Clinical Trials for Sickle Cell Disease

Source: ClinicalTrials.gov — U.S. National Library of Medicine — A database of privately and publicly funded clinical studies conducted around the world (U.S. National Library of Medicine, public domain).

This page lists clinical trials related to Sickle Cell Disease from the ClinicalTrials.gov database.

Trial Summary

• Total studies: 50
• Recruiting: 19
• Active, not recruiting: 9
• Completed: 13
• Other: 9

Results are refreshed periodically and may not reflect the most current information available from ClinicalTrials.gov.

Recruiting Trials

NCT00081523 — Natural History of Sickle Cell Disease

This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated. Subjects will receive their general medical care outside the NIH and will be seen at our clinic or at CNHS with varying frequency. Subjects may be seen for multiple visits. Subjects may be …

NCT03121001 — Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT).

The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical h…

NCT03653338 — T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a s…

NCT03937817 — Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

Background:

Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders. To do this, they need to collect biological samples from people with blood disorders. They also need to collect samples from healthy people.

Objective:

To collect samples to use for research on blood disorders.

Eligibility:

People ages …

NCT04093986 — Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation

The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation.

NCT04819841 — Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

NCT05213572 — Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies

Background:

People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens.

Objective:

To study the benefits of treatments that are intended to cure SCD.

Eligibility:

People aged 18 and older with SCD who are either receiving curative therapy …

NCT06318143 — mAnaging siCkle CELl disEase Through incReased AdopTion of hydroxyurEa in Nigeria

Large knowledge gaps remain regarding strategies to promote the adoption of hydroxyurea (HU), particularly in sub-Saharan African countries including Nigeria, where more than 75% of annual sickle cell anemia births occur. The vast majority of people with SCD in Africa do not receive evidenced-based health care (e.g., newborn screening, health education, prophylaxis for infection, optimal nutrit…

NCT06481306 — A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

NCT06546670 — A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

NCT06477289 — Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease

This study will examine sleep disordered breathing and sleep quality in participants (ages 12-25) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotely in the homes of participants.

Neurocognitive, behavioral, and neuroimaging evaluations will occ…

NCT06439082 — A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)

A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

NCT05904093 — Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

Background:

Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves. People with SCD are also at increased risk of forming blood clots in the veins and lungs, but the standard treatments for these clots can cause increased bleeding in peop…

NCT06612268 — A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people w…

NCT06975865 — The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD).

Study details include:

• Study duration: a 52-week double-blind period (Part A), followed by an open-label …

NCT06818266 — Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Patients With Sickle Cell Disease

The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients with sickle cell disease (SCD) during acute chest syndrome (ACS).

NCT07226219 — Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease

The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects.

The study will assess any thinking or attention problems participants may have both before taking this drug and after. Additiona…

NCT07566494 — Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease

Background:

Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen. Research has shown that curcumin, a natural compound found in turmeric, can improve the health of red blood cells in people with SCD. But the body cannot absorb curcumin well when it is taken by mouth. Researchers want to know if a skin gel (VAS-101) can hel…

NCT07599176 — Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.

Active, Not Recruiting

NCT01499888 — Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)

The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy in patients with aggressive sickle cell disease who are not candidates for or experienced complications from …

NCT01966731 — Realizing Effectiveness Across Continents With Hydroxyurea (REACH)

REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for for pediatric patients with sickle cell anemia (SCA). 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 hydroxyure…

NCT03655223 — Early Check: Expanded Screening in Newborns

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health condi…

NCT03708731 — Exploring Adherence Monitoring in Sickle Cell Disease

Despite the well-documented benefits of hydroxyurea (HU) therapy in decreasing morbidity and mortality in youth with Sickle cell disease (SCD), pediatric HU adherence rates range as low as 49% and lead to discontinuation of HU regimens in 8-20%. In addition, treatment non-adherence may lead to unnecessary increases in medication dosage resulting from erroneous assumption that a patient is non-r…

NCT04610866 — Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

Background:

Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work. Researchers want to see if a drug called mitapivat can help people with SCD.

Objective:

To test the long-term tolerability and safety of mitapivat (or AG-348) in people with SCD.

Eligibility:

Adults age 1…

NCT05031780 — A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on effic…

NCT05329649 — Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

NCT06417411 — LEARNER- Low dosE AspiRiN prEterm tRial (Angola)

This study is being conducted to evaluate the safety and effect of starting daily use of low dose (100 mg) aspirin in pregnant women with sickle cell disease, who are being followed in two county hospitals in Angola, in the first trimester versus the second trimester of the gestational period.

NCT05675436 — Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

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 …

Not Yet Recruiting

NCT07562451 — Assessing Molecular Mechanisms and Effects of Music Therapy in Youth With Sickle Cell Disease Using Single-cell RNA-sequencing

The goal of this clinical trial is to evaluate whether a 4-week music therapy (MT) intervention can reduce chronic pain and improve psychosocial outcomes in youth with sickle cell disease (SCD).

The main questions it aims to answer are:

• Does MT reduce pain intensity, frequency of pain episodes, and improve health-related quality of life (HRQoL)?
• Does MT alter immune cell composition and g…

NCT07656415 — A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.

NCT06797583 — Transcranial Photobiomodulation Treatment in Patients With Sickle Cell Disease

Participants are being asked to take part in this clinical trial, a type of research study, because investigators want to learn more about oxygen usage in the brain. Patients diagnosed with sickle cell disease are at risk for difficulties with thinking and academic skills. The brain requires a consistent supply of oxygen for normal function, but this supply is reduced among patients with sickle…

NCT07682662 — Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis

The goal of this study is to learn if Ketamine works more efficiently, as compared to Opioids, for Sickle Cell Pain The main questions it aims to answer are:

Does Ketamine lower the number of times participants need to be admitted for continued pain control during a Sickle Cell Pain Crisis.

