Clinical trials: Sickle Cell Disorders
Sickle Cell Disease
Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease
The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care.
- Trial name
- iTransition (NCT02200510)
- Testing
- Self-management intervention for Adolescents with SCD, Patient Portal Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Sickle Cell Nephropathy
Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease
The purpose of this research study is to learn about the effect of the drug, atorvastatin, on blood vessels in patients with sickle cell disease.
- Trial name
- ENDO (NCT01732718)
- Testing
- Atorvastatin
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease, Quality of Life
Improving Disease Knowledge in Adolescents With Sickle Cell Disease
Increased knowledge about an illness can increase self-management among those afflicted. In order to facilitate people with sickle cell disease living a longer and healthier life, they should be taught to manage their illness.An…
- Trial name
- KNOW-IT (NCT01945073)
- Testing
- Educational Booklet (BK), Formal Counselling (CB)
- Where
- Jamaica
- Volunteers
- 150 people
- Sponsor
- The University of The West Indies
Sickle Cell Disease, Sickle Cell Anemia
A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy
This trial is a follow-up companion study to Protocol ICA-17043-10, a Phase III, multi-center, efficacy and safety study of ICA-17043.
- Trial name
- NCT00294541
- Testing
- ICA-17043
- Where
- 31 sites in United States
- Volunteers
- 51 people
- Sponsor
- Icagen
Sickle Cell Disease
Algorithm for Apherisis Monitoring and Prescription Assistance in Sickle Cell Patients (ALGODREP)
The main objective of this study is to prove the superiority of a procedure which calculates the volume of RBCs to transfuse and the time between apheresis based on this algorithm, compared to the current procedure.
- Trial name
- ALGODREP (NCT04076683)
- Testing
- Algodrep
- Where
- 8 sites in France, Martinique
- Volunteers
- 65 people
- Sponsor
- Etablissement Français du Sang
Pneumococcal Infections
Immunogenicity and Safety Study of Pneumococcal 7-Valent Conjugate Vaccine in Sickle Cell Disease Infants.
The primary objectives of this study were to assess the immunogenicity and the tolerance of the heptavalent pneumococcal conjugate vaccine (Prevenar) in young infants (2 months of age) with sickle cell disease when administred at…
- Trial name
- NCT00368186
- Testing
- Pneumo 23
- Where
- France
- Volunteers
- 51 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Sickle Cell Disease
BEATS 2: Music Therapy in Sickle Cell
The purpose of this study is to investigate the effects of the BEATS music therapy program on the self-efficacy, trust, knowledge, and adherence of young adult patients with SCD.
- Trial name
- NCT03233269
- Testing
- Music Therapy
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
- Trial name
- NCT07023666
- Testing
- Deferoxamine, Deferasirox, Deferiprone
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- Inova Health Care Services
Genotype, Pain
Genetics and Pain Severity in Sickle Cell Disease
Background: Pain is the most common symptom of sickle cell disease. Episodes of severe sickle cell pain are known as "crises." High rates of pain crises are associated with a higher risk of early death.
- Conditions
- Genotype, Pain, Genetic Variation, Quantitative Sensory Testing (QST)
- Trial name
- NCT01441141
- Where
- United States
- Volunteers
- 67 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Vaso-occlusive Crisis, Sickle Cell Disease
Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia
The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease.
- Conditions
- Vaso-occlusive Crisis, Sickle Cell Disease, Sickle Cell Anemia in Children
- Trial name
- NCT04301336
- Testing
- Omega 3, Vit D, Zinc sulfate
- Where
- 4 sites in Egypt, Saudi Arabia
- Volunteers
- 350 people
- Sponsor
- Beni-Suef University
Anemia, Sickle Cell
Quality of Life of Children With Sickle Cell Disease Who Are Getting Chronic Transfusions With a Lifeport
This study is being done to see what impact having a Lifeport device has on quality of life for children with sickle cell who are getting chronic transfusions, from the child's perspective.
- Trial name
- NCT00246077
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Mercy Hospital Kansas City
Sickle Cell Anemia
Therapeutic Application of Intravascular Nitrite for Sickle Cell Disease
This study examines ways in which nitric oxide (NO), an important molecule that controls how blood flows through the body's vessels, might be restored with a compound called sodium nitrite.
- Trial name
- NCT00095472
- Testing
- L-NMMA, Sodium Nitrite
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Pulmonary Hypertension
Imatinib and Carvedilol for High Blood Pressure in the Lungs in Adults With Sickle Cell Disease
Background: About one-tenth of adults with sickle cell disease have pulmonary hypertension (high blood pressure in the lungs). This condition can cause shortness of breath, pain crisis, and congestive heart failure.
- Trial name
- NCT01568645
- Testing
- Carvedilol, Imatinib
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Sickle Cell Disease
Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text Messaging
The investigators will explore barriers to improving self-efficacy, or the ability to feel in control of their disease, and medication adherence with text messaging through surveys and interviews with adolescents and adults with…
- Trial name
- NCT02785848
- Testing
- Interviews and Surveys
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Vanderbilt University
Sickle Cell Disease
Music Therapy in Sickle Cell Transition Study
The purpose of this study is to investigate the effects of the BEATS Music Therapy Program on the self-efficacy, trust, knowledge, and adherence of young adult patients with sickle cell disease during transition.
- Trial name
- NCT02466555
- Testing
- Music Therapy
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Disease
Methadone in Pediatric and Adult Sickle Cell Patients
To determine the pharmacokinetics of methadone in children and adults with SCD who are experiencing a painful episode.
- Trial name
- MSCD (NCT00761085)
- Testing
- Morphine, Methadone
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.
The purpose of this study is to evaluate the Safety and Efficacy of DREAM01, a gene therapy for Sickle Cell Disease (SCD). The therapy consists of transplanting autologous CD34+ cells transduced ex vivo with a bifunctional…
- Trial name
- DREPAMIR (NCT07432867)
- Testing
- DREAM01 drug product, anti-inflammatory therapy
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Hemoglobin Desaturation in Sickle Cell Disease
As part of routine care for SCD, some people are found to have low oxygen levels (≤ 88%) while sleeping, at rest, or with exercise.
- Trial name
- NCT03908385
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University Hospitals Cleveland Medical Center