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Anemia, Sickle Cell clinical trials

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NCT ID: NCT02261480 Completed - Sickle Cell Disease Clinical Trials

A New Reagent Assay Examining Natural Parvovirus B19 Infection in Sickle Cell Disease

Start date: October 2014
Phase: N/A
Study type: Observational

Parvovirus B19 is a small virus that is the cause of "fifth" disease, a common infection in childhood. In people with sickle cell disease (SCD), parvovirus B19 infection causes the bone marrow to stop producing red blood cells temporarily, which can be life-threatening. A novel vaccine is currently in development for children with SCD. This study is the first step within a larger parvovirus B19 multi-institutional project that will help develop this new vaccine, as it will define the value and utility of using a novel assay for measurement of parvovirus-specific antibodies. The main objective is to investigate the relationship between the newly developed VP1u ELISA assay and the gold standard neutralization assay for parvovirus B19 infection. The most accurate test, called a neutralizing antibody assay, to see if a person has had or currently has the infection is very complex and expensive and would be very difficult to use in a large research study to test the new vaccine. A new and simpler test has developed. The main goal of this study, iSCREEN, is to find out if this new test works. There will be distinct labs performing the VP1u ELISA and the neutralization assays and the respective laboratories will not have access to each other's results for individual subjects. The VP1u ELISA will be performed at St. Jude Children's Research Hospital. Neutralization assays will be conducted at the National Heart, Lung and Blood Institute.

NCT ID: NCT02258997 Completed - Sickle Cell Anemia Clinical Trials

Hepcidin Levels in Sickle Cell Disease (SCD)

Start date: March 2014
Phase: N/A
Study type: Observational

The investigators propose that patients with HbSβ-thalassemia have lower levels of hepcidin and higher levels of GDF-15 than HbSS patients during the non-crisis, "steady states." In addition, the investigators propose that when controlled for RBC transfusion, patients with HbSβ-thalassemia will have higher levels of storage iron (based on serum ferritin). Participants: Total number of subjects is 42 - 21 subjects with HbSS, and 21 subjects with HbSβ-thalassemia ). Procedures (methods): Eligible subjects with documented SCD (HbSS, HbS-β 0-thalassemia or HbS-β+-thalassemia) followed at the University of North Carolina (UNC) Comprehensive Sickle Cell Program will be evaluated in this single-center, prospective, cross-sectional study. The patients will be screened for eligibility at the time of a routine sickle cell clinic visit. Patients' data will be obtained in person at the time of evaluation and through review of their medical records. Investigators will obtain information on SCD-related clinical complications and obtain an estimate of the number of lifetime RBC transfusions. Blood samples will be obtained for laboratory tests. Plasma samples for hepcidin, growth differentiation factor 15 (GDF -15), and high-sensitivity CRP will be stored at -80 degrees Celsius until analysis. Other routine laboratory studies including complete blood count (CBC) with differential and reticulocyte count, serum iron profile and ferritin, and liver function tests will be performed at the clinical laboratories of UNC Hospitals.The subjects will have 30 ml. of blood drawn for this research study. Females of child bearing potential will have a urine pregnancy test at the time of the study.

NCT ID: NCT02242058 Completed - Clinical trials for SCD With Severe Phenotype (HbSS, HbSß0 Thalassemia, HbSOARab)

QST-Pupillometry in Sickle Cell Disease Patients

QST
Start date: August 2013
Phase:
Study type: Observational

There has been little progress for effective treatment of pain in sickle cell disease (SCD) patients. Many organizations have recognized that understanding the causes and reducing the burden of pain in SCD is critical in order to improve the quality of life in SCD patients. As patients with SCD face the challenge of living with both acute and chronic pain which is often improperly treated, our translational and interdisciplinary project aims to identify objective measures of pain sensitivity and its biochemical and genetic correlates. We hypothesize that SCD patients will have decreased tolerance to thermal and electrical stimuli.

NCT ID: NCT02239016 Completed - Sickle Cell Disease Clinical Trials

Screening Patients With Sickle Cell Disease for Kidney Damage

Start date: April 2009
Phase:
Study type: Observational

This study aims to study the temporal course of sickle nephropathy and assess novel biomarkers that can predict patients prone to nephropathy.

NCT ID: NCT02227472 Completed - Sickle Cell Disease Clinical Trials

Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease

Start date: September 8, 2014
Phase:
Study type: Observational

Children with sickle cell disease (SCD) are at risk for neurobehavioral problems because of the impact the disease can have on the central nervous system. Specific impairments in working memory are particularly prevalent in school-aged children with SCD. Working memory is more strongly associated with school readiness and academic success than intellectual ability in the general population. The adverse effects of low socioeconomic status (SES) and poverty on cognition and neurodevelopment emerge early, before children have entered formal education. In addition, they affect language and executive function skills (e.g., working memory) more than other skills. SES is a proxy variable for other risk factors. Higher SES is associated with less parental stress, more supportive parenting practices, and better cognitive stimulation based on the availability of books, computers, and outings. PRIMARY OBJECTIVE: - To examine working memory and school readiness in young children with sickle cell disease in comparison to demographically matched control children without sickle cell disease. SECONDARY OBJECTIVE: - To examine the relationships of family/environmental factors (caregiver stress, parental responsiveness, and cognitive stimulation in the home) and disease severity to working memory and school readiness skills in preschool-aged children with SCD.

