
Donor Stem Cell Transplant in Treating Patients With Myeloid Cancer or Other Disease
LeukemiaMyelodysplastic SyndromesRATIONALE: Giving total-body irradiation and chemotherapy, such as fludarabine and thiotepa, before a donor stem cell transplant helps stop the growth of cancer or abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin and removing the T cells from the donor cells before transplant may stop this from happening. PURPOSE: This phase II trial is studying how well a donor stem cell transplant works in treating patients with myeloid cancer or other disease.

Azacitidine, Darbepoetin Alfa, and Erythropoietin and Filgastrim (G-CSF) in Treating Patients With...
LeukemiaMyelodysplastic SyndromesRATIONALE: Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. Colony-stimulating factors, such as darbepoetin alfa and G-CSF, may increase the number of red blood cells and white blood cells found in bone marrow or peripheral blood and may help the immune system recover from the side effects of chemotherapy. Giving azacitidine together with darbepoetin alfa and G-CSF may be an effective treatment for myelodysplastic syndromes. PURPOSE: This clinical trial is studying how well giving azacitidine together with darbepoetin alfa and G-CSF works in treating patients with myelodysplastic syndromes.

Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)
Prader-Willi SyndromeThe purpose of this study is to investigate over a 6 month period the effect of octreotide therapy on food intake, sense of hunger, body weight, body composition, efficiency of burning calories, biomarkers of weight regulation and growth hormone markers in children and young Adults with Prader-Willi Syndrome(PWS).

Phase 2, Open-Label, Multi-Dose Study of Panhematin in Patients With MDS
Myelodysplastic SyndromeThis is a Phase II, open-label clinical trial examining the role of Panhematin® in patients with MDS. The objective of this study is to evaluate the safety and efficacy of Panhematin® (hematin for injection) in the treatment of adult patients (≥ 18 years of age) with low-risk MDS. The study will be conducted on an outpatient basis and will consist of the following: A Screening Period (within 28 days of the Day 1) Screening bone marrow aspiration and biopsy up to 60 days prior to receiving study medication An 8-week Treatment Period (Days 1 through 4 of Week 1, and weekly visits during Weeks 2 through 8); partial and complete responders in any of the three cell lines may continue treatment for an additional 4 weeks A 6-month Post treatment Follow-up Period (monthly clinic visits during Weeks 12 40)

Systemic Steroids Plus Antibiotics in Sleep Apnea Syndrome in Children
Obstructive Sleep Apnea SyndromeThe purpose of this study is to investigate the effect of treatment with a steroid and antibiotic on the size of the tonsils and symptoms of children with OSAS.

Donor Peripheral Stem Cell Transplant and Donor Natural Killer Cell Transplant After Total-Body...
Graft Versus Host DiseaseLeukemia1 moreRATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell and donor natural killer cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Removing the T cells from the donor cells before transplant may stop this from happening. PURPOSE: This phase II trial is studying how well giving a donor peripheral stem cell transplant and a donor natural killer cell transplant after total-body irradiation, thiotepa, fludarabine, and muromonab-CD3 works in treating patients with leukemia or other blood diseases.

A Study of CC-90002 in Subjects With Acute Myeloid Leukemia (AML) and High-risk Myelodysplastic...
LeukemiaMyeloid2 moreStudy CC-90002-AML-001 is an open-label, Phase 1 dose escalation (Part A) and expansion (Part B), clinical study of CC-90002, administered by intravenous (IV) infusion, in subjects with relapsed and/or primary refractory AML and high-risk MDS. The study will explore escalating doses of CC-90002 using a 3 + 3 dose escalation design in Part A, followed by dose expansion in Part B. The primary objective is to determine the safety and tolerability of CC-90002 and also to define the non-tolerated dose (NTD), the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D) of CC-90002.

UCB Proof of Concept Study in Patients With Primary Sjögren's Syndrome
Primary Sjögren's SyndromeThis is a Phase 2, multicenter, double-blind, placebo-controlled, 12-week proof-of-concept study to assess the efficacy, safety, and tolerability of UCB5857 in subjects with primary Sjögren's Syndrome (pSS). The primary objective of this study is to evaluate the efficacy on overall disease activity and safety of UCB5857 added to current treatment relative to placebo in subjects with pSS.

Ofatumumab in Children With Drug Resistant Idiopathic Nephrotic Syndrome
Nephrotic SyndromeDouble-blind, two-parallel-arm, placebo-controlled randomized clinical trial testing the superiority of Ofatumumab versus placebo in the treatment of children with DR-INS. Participants will be stratified according to eGFR at enrollment. Eligible participants will enter a 3-months run-in period, during which instructions on urine collection and dipstick readings will be carefully reviewed, compliance assessed and any immunosuppressive therapies withdrawn according to the following schemes: prednisone will be tapered off by 0.3 mg/kg per week until complete withdrawal; calcineurin inhibitors and mofetile mycophenolate will be decreased by 50% and withdrawn after 2 additional weeks In order to minimize the risk of complications of uncontrolled INS a treatment with ACE-inhibitor at 6 mg/m2 will be maintained or started in all patients. After run-in period, children will be randomized to the intervention arm (Ofatumumab) or comparator arm (placebo). Randomization will be stratified by eGFR at randomization: ≥90 and <90 ml/min/1.73 m2. All patients will be followed up to 12 months and they will leave the study at time of relapse. Relapse will be defined as uPCR ≥2000 mg/g (≥200 mg/mmol) or ≥ 3+ protein on urine dipstick for 3 consecutive days.

A Pilot Study of Cognitive-Behaviour Therapy for Irritable Bowel Syndrome and the Gut Microbiome...
Irritable Bowel SyndromeThis study evaluates the effectiveness of a group cognitive behaviour therapy for Irritable Bowel Syndrome (IBS) in decreasing symptoms of IBS, improving quality of life, and normalizing the gut microbiome. Patients will complete an 11-week group cognitive behaviour therapy for IBS within 1 month of being deemed eligible for the study.