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195 active trials for Sickle Cell Disease

Sickle Cell Uric Acid (SCUA) - Cohort Repository

The purpose of this research is to study the causes of Sickle Cell kidney disease, as well as to collect and store samples and information about people with Sickle Cell Disease.

Start: July 2019

Medication Adherence and Non-adherence in Adults With Rare Disease

The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease, and to identify the top patient-reported reasons for non-adherence. Internal reliability of the MAR-Scale will also be assessed in each condition.

Start: January 2021

Utilizing A Single Session Problem-Solving Intervention With Caregivers of Pediatric Patients Receiving Chronic Transfusion to Treat Sickle Cell Disease

This study seeks to utilize an innovative approach of a single session problem-solving intervention to address psychosocial factors affecting patient outcomes within the pediatric sickle cell population. The study will be a randomized control trial of a single session problem-solving intervention. This original research will investigate the feasibility and efficacy of utilizing a single-session problem-solving intervention to address problems affecting children and families receiving chronic blood transfusions for sickle cell disease in order to: 1) contribute to literature related to single session problem solving interventions with the chronic transfusion sickle cell population and 2) identify a model of sustainable care that reduces the burden of a multiple session intervention and increases access to services. Additionally, this research aims to provide relatively low burden and potentially highly effective intervention into regular care for this population in order to evaluate the feasibility of integrating a single-session problem solving intervention into routine clinical flow, thereby addressing needs more systematically that have been identified by families. Further, this research aims to identify potential utility of medical providers being trained on providing the intervention, which could be part of a future study.

Start: December 2020

Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers

The investigators propose a Phase II study, prospective and placebo controlled, of a topical cream containing sodium nitrite compared to the current standard of care. Sodium nitrite is a local donor of nitric oxide, which is known to improve blood flow and decrease bacterial load in the ulcer bed. The primary objectives are to evaluate the safety of topical sodium nitrite cream treatment in patients with sickle cell disease and chronic leg ulcers and to determine its effectiveness in accelerating the healing process and decreasing the pain associated with ulceration. Potential benefit will be a durable resolution or improvement of the leg ulcer and its associated pain. Possible side effects include decreased blood pressure and methemoglobinemia, secondary to sodium nitrite absorption through the ulcerated skin. Funding source FDA OOPD.

Start: April 2018

Outcomes Mandate National Integration With Cannabis as Medicine for Prevention and Treatment of COVID-19

This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions.

Start: December 2018

Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia

MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients with sickle cell disease. The goal of this research is to study if MitoQ, a molecule that works as an antioxidant by removing potentially damaging agents in a living organism, improves platelet function in patients with sickle cell disease (SCD).

Start: March 2020

Sleep Apnea in Sickle Cell Disease

Despite the fact that obstructive sleep apnoea (OSA) is highly prevalent in the sickle cell population, studies focusing on the associations of the two diseases and their common pathophysiological mechanisms are scarce. OSA is one of the most common conditions responsible for hemoglobin desaturation. The nocturnal hemoglobin desaturation occurring in some sickle cell disease (SCD) patients with OSA could trigger hemoglobin S polymerization and red blood cell (RBC) sickling, leading to further blood rheological alterations, hence increasing the risks for VOC. Moreover, OSA has been demonstrated to increase oxidative stress and inflammation in non Sickle Cell Disease (SCD) patients, which, in SCD patients, could increase the risk for complications. Finally, OSA is accompanied by impaired vascular function and autonomic nervous system dysfunction in the general population. Indeed, the presence of OSA in SCD could increase the clinical severity of patients and the frequency of VOC.

Start: May 2018

Inhaled Mometasone to Promote Reduction in Vasoocclusive Events 2

The study team proposes a triple-blind, placebo-controlled, phase II clinical trial of once-daily inhaled mometasone for 48 weeks (with 4-week washout at study completion) in individuals with Sickle Cell Disease (SCD) who report episodic cough or wheeze (ECW) but do not have asthma. Patients will be recruited from and followed in SCD clinics at participating sites. The primary endpoint will be a reduction in sVCAM level of 20% or more in comparison to placebo.

Start: November 2018

Links Between Cognitive Functions and Clinical, Biological and Neuroradiological Outcomes in Adults With Sickle Cell Disease.

Sickle cell disease (SCD) is an inherited blood disorder. Symptoms include acute and chronic complications. Due to progress in SCD care, patients with SCD are living longer than before and we focus more attention in chronic complications. Children with SCD experience worse cognitive functions than healthy children, and fewer is known about cognitive functions in adults. Studies suggest lower cognitive performance in SCD, mostly in executive functions and processing speed, but the biological and anatomical substrates of cognitive decline are not yet well established in SCD. Often times, cognitive impairments and cerebral disorders are not diagnosed and treated in adults with SCD. The main objective of this study is to propose a deep neuropsychological assessment in adults with SCD and cognitive complaints and to highlight links between cognitive functions and clinical, biological and neuroradiological markers. The hypothesis of this study is that cognitive functions are associated with severity of the SCD, with bood abnormalities, with MRI markers and Transcranial Doppler (TCD) markers of cerebrovascular disease. The secondary objective of this study is to validate a brief cognitive assessment tool (BEARNI tool) in adults with SCD. This study is an observational cross-sectional study that will enroll adults with SCD and cognitive complaint.