NCT ID: NCT02225145 Completed - Aplastic Anemia Clinical Trials

Fertility Preservation in Women Who Will Have Gonadotoxic Therapy or Hematopoetic Stem Cell Transplantation, and in Women With Sickle Cell Disease

Start date: August 15, 2014
Phase:
Study type: Observational

Background: - Some treatments for cancer or other diseases can lead to infertility in women. These treatments include chemotherapy, some stem cell transplants, and pelvic radiotherapy. They are called gonadotoxic therapies. Women can now have their eggs frozen before they have these treatments. This may allow them to get pregnant later. Researchers want to learn more about this technology and processes. Objectives: - To provide egg freezing for women having gonadotoxic therapies at NIH. To learn more about the effects of these therapies. Eligibility: - Women at least 18 years old who are past puberty and before menopause. They must be scheduled to have gonadotoxic therapies. Design: - Participants will be screened with medical history and blood and hormone tests. They will also have a physical exam and transvaginal ultrasound. - Ovary stimulation: participants will have medications injected under the skin. These increase the chance of fertility. This phase will take about 8 20 days. Participants will have blood drawn and transvaginal ultrasound daily or every other day. Some participants will also have blood thinner injected daily. - Egg retrieval: participants will check in to the hospital. Eggs will be removed with a needle during a short surgery. Participants will be awake but sedated. - Participants may stay overnight in the hospital. - They will return every 1 3 days for 1 3 weeks for blood tests. - Mature eggs will be frozen after egg retrieval and immature eggs (which cannot be fertilized for clinical use) will be used for research. Participants can use their eggs in the future at outside, private fertility clinics to try to become pregnant. If the eggs are stored for more than 5 years, participants must pay for storage.

NCT ID: NCT02225132 Completed - Sickle Cell Disease Clinical Trials

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

Start date: August 23, 2014
Phase: Phase 1/Phase 2
Study type: Interventional

Background: - Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood. This will help them learn about the right dosage of HU to give to people with SCD. Objective: - To improve hydroxyurea dosing in people with SCD. Eligibility: - People age 15 or older with homozygous SCD (HbSS). Design: - Participants will be screened with medical history, physical exam, medication review, and blood and urine tests. - Participants will be in the study for about 15 months. - First 3 months: monthly study visits with blood and urine tests. - After 3 months: participants will take HU as a capsule by mouth. If you are already taking HU, your dose will be increased. - Within a month of starting or increasing HU: participants will keep a daily pain diary for 2 weeks. They will have an echocardiogram (ultrasound) of the heart, a 6-minute walk test. They will complete a quality-of-life questionnaire. - Participants will visit every month until they reach their highest tolerated dose of HU. They may need to come as often as every week sometimes to closely monitor their blood counts. Then they will alternate a phone call one month and a visit the next. At the visits, participants will bring their pill bottle, answer questions about side effects, and have blood tests. - Every 2 months, participants will have a medical history, physical exam, and blood tests. - Every 4 months, participants will have blood and urine tests. They will also complete another 2-week pain diary and quality-of-life questionnaire. - About 12 months after starting or increasing HU, participants will have blood tests, an echocardiogram, and a 6-minute walk test.

NCT ID: NCT02222246 Completed - Sickle Cell Disease Clinical Trials

Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease

Start date: March 15, 2015
Phase: Phase 4
Study type: Interventional

The goal of this pilot study is to improve emergency department (ED) pain management for adults with sickle cell disease. Sickle cell disease (SCD) is the most common genetic disorder in the United States, and occurs primarily among African Americans. Management of painful episodes associated with SCD, referred to as vaso-occlusive crises (VOC), is the most common reason for SCD patients to visit the ED. Currently, there is no standard approach to managing VOC pain in the ED that is widely accepted and used, and pain management for vaso-occlusive crisis in persons with SCD is very different between providers and not based on research. Many times, patients who come to the ED with sickle cell pain feel that they do not receive adequate pain control. If EDs could provide efficient, effective, safe, patient-centered analgesic management, it may be possible to improve pain management for adults with SCD experiencing a VOC. Guidelines for treating vaso-occlusive crises caused by sickle cell disease will soon be published by the National Heart, Lung and Blood Institute of the National Institutes of Health. These guidelines recommend patient-specific pain treatment protocols or a standardized pain management protocol for SCD when a patient does not already have a pain treatment protocol designed for them. The purpose of this pilot study is to compare these two ways to treat vaso-occlusive pain in the ED for adults with sickle cell disease, and to determine if a large randomized controlled trial is feasible and required.

NCT ID: NCT02214121 Completed - Clinical trials for Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease

A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease

HESTIA 1
Start date: September 11, 2014
Phase: Phase 2
Study type: Interventional

The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with sickle cell disease

NCT ID: NCT02212691 Completed - Sickle Cell Disease Clinical Trials

Functional Neuroimaging of Pain Using EEG and fMRI

Start date: January 2012
Phase:
Study type: Observational

The purpose of this research is to use non-invasive imaging technologies to study how the human brain processes pain. The investigators will use contact heat to induce pain and record data scalp EEG and functional magnetic resonance imaging (fMRI). What the investigators learn from this study will help us gain insights in pain management with broad socioeconomic impacts