Does Ketamine decrease the amount of time it takes to reach adequate pain control/pain score improvemen…

NCT07674277 — Virtual Reality for SCD VOC

This pilot study will evaluate the feasibility, tolerability, and preliminary analgesic effect of headset-based virtual reality interventions for adults with sickle cell disease experiencing vaso-occlusive crisis treated in an infusion center. Participants will be enrolled during routine outpatient sickle cell clinic visits and may receive study interventions during future qualifying infusion c…

NCT07217548 — Solving Stigma Through POV Simulation: Enhancing Pharmacist Empathy-based Practices With Sickle Cell Disease Patients

Why This Research Matters (Significance)

1 in 5 of the 50+ million people with chronic pain in the U.S. face stigma when using opioids, especially women and racial minorities. This stigma often comes from pharmacy staff treating chronic pain patients as drug seekers, who then refuse to fill the patient’s script, and leave the patient in severe pain. The team has found that having pharmacists w…

Enrolling by Invitation

NCT00047996 — Blood Collection for Research Related to Certain Diseases Involving Blood Vessels

The collection of human specimens from both patients and healthy volunteers is necessary for the development of laboratory assays to further basic and clinical research studies. This protocol defines the purposes for which specimens will be collected and establishes general conditions under which sample collection will be performed….

Completed Trials

NCT00011648 — Secondary Pulmonary Hypertension in Adults With Sickle Cell Anemia

The purpose of this study is to determine how often people with sickle cell anemia develop pulmonary hypertension a serious disease in which blood pressure in the artery to the lungs is elevated.

Men and women 18 years of age and older with sickle cell anemia may be eligible for this study. Participants will undergo an evaluation at Howard University s Comprehensive Sickle Cell Center in Washi…

NCT01633021 — Developing the Family Map: Looking at Communal Coping

Background:

- Knowing one s family medical history is a part of staying healthy. Some health risks run in families, and knowing these risks can promote more healthy behavior. Different social and cultural factors may affect how family members share this information. Genetic risk information that is shared in one family may not be shared in the same way in another. This information may also be…

NCT02156102 — Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study)

Background:

- People with sickle cell disease and other blood disorders sometimes get chronic leg ulcers. These are wounds that develop on the skin and don t go away. Current treatments do not work very well, so researchers want to learn more about why the ulcers happen. They want to find out which bacteria may cause it, and if external factors play a role.

Objective:

- To study social and…

NCT02580565 — Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease

The use of analgesics can lead to cases of drug abuse and dependence. It can also cause pseudo-addiction in patients suffering from pain. What is the actual situation in patients suffering from severe sickle-cell disease, exposed to acute pain during vaso-occlusive crises? Evaluation of the use of analgesics, on the basis of Diagnostic and Statistical Manual of Mental Disorders criteria for sub…

NCT03049475 — Pathophysiology of Acute Pain in Patients With Sickle Cell Disease

Background:

Sickle Cell Disease (SCD) is a blood disorder that occurs mainly in people of African descent. Researchers want to learn more about the painful attacks and complications associated with SCD. They want to look for a relationship between SCD and specific changes in the blood. They want to study the role of genetics, inflammation, and blood clotting factors in SCD. They will do this w…

NCT03263559 — Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe SCD; and (2) adults with severe SCD.

NCT04839354 — Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial

The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.

NCT07532564 — Risk Factors, Costs, and Impacts of ED Boarding

The goal of this observational study is to learn about the risk factors, costs, and operational impacts of emergency department boarding (patients admitted to the hospital but remaining in the emergency department awaiting placement on an inpatient floor)

The main questions it aims to answer is:

1. What characteristics of patients make them more likely to experience ED boarding?
2. What is th…

NCT05405114 — Research Study Investigating How Well NDec Works in People With Sickle Cell Disease

This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo. Participants who are on HU treatment befo…

NCT05714098 — Gerofit Exercise Intervention for Older Adults With Sickle Cell Disease (SICKLE-FIT Study)

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

NCT05789355 — Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)

This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety and tolerability parameters as well as to measure the plasma and urinary residues of daily oral doses of NUV001. Secondly, the study will evaluate the impact of NUV001…

NCT05791591 — Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients

This multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study evaluated the safety, tolerability, and exploratory efficacy of orally administered NUV001 in adult participants with sickle cell disease (HbSS or HbSβ0 genotypes). A total of 168 participants were randomized in a 1:1:1 ratio to receive NUV001 immediate-release (IR), NUV001 gastro-resistant (GR), or place…

NCT06397118 — Prevention of Malnutrition in Children With Sickle Cell Disease

The investigators are studying how to prevent malnutrition in children with sickle cell disease (SCD) in northern Nigeria. Community health workers will teach mothers about nutritious local foods that might help children grow better than the usual advice from doctors. The investigators will enroll 148 children with SCD aged 6 to 18 months old. The investigators will determine if their weight an…

Other (Terminated)

NCT04076748 — Evaluation of the Efficacy of Intra-nasal Sufentanil for Analgesia of Vaso-occlusive Crisis in Sickle-cell Adults.

The analgesic treatment for vaso-occlusive crisis (VOC) in sickle-cell patients is an emergency. The reference treatment is morphine, which requires a venous way sometimes difficult to obtain in these patients. Sufentanil intranasal has been shown to be effective in traumatology. The objective is to evaluate, in VOC, the efficacy of intranasal sufentanil relayed by morphine IV compared to the u…

NCT07431398 — Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).