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Anemia, Sickle Cell clinical trials

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NCT ID: NCT05799118 Recruiting - Sickle Cell Disease Clinical Trials

Study of the Role of Genetic Modifiers in Hemoglobinopathies

INHERENT
Start date: October 1, 2022
Phase:
Study type: Observational

This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).

NCT ID: NCT05780775 Recruiting - Sickle Cell Disease Clinical Trials

Lipid Balance in Adult Sickle Cell Patients

HDL2
Start date: November 30, 2022
Phase: N/A
Study type: Interventional

This study aims to describe and/or searches for, in cohorts of adult sickle cell anemia (SCA) and SC sickle cell patients living in the French West Indies and followed by SCD Reference and Competence Centers: 1-lipids profiles and associations at steady state with occurrence of sickle cell disease (SCD) complications, 2-lipids profile evolution during and after prospective acute complications (vasoocclusive crises (VOC) and priapism), 3-lipids profile variation (inter /intra individuals) during 4 prospective years, 4- Genetic primary modulators of SCD complications, 5- insulin resistance (HOMA), free fatty acids and glycerol dosages, 6- lipids enzymes, lipidome and functionality of HDL in sub-groups of SCD population.

NCT ID: NCT05758766 Recruiting - Sickle Cell Disease Clinical Trials

Study on Use of Omega-3 Fatty Acids to Improve Outcomes in Individuals With Sickle Cell Disease

Start date: August 30, 2023
Phase: N/A
Study type: Interventional

Sickle cell disease (SCD) is associated with significant morbidity and mortality. Pain and many adverse outcomes occurring in sickle cell disease are inflammatory driven. Recent data has shown that gut dysbiosis is present in individuals with sickle cell disease. Gut dysbiosis has been linked to inflammation in certain diseases. Omega -3-fatty acids (fish oil) has been shown to improve pain outcomes in individuals with sickle cell disease, but its acceptance is variable. The aim of this study is to determine if a plant-based omega-3-fatty acids will be more acceptable and also improve outcomes in individuals with sickle cell disease

NCT ID: NCT05748717 Recruiting - Sickle Cell Disease Clinical Trials

Arterial Function Parameters and Transcranial Doppler Velocity in Paediatric Patients With Sickle Cell Disease

AFAT
Start date: February 1, 2023
Phase:
Study type: Observational

Structural and functional changes in arteries are increasingly being recognized as significant features of sickle cell disease. This study aims to determine whether there are differences in arterial function parameters between children with sickle cell disease with normal and abnormal transcranial Doppler velocity. After informed consent is obtained, participants will have vascular, Transcranial Doppler, haematological and biochemical parameters measured. Researchers will compare children with sickle cell disease who have normal Transcranial Doppler velocity and no history of stroke with children with those who have an abnormal Transcranial Doppler velocity with or without a history of stroke to see if there are significant differences in arterial function parameters.

NCT ID: NCT05736419 Recruiting - Sickle Cell Disease Clinical Trials

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Start date: February 9, 2023
Phase: Phase 2
Study type: Interventional

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.

NCT ID: NCT05730205 Recruiting - Sickle Cell Disease Clinical Trials

Effects of the Contraceptive Implant in Women With Sickle Cell Disease

SCD CURE
Start date: June 8, 2023
Phase: Phase 4
Study type: Interventional

The objective of this study is to measure the acceptability and impact of the progestin implant on frequency of vaso-occlusive crises, quality of life, and hematologic parameters in women with SCD.

NCT ID: NCT05714098 Recruiting - Sickle Cell Disease Clinical Trials

Gerofit Exercise Intervention for Older Adults With Sickle Cell Disease (SICKLE-FIT Study)

SICKLE-FIT
Start date: March 27, 2023
Phase: N/A
Study type: Interventional

The purpose of this study to assess the feasibility, acceptability, and safety of a personalized exercise training program adapted from Gerofit to improve physical health and quality of life for adults with SCD

NCT ID: NCT05687474 Recruiting - Cystic Fibrosis Clinical Trials

Baby Detect : Genomic Newborn Screening

Start date: September 1, 2022
Phase:
Study type: Observational

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.

NCT ID: NCT05662098 Recruiting - Sickle Cell Disease Clinical Trials

Alternative Dosing And Prevention of Transfusions (ADAPT)

Start date: June 16, 2022
Phase: Early Phase 1
Study type: Interventional

ADAPT is a prospective cohort study at Jinja Regional Referral Hospital (JRRH) primarily to assess the effect of hydroxyurea on blood transfusion utilization and secondarily to determine the feasibility of PK-guided hydroxyurea dosing.

NCT ID: NCT05660265 Recruiting - Clinical trials for Hematologic Diseases

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years

Start date: August 26, 2023
Phase: Phase 1
Study type: Interventional

This will be a first time in human (FTIH) study in sickle cell diseases (SCD) participants. The FTIH study is planned to evaluate the safety, tolerability, and pharmacokinetics of GSK4172239D. The study will be composed of 3 periods for all participants (Screening, Treatment, and Follow up). Participants will be screened and, prior to first dose on Day 1, will be randomized to receive either GSK4172239D or placebo. GSK4172239D is a prodrug that is converted in vivo into GSK4106401. This study will be a single dose, dose-escalation study. The initial dosing for all cohorts will be staggered so that 2 participants will be dosed as sentinel participants. Provided there are no safety concerns in 48 hours (h), the remaining 6 participants scheduled for the cohort may be dosed. One selected cohort of participants will also receive an additional single dose of GSK4172239D (or matching placebo) under fed (high calorie and high fat) conditions after a washout period of a minimum of 20 days or 5 half-lives, whichever is longer, designated as the Food Effect Cohort.