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Sickle Cell Disease clinical trials

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NCT ID: NCT06301893 Recruiting - Sickle Cell Disease Clinical Trials

Uganda Sickle Surveillance Study (US-3)

Start date: September 7, 2013
Phase:
Study type: Observational [Patient Registry]

It is estimated that over 250,000 babies are born with sickle cell disease (SCD) annually in sub-Saharan Africa, and only 10% - 50% of them survive beyond five years of age. Data describing the magnitude of the sickle cell problem are lacking in most African countries. The available data on prevalence were mainly from older studies and small numbers of hospitalized patients. In Uganda, approximately 25,000 children are born with SCD but 70-80% die before their 5th birthday. Lehmann and Raper found 'sicklaemia' prevalence of 0.8% and 45% in the Sebei and Bambaa ethnic groups, respectively. A recent study found a SCT and SCD prevalence of 3% - 19% and 0% - 3%, respectively but this study addressed only 5 of Uganda's 111 districts and used a small convenience sample of children aged 6 - 60 months. The objective of this study is to determine the prevalence and map out the burden of SCT and SCD in Uganda.

NCT ID: NCT06213402 Recruiting - Sickle Cell Disease Clinical Trials

RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)

RADeep
Start date: November 30, 2021
Phase:
Study type: Observational [Patient Registry]

Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation. Different medical entities classified as RADs by ORPHA classification are most of them chronic life threating disorders with many unmet needs for their proper clinical management creating an impact on European health systems. RADs present diagnostic challenges and their appropriate management requires from specialised multidisciplinary teams in Centers of expertise. Although there are some examples of well-established national registries on RADs in EU, the lack of recommendations for Rare disease registries implementation and the lack of standards for interoperability has led to the fragmentation or unavailability of data on prevalence, survival, main clinical manifestations or treatments in most of the European countries.

NCT ID: NCT06198712 Recruiting - Sickle Cell Disease Clinical Trials

A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

Start date: February 7, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Eligible participants who enter the study will start a 96-week treatment period. At the end of the 96 weeks, participants will have an end of study visit that occurs 4 weeks later. The participants will receive etavopivat every day throughout the treatment period.

NCT ID: NCT06171217 Recruiting - Sickle Cell Disease Clinical Trials

Realizing Effectiveness Across Continents With Hydroxyurea

REACH
Start date: October 27, 2023
Phase: Phase 2
Study type: Interventional

REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for children with confirmed SCA between 3 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.

NCT ID: NCT06158945 Recruiting - Sickle Cell Disease Clinical Trials

The Role of Endothelin 1 as a Marker of Renal Impairment in Sickle Cell Disease

Start date: October 24, 2023
Phase:
Study type: Observational

Sickle cell disease (SCD) refers to a group of hemoglobinopathies that include mutations in the gene encoding the beta subunit of hemoglobin. Within the umbrella of SCD, many subgroups exist, namely sickle cell anemia (SCA), hemoglobin SC disease (HbSC), and hemoglobin sickle-beta-thalassemia (beta-thalassemia positive or beta-thalassemia negative). Several other minor variants within the group of SCDs also, albeit not as common as the varieties mentioned above. It is essential to mention the sickle cell trait (HbAS), which carries a heterozygous mutation and seldom presents clinical signs or symptoms. Sickle cell anemia is the most common form of SCD

NCT ID: NCT06145282 Recruiting - Sickle Cell Disease Clinical Trials

Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function

Start date: December 28, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

Background: Sickle cell disease (SCD) is a genetic disorder where red blood cells, that carry oxygen, are stiff and become stuck in small blood vessels. As a result, affected patients can experience severe pain and serious organ damage. SCD can be cured with a hematopoietic cell transplant (HCT), that is, when they receive blood stem cells from a family donor. But HCT can also have serious side effects, especially in people with organ damage. Researchers want to find ways to make HCT safer for everyone. Objective: To test a new combination of drugs (briquilimab, abatacept, and alemtuzumab), used along with radiation, in people undergoing HCT for SCD. Eligibility: People aged 16 and older with SCD. They must be eligible for HCT and have a family member who is a good donor match. Donors must be aged 4 and older. Design: Participants with SCD will be screened. They will have blood tests and tests of organs including their heart and lung function. Donors will have blood drawn. Participants with SCD will have a tube inserted into a blood vessel in their chest (intravenously). This line will remain in place up to 2 months; it will be used to draw blood and administer the donor cells and other medications. Briquilimab will be administered intravenously 1 time, along with other drugs used to prepare for HCT. Participants will receive abatacept 6 times, from just before they receive their donor cells until 6 months after. Participants will undergo radiation therapy and take other drugs that are standard for HCT. Most HCT recipients remain in the hospital for about 30 days after HCT. Follow-up visits will continue for 5 years.

NCT ID: NCT06110754 Recruiting - Sickle Cell Disease Clinical Trials

Development and Adaptation of I-STRONG for SCD

Start date: October 24, 2023
Phase: N/A
Study type: Interventional

This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.

NCT ID: NCT06074198 Recruiting - Sickle Cell Disease Clinical Trials

Sickle Cell Children's Exercise Study (SuCCESs)

SuCCESs
Start date: November 3, 2023
Phase: N/A
Study type: Interventional

The Sickle Cell Children's Exercise Study (SuCCESs) will explore the feasibility and effects of a moderate intensity strengthening, balance, speed, and agility intervention program in children with sickle cell disease.

NCT ID: NCT06072287 Recruiting - Obesity Clinical Trials

The Living With a Long-Term Condition Study

LTC
Start date: June 28, 2023
Phase:
Study type: Observational

Psychological distress (anxiety and depression) is common in and experienced differently by people living with long-term health conditions (LTCs). Being able to measure whether psychological distress is related to living with a LTC would allow researchers and clinicians to provide interventions specifically tailored to the challenges of living with a LTC and therefore provide the most appropriate support for these patients. Such a measure would also be useful in research to identify the presence of illness-related distress in different patient groups. This project will therefore create a new measure of illness-related distress that has applications for both research and clinical practice. This will involve the psychometric validation of the new illness-related distress measure to test how valid and reliable the measure is. The aim of the project is to provide initial validation of the Illness Related Distress Scale in a community sample, recruited through online platforms. The objective of the study is to gather initial validity and reliability data for the scale.

NCT ID: NCT06035939 Recruiting - Sickle Cell Disease Clinical Trials

SMYLS Multi-site Trial

Start date: March 12, 2024
Phase: N/A
Study type: Interventional

The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms.