Clinical trials: Sickle Cell Disorder
Sickle Cell, Children
VO2max & HRQoL in Children With Sickle Cell Disease
Sickle cell disease is the most common inherited genetic disorder, accounting for 300,000 births worldwide per year. It is caused by an autosomal recessive mutation of the β-globin gene, responsible for an abnormal hemoglobin…
- Trial name
- VO2drépano (NCT05995743)
- Where
- France
- Volunteers
- 72 people
- Sponsor
- University Hospital, Montpellier
Sickle Cell Disease
Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own…
- Trial name
- GRASP-EA (NCT07640815)
- Testing
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Where
- United States
- Sponsor
- David Williams
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Chronic Pain, Depression, Anxiety
Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell Via Mobile Applications
The investigators are conducting a comparative effectiveness trial among adult patients with sickle cell disease (SCD) who report chronic pain (N = 350), randomized to receive either mobile phone-delivered computerized cognitive…
- Conditions
- Chronic Pain, Depression, Anxiety, Opioid Use
- Trial name
- CaRISMA (NCT04419168)
- Testing
- cCBT, m-Education
- Where
- 7 sites in United States
- Volunteers
- 359 people
- Sponsor
- University of Pittsburgh
HbE Trait, Hemoglobinopathies
Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh
Hemoglobinopathies are inherited disorders that affect haemoglobin and pose a considerable global health challenge. This research addresses the knowledge gap regarding the prevalence of a typical haemoglobin fraction within urban…
- Trial name
- Hb Variants (NCT07305181)
- Where
- Bangladesh
- Volunteers
- 8,400 people
- Sponsor
- Bangladesh Bioscience Research Group
Sickle Cell Disease
Web-MAP Intervention for Youth With Sickle Cell Disease
The proposed study will determine whether the efficacy of WebMAP cognitive behavioral therapy (CBT) treatment study generalizes to pediatric sickle cell disease (SCD), and explore whether the intervention is feasible and…
- Trial name
- Web-MAP (NCT02511769)
- Testing
- WEB-MAP Educational Control Group, WEB-MAP CBT
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Connecticut Children's Medical Center
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
Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease
The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV…
- Trial name
- NCT07247188
- Testing
- PCV21, 20vPCV
- Where
- 7 sites in United States, Dominican Republic
- Volunteers
- 100 people
- Sponsor
- Sanofi
Sickle Cell Disease
Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia
MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients with sickle cell disease.
- Trial name
- MitoQ (NCT04109820)
- Testing
- MitoQ
- Where
- 5 sites in United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Sickle Cell Anemia, Chronic Kidney Disease
Efficacy, Safety Study and Benefit of Alkali Therapy in Sickle Cell Disease
The objective of this study is to assess the effect of alkali administration on bicarbonate and potassium levels in patients with Sickle Cell Disease (SCD) and depressed serum bicarbonate levels.
- Conditions
- Sickle Cell Anemia, Chronic Kidney Disease, Metabolic Acidosis
- Trial name
- NCT01894594
- Testing
- Sodium Bicarbonate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Disease
Albuminuria Reduction With Renin Angiotensin System Inhibitors in SCA Patients
The prevalence of Sickle Cell Associated Nephropathy (SCAN) is increasing and is a growing concern. Microalbuminuria is detected in the early onset of SCAN.
- Trial name
- RAND (NCT01195818)
- Testing
- RAS Inhibitors
- Where
- France
- Volunteers
- 53 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Covid19
Comorbidities and Risk Score in COVID-19 Patients
Retrospective multi-center cohort study. Consecutive patients hospitalized for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) up to October 2020 will be included.
- Trial name
- Comorbidities (NCT04670094)
- Where
- 5 sites in Italy
- Volunteers
- 4,555 people
- Sponsor
- University of Milano Bicocca
Pain Crisis
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.
- Trial name
- NCT00081523
- Where
- 3 sites in United States
- Volunteers
- 3,500 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease
Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures.
- Trial name
- NCT03077542
- Testing
- haploidentical stem cell transplant, Sirolimus, Alemtuzumab
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Pregnancy, High Risk
SCRIPT: Sickle Cell Risk in Pregnancy Tool
Sickle Cell Disease (SCD), common in persons of Black ancestry, affects the shape of hemoglobin, the oxygen-carrying part of red blood cells (RBC).
- Conditions
- Sickle Cell Disease, Pregnancy, High Risk, Pregnancy Complications
- Trial name
- SCRIPT (NCT06529042)
- Testing
- Non-Interventional
- Where
- 3 sites in United States, Canada
- Volunteers
- 1,000 people
- Sponsor
- Mount Sinai Hospital, Canada
Sickle Cell Disease
Re-Aiming at Hydroxyurea Adherence for Sickle Cell With mHealth
National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend that hydroxyurea be offered to symptomatic adults and all children with sickle cell disease (SCD) (HbSS and HbSβ0-thal…
- Trial name
- NCT03344900
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Sickle Cell Trait
CHOICES3: Sickle Cell Disease Parenting CHOICES
The study will use web-based data collection (SCKnowIQ) and intervention delivery strategies enhanced by nudges and tailored boosters in a sample of 430 adult men and women, aged 18-45 yr with SCD (Sickle Cell Disease) or SCT…
- Trial name
- CHOICES3 (NCT05292781)
- Testing
- CHOICES, eBook
- Where
- United States
- Volunteers
- 506 people
- Sponsor
- University of Florida
Sickle Cell Disease
Pilot Study on the Effects of Intravenous Ketamine on Acute Pain Crisis in Patients With Sickle Cell Disease
The purpose of this pilot study is to provide a preliminary assessment of the feasibility and efficacy of intravenous ketamine in controlling pain in patients with sickle cell disease (who are admitted to the hospital with…
- Trial name
- NCT00252122
- Testing
- Ketamine
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Hospital of Philadelphia
Sickle-cell Disease (SCD)
Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve…
- Trial name
- DREPAtient (NCT04413539)
- Where
- France
- Volunteers
- 1,088 people
- Sponsor
- Argo Sante
Sickle Cell Disease
Stem Cell Transplant in Patients With Severe Sickle Cell Disease
This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen…
- Trial name
- NCT03421756
- Testing
- Alemtuzumab, Total Body Irradiation, Sirolimus
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Kathleen Dorritie
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