Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
New Hemolysis Parameters in Sickle Cell Disease
The 6-minute walk (6MWT) test is used in adults and children affected by a wide range of chronic diseases to evaluate their sub-maximal exercise capacity.
- Trial name
- HEMODREP (NCT03040908)
- Volunteers
- 150 people
- Sponsor
- Queen Fabiola Children's University Hospital
Sickle Cell Disease
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…
- Trial name
- SPARKLE (NCT06439082)
- Testing
- Crizanlizumab
- Where
- 36 sites in United States, Brazil and 3 more
- Volunteers
- 354 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening
Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-long debilitation, evidence-based strategies to control SCD are not…
- Trial name
- SIMCS SCD (NCT06563440)
- Testing
- Digital app and information system
- Where
- 2 sites in United States, Uganda
- Volunteers
- 24,000 people, healthy volunteers welcome
- Sponsor
- Makerere University
Sickle Cell Disease
Advancing Feasibility and Acceptability of Digital Cognitive Rehabilitation in Sickle Cell Disease
This is a single site nonrandomized pilot clinical trial of the feasibility, acceptability, and efficacy of a combination treatment involving digital Cogmed working memory skills training, social support components, and memory…
- Trial name
- ALL IN (NCT06770010)
- Testing
- Cogmed+
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's National Research Institute
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
Iron Overload
MRI Evaluation of Iron Overload in the Heart, Liver and Pancreas in Patients Receiving Multiple Blood Transfusions.
Sickle cell anemia and Sickle cell β thalassemia patients require multiple transfusions in order to avoid chronic anemia sequel.
- Trial name
- NCT00629291
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- Sheba Medical Center
Leg Ulcer
SANGUINATE™ in Sickle Cell Disease Associated Leg Ulcer
SANGUINATE™ Sickle Cell Disease associated Leg Ulcers.
- Trial name
- LU (NCT02600390)
- Testing
- SANGUINATE
- Where
- 2 sites in Dominican Republic, Panama
- Volunteers
- 10 people
- Sponsor
- Prolong Pharmaceuticals
Sickle Cell Disease
Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease.
- Trial name
- NCT01835496
- Testing
- single 1500 mg dose of Ferriprox
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- ApoPharma
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Conditions
- Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders
- Trial name
- FAM BMT (NCT03128996)
- Testing
- RIC regimen, GVHD prophylaxis regimen
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Sickle Cell Disease, Acute Chest Syndrome
sPLA2 in EBC During Acute Chest Syndrome
Secretory phosholipases A2 (sPLA2) are significantly elevated in the plasma of sickle cell disease patients with acute chest syndrome (ACS), and similar enzymes have been measured in exhaled breath condensate (EBC), which is…
- Trial name
- NCT03250585
- Testing
- Exhaled Breath Condensate (EBC), Plasma Sample
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Virginia Commonwealth University
Sickle Cell Disease
Laboratory-based Hypnosis Intervention on Pain Responsivity in Adolescents With Sickle Cell Disease
The purpose of this study is to test the effects of a laboratory-based hypnosis session compared to an attention control condition on peripheral blood flow, autonomic stress responses, and acute pain responses in adolescents…
- Trial name
- NCT02987725
- Testing
- Hypnosis, Attention Control
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, Los Angeles
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Stroke (CVA) or TIA, Diabetes
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…
- Conditions
- Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease)
- Trial name
- NCT07532564
- Where
- United States
- Volunteers
- 30,486 people, healthy volunteers welcome
- Sponsor
- University of Maryland, Baltimore
Sickle Cell Disease, Cerebral Ischemia
Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia
The purpose of the present observational study is to remotely reevaluate the cohort of 67 sickle cell patients with transcranial Doppler-detected cerebral vasculopathy included in the national "Sickle Cell Transplant" protocol…
- Conditions
- Sickle Cell Disease, Cerebral Ischemia, Stenosis
- Trial name
- DREPAGREFFE2 (NCT05053932)
- Testing
- blood collection
- Where
- 14 sites in France
- Volunteers
- 67 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
The purpose of this research study is to study the safety of increasing doses of FBS0701, and to see how quickly the study medication is absorbed and how quickly it disappears from the bloodstream.
- Trial name
- NCT01039636
- Testing
- FBS0701
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 20 people
- Sponsor
- FerroKin BioSciences, Inc.
Sickle Cell Disease
Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor
With recent advances in gene editing, gene therapy is becoming a viable curative treatment option for sickle cell disease. In order to do genetic manipulation, investigators need to collect hematopoietic stem cells from patients…
- Trial name
- NCT04817345
- Testing
- Plerixafor
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Writing Relaxing Beats in Adolescents Who Have Sickle Cell Disease
This research aims to see if songwriting can help reduce anxiety in adolescents with Sickle Cell Disease. The purpose of the study is to discover if participants find songwriting and playing their songs to be practical and…
- Trial name
- NCT07222475
- Testing
- Songwriting intervention (active music therapy)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Mayo Clinic
Sickle Cell Disease, Vitamin D Deficiency
Vitamin D for Sickle-cell Respiratory Complications
This study aims to answer the question whether oral vitamin D supplementation can decrease lung complications in children and adolescents with sickle cell disease.
- Conditions
- Sickle Cell Disease, Vitamin D Deficiency, Acute Chest Syndrome, Asthma
- Trial name
- NCT01443728
- Testing
- Experimental: Vitamin D3 100,000 IU, Active Comparator: Vitamin D3 12,000 IU
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Gary M Brittenham, MD
Sickle Cell Disease
Development and Adaptation of I-STRONG for SCD
This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.
- Trial name
- NCT06110754
- Testing
- I-STRONG for SCD
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- Emory University
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