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Sickle Beta Zero Thalassemia clinical trials

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NCT ID: NCT05675436 Enrolling by invitation - Sickle Cell Anemia Clinical Trials

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

Start date: May 9, 2024
Phase:
Study type: Observational

Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys. SCD can lead to serious illness and death. Treatments such as bone marrow transplants and gene therapies can cure SCD, but they are not widely available. Current drug treatments for SCD are not always effective. This natural history study will examine how a study drug (mitapivat) affects red blood cells in people with SCD. Objective: To learn how mitapivat affects red blood cells in people with SCD. Eligibility: People with SCD who are enrolled in the parent study, NIH protocol IRB001565-H. Design: Procedures for this study will be done during visits already scheduled for the parent study. Participants will have additional blood drawn during study visits. The additional amount will be about 3.5 teaspoons. Participants will undergo a test called near infrared spectroscopy (NIRS) up to 9 times. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or to hold their breath during these measurements. NIRS measures oxygen levels, blood flow, and the makeup of skin and muscle. Researchers will draw additional information for this study from participants medical records.

NCT ID: NCT04691323 Active, not recruiting - Sickle Cell Disease Clinical Trials

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

Start date: March 2, 2021
Phase: N/A
Study type: Interventional

The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.

NCT ID: NCT04688411 Recruiting - Sickle Cell Disease Clinical Trials

An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease

Start date: September 28, 2020
Phase: N/A
Study type: Interventional

The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and acceptability of MED-Go app in adolescents and young adults (AYA) with sickle cell disease (SCD). The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.

NCT ID: NCT04678037 Completed - Sickle Cell Disease Clinical Trials

Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study

Start date: April 4, 2017
Phase: N/A
Study type: Interventional

The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox feature, which will be integrated into our existing smartphone application platform (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers.

NCT ID: NCT04675645 Completed - Sickle Cell Disease Clinical Trials

Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App

Start date: May 15, 2018
Phase: N/A
Study type: Interventional

This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to complete them, and (3) other procedures related to the use, uptake and effect of the HU-Go app as a tool to improve hydroxyurea (HU) adherence. Second, this study seeks to utilize novel modern mobile technology using a multi-functional personalized platform to improve adherence to HU and measure HRQOL in youth with SCD, using NIH-endorsed PROMIS® measures, based on a conceptual model with predefined behavioral targets and mediators. Third, we plan to assess HRQOL changes and identify modifiable behavioral strategies that could serve as surrogates or predictors for HU adherence. This real-time feedback might empower self-directed changes in behavior that could improve adherence to HU.

NCT ID: NCT02380079 Active, not recruiting - Sickle Cell Disease Clinical Trials

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

Start date: February 2015
Phase: Phase 1
Study type: Interventional

The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.