Start: October 2020

A Study Aimed to Assess the Needs of Subjects With Sickle Cell Disease and Healthcare Professionals Managing Sickle Cell Disease Patients in Selected Nigerian Centres

This pilot research is aimed to assess the needs of patients and health workers involved in Sickle Cell Disease (SCD) management in Nigeria. To achieve this, a questionnaire will be administered to SCD patients or parents of children affected by SCD. Another questionnaire will be administered to doctors and nurses working with SCD patients. A focus group discussion with patients/parents willing to participate will be also scheduled. Participants from the following centres will be involved: Barau Dikko Teaching Hospital Kaduna State University, Ahmadu Bello University Teaching Hospital Zaria, National Hospital Abuja, Federal Medical Centre Katsina. Data will be qualitatively and quantitatively analysed and presented as aggregated data. Consent from all the study participants will be sought. Questionnaires will be coded and no personal data will be disclosed to authorised third parties.

Start: September 2020

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, TEPA. The goal of this study is to determine what causes some children to have different drug concentrations of thiotepa and TEPA in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that certain clinical and genetic factors cause changes in thiotepa and TEPA drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

Start: January 2018

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and individual factors cause changes in clofarabine and fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

Start: February 2016

Realizing Effectiveness Across Continents With Hydroxyurea (REACH)

REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for children with confirmed SCA between 12 months and 10 years of age. The short-term goal is to obtain critical pilot data regarding the feasibility, safety, and benefit of hydroxyurea for children with SCA in multiple distinct research settings in Africa. Based on that information, the longer-term goal is to make hydroxyurea more widely available for children with SCA in Africa, particularly those identified with SCA through expanded newborn screening programs.

Start: June 2014

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCR??+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).

Start: May 2020

iCanCope With Sickle Cell Pain

The project will test a tailored web and smartphone-based application (iCanCope with SCD) to improve pain self-management and functioning in youth (aged 12-18) with sickle cell disease. The program will include goal setting, peer-based social support, and pain self-management training. The investigators will determine initial program effectiveness through a pilot three-site randomized controlled trial in 160 youth randomized to treatment compared to attention control.

Start: January 2018

Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment

Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP). Those subjects who pass Screening will enter into the 2-week Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the 2-week Run-in Period will be randomized into active and control groups (2 active to 1 placebo) and enter the 12-week Treatment Period. At each visit during the Treatment Period, the target ulcer will be measured by digital photographic planimetry, the Principal Investigator will assess the wound qualitative attributes, and the DIDP (or placebo patch) will be placed as the primary wound dressing. At each visit the subject will also receive/review a daily diary to document pain , study drug compliance, and analgesic use.

Start: July 2020

Using Patient-Centered Guidelines in a Technology Platform to Improve Health Care in Adults With Sickle Cell Disease

SCD is an inherited disorder of hemoglobin that affects over 100,000 Americans, most of whom live in low-resourced neighborhoods. Acute SCD complications result in 230,000 emergency department visits and $1.5 billion annually in acute-care expenditures. Prior research indicates that increased disease-specific knowledge correlates with improved clinical outcomes in SCD. Thus, targeting strategies to improve disease-specific knowledge is a high priority in the care of individuals with SCD. Significant evidence describes how educational materials, including online educational programs, can be used to increase disease-specific knowledge. In this study, the investigators will evaluate a mobile phone technology intervention based on the prior evidence that technologies can improve SCD-specific knowledge.

Start: July 2018

Assessing the Safety of Buprenorphine in People With Sickle Cell Disease

This study will assess the safety of changing pain medications (opioids) adult sickle cell patients take to another type of medication therapy (buprenorphine). Patients will be asked questions about their quality of life. Other tools for assessment will also be administered.

Start: August 2018

PINPOINT: Gaming Technology for SCD Pain

Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking. Research shows that interactive gaming technology can enhance adolescents' learning, and can be especially effective in delivering health-related messages and tools to improve self-care. Pinpoint is an interactive gaming app that will be tested in a Phase II project to determine whether the app assists SCD teens with improving their communication and identification skills for pain self-report.

Start: September 2018

Microvascular Blood Flow in Sickle Cell Anemia

Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. Sickle-shaped cells can cause problems by getting stuck in blood vessels, blocking blood flow, and can cause inflammation and injury to important body parts. There are no specific treatments that improve this condition and promote blood flow hindered by sickle cell blockages. Another big challenge in managing sickle cell disease is that there are no good measures to determine changes and improvements in blood flow. Contrast-enhanced ultrasound is a technique currently used to detect blood flow in the heart, muscles, and other organs. It is extremely sensitive and can detect blood flow in the smallest of blood vessels. It would be very useful in helping healthcare providers know whether treatment strategies are improving blood flow during sickle cell blockages. The hypothesis is that contrast-enhanced ultrasound will be a feasible tool for determining changes in blood flow of subjects with sickle cell disease.

Start: July 2012