients is underway.

Evaluation of the Efficacy and Safety of Oral Azacitidine plus Best Supportive care versus Placebo and Best Supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to International Prognostic Scoring System (IPSS) lower risk myelodysplastic syndromes (MDS).

Patients will be receiving a stem cell transplant as treatment for their disease. As part of the stem cell transplant, patients will be given very strong doses of chemotherapy, which will kill all their existing stem cells. A close relative of the patient will be identified, whose stem cells are not a perfect match for the patient's, but can be used. This type of transplant is called "allogeneic", meaning that the cells are from a donor. With this type of donor who is not a perfect match, there is typically an increased risk of developing GvHD, and a longer delay in the recovery of the immune system. GvHD is a serious and sometimes fatal side-effect of stem cell transplant. GvHD occurs when the new donor cells (graft) recognize that the body tissues of the patient (host) are different from those of the donor. In this study, investigators are trying to see whether they can make special T cells in the laboratory that can be given to the patient to help their immune system recover faster. As a safety measure, we want to "program" the T cells so that if, after they have been given to the patient, they start to cause GvHD, we can destroy them ("suicide gene"). Investigators will obtain T cells from a donor, culture them in the laboratory, and then introduce the "suicide gene" which makes the cells sensitive to a specific drug called AP1903. If the specially modified T cells begin to cause GvHD, the investigators can kill the cells by administering AP1903 to the patient. We have had encouraging results in a previous study regarding the effective elimination of T cells causing GvHD, while sparing a sufficient number of T cells to fight infection and potentially cancer. More specifically, T cells made to carry a gene called iCasp9 can be killed when they encounter the drug AP1903. To get the iCasp9 gene into T cells, we insert it using a virus called a retrovirus that has been made for this study. The AP1903 that will be used to "activate" the iCasp9 is an experimental drug that has been tested in a study in normal donors with no bad side-effects. We hope we can use this drug to kill the T cells. The major purpose of this study is to find a safe and effective dose of "iCasp9" T cells that can be given to patients who receive an allogeneic stem cell transplant. Another important purpose of this study is to find out whether these special T cells can help the patient's immune system recover faster after the transplant than they would have otherwise.

The purpose of this study is to evaluate the safety, effectiveness and pharmacokinetics of a study drug called Faslodex (fulvestrant) in the treatment of progressive precocious puberty (early puberty) in girls with McCune-Albright syndrome (MAS)

The purpose of this study is to determine if budesonide prophylaxis starting day 5 after transplant reduces engraftment fever in autologous and allogeneic stem cell transplant recipients.