Clinical trials: Sickle Cell Disorders
Sickle Cell Disease
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).
- Trial name
- NCT03263559
- Testing
- Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG
- Where
- 32 sites in United States
- Volunteers
- 95 people
- Sponsor
- Medical College of Wisconsin
Benign and Malignant Hematologic Diseases
The American Society of Hematology (ASH) Research Registry: A Multicenter Research Registry of Patients With Hematologic Disease
This is a multicenter, retrospective and prospective, long-term registry of patients with benign or malignant hematologic diseases, whether or not these patients were or were not treated with disease-specific treatments.
- Trial name
- NCT03535220
- Testing
- Protease Inhibitors
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- American Society of Hematology
Anemia, Sickle Cell
Systematic Psychological and Medical Care for Children With SCD
Sickle cell disease (SCD), a genetically transmitted blood disease, necessitates life-long care. In children, the disease may cause intense pain and other severe complications.
- Trial name
- Drépano (NCT02022891)
- Testing
- systematic psychological care
- Where
- France
- Volunteers
- 160 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Sickle Cell Disease
Study of Crizanlizumab for Prevention of Silent Cerebral Infarcts in SCA
In this prospective, single-arm, open-label, imaging and treatment study, the investigator will test the hypothesis that crizanlizumab will prevent the progression of silent cerebral infarcts in patients with sickle cell disease.
- Trial name
- CRIZ (NCT05334576)
- Testing
- Crizanlizumab
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Andria Ford
Sickle Cell Disease
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the…
- Trial name
- NCT00911495
- Testing
- GMI-1070
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- GlycoMimetics Incorporated
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
Anemia, Sickle Cell
Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.
- Trial name
- NCT02187003
- Testing
- Rivipansel
- Where
- 142 sites in United States, Canada
- Volunteers
- 345 people
- Sponsor
- Biossil Inc.
Sickle Cell Disease
Nasopharyngeal Bacterial Carriage and Antibiotic Resistance in Children With Sickle Cell Disease in Ile-De-France
The objective of this study is to to determine the rate of nasopharyngeal carriage of Streptococcus pneumoniae (Sp) in children with sickle cell disease over 6 months and under 15 years of age over a 9-month period in…
- Trial name
- DREPANO-BACT (NCT05197205)
- Testing
- nasopharyngeal swabbing
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen
Sickle cell disease (SCD) is the most common inherited blood disorder in Saudi Arabia . Its clinical severity is widely heterogeneous among patients who share the same genetic mutation .
- Trial name
- NCT02776202
- Testing
- Fludarabine monophosphate
- Where
- Saudi Arabia
- Volunteers
- 15 people
- Sponsor
- National Guard Health Affairs
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
Sickle Cell Disease
Preservation and Transfer of HBV Immunity After Allogeneic HSCT for SCD
Sickle cell disease (SCD) patients ending with mixed mononuclear chimerism after non-myeloablative HSCT with alemtuzumab/TBI conditioning will probably preserve their immune response to vaccinations administered prior to the…
- Trial name
- PROTECT (NCT05200338)
- Testing
- Engerix-B
- Where
- Netherlands
- Volunteers
- 30 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Heart Rate Variability (ANS Function)
Autonomic Nervous System and Sickle Cell Disease
Sickle cell disease (SCD) children and adults with asthma have an increased rate of vaso-occlusive crisis, acute chest syndrome episodes, and premature mortality when compared to those without asthma.
- Trial name
- DrepaSympa (NCT04062409)
- Where
- France
- Volunteers
- 90 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Anemia, Beta-thalassemia Major
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…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Sickle Cell Disease
Uganda Sickle Surveillance Study (US-3)
It is estimated that over 250,000 babies are born with sickle cell disease (SCD) annually in sub-Saharan Africa, and only 10% - 50% of them survive beyond five years of age.
- Trial name
- NCT06301893
- Where
- 4 sites in Uganda
- Volunteers
- 1,000,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Angiotensin-converting Enzyme Inhibitors and Early Sickle Cell Renal Disease in Children
Patients with sickle cell anaemia may develop renal disease. In fact, renal disease occurred in 40% of adults patients (macroalbuminuria) with evolution to end-stage renal disease for half of them.
- Trial name
- MADREPIEC (NCT01096121)
- Testing
- Enalapril
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Anemia
Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE)
The primary goal of the Phase III SCATE trial is to compare 30 months of alternative therapy (hydroxyurea) to standard care (observation) in children with sickle cell anemia and conditional (170 - 199cm/sec) Transcranial Doppler…
- Trial name
- SCATE (NCT01531387)
- Testing
- Hydroxyurea
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 38 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Vaso-occlusive Crisis
Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis
GMI-1070 is a new drug that may reduce the stickiness of cells in the blood. The purpose of this study is to evaluate whether GMI-1070 can reduce the time it takes for pain to go away in patients with vaso-occlusive crisis (also…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Pain Crisis
- Trial name
- NCT01119833
- Testing
- GMI-1070
- Where
- 22 sites in United States, Canada
- Volunteers
- 81 people
- Sponsor
- GlycoMimetics Incorporated
Sickle Cell Anemia, Muscle Dysfunction
Physical Rehabilitation in Sickle Cell Anemia
Sickle cell anemia (SCA) is one of the most neglected diseases worldwide, according to the World Health Organization. In the adult population with SCA, the systemic effects of the disease, such as respiratory and peripheral…
- Conditions
- Sickle Cell Anemia, Muscle Dysfunction, Quality of Life, Physical Activity
- Trial name
- NCT04705792
- Testing
- Physical activity
- Where
- Brazil
- Volunteers
- 40 people
- Sponsor
- Centro Universitário Augusto Motta
Sickle Cell Disease
N-Acetylcysteine in Patients With Sickle Cell Disease
The primary aim of this study is to evaluate the effect of the drug N-Acetylcysteine on the frequency of pain in daily life in patients with Sickle Cell Disease (SCD).
- Trial name
- NAC (NCT01849016)
- Testing
- N-Acetylcysteine
- Where
- 11 sites in Belgium, Netherlands and 1 more
- Volunteers
- 96 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Smoking, Cancer
Parent Willingness to Participate in Tobacco Trials in the Pediatric Clinical Setting
Little is known about what factors influence parental decisions to participate or to decline participation in tobacco trials offered in the pediatric clinical setting.
- Conditions
- Smoking, Cancer, Sickle Cell Disease
- Trial name
- NCT01673243
- Testing
- Questionnaire
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- St. Jude Children's Research Hospital
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