Clinical trials: Sickle Cell Disorder
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Sickle Cell Anemia
Effect of Exercise With and Without HMB on Body Composition and Muscle Strength in Sickle Cell Anaemia
Wasting is a common and significant problem in sickle cell anaemia (SCA) that correlates with poorer clinical outcome such as frequent painful crises, acute chest syndrome and sub normal resistance to infection.
- Trial name
- NCT04001907
- Testing
- Resistance exercise, β-hydroxy-β-methylbutyrate
- Volunteers
- 24 people
- Sponsor
- The University of The West Indies
SC Disease, Pain, Chronic
Ketamine Sickle Cell Disease
Sickle cell disease (SCD) often results in acute vaso-occlusive crisis (VOC), an obstruction of blood vessels resulting in ischemic injury and pain.
- Trial name
- SCD (NCT03502421)
- Testing
- Ketamine
- Sponsor
- University of South Florida
Venous Ulcer, Sickle Cell Ulcer
Fetal Cell Receptors Repertoire
The purpose of this study is to describe the transcriptomic profile of foetal cells in post-partum and more specifically to determine which chemokine receptors are overexpressed in foetal cells in post-partum women with wounds To…
- Conditions
- Venous Ulcer, Sickle Cell Ulcer, Diabetic Ulcer, Post-partum Women
- Trial name
- MoreCCR (NCT06031714)
- Testing
- Saliva sampling, Blood sampling, Interviews
- Where
- France
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Codeine in Sickle Cell Disease
The objective of this study is to determine if a subject's genetic make-up would affect the treatment response to codeine in subjects with sickle cell disease.
- Trial name
- NCT00174538
- Testing
- Codeine (30 mg)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- PriCara, Unit of Ortho-McNeil, Inc.
Chronic Pain
Effectiveness, Safety, and Tolerability Study of Oxymorphone Immediate Release (IR) Oral Liquid in Opioid Tolerant Pediatric Subjects
The purpose of this study is to evaluate the effectiveness, tolerability, and safety of oxymorphone immediate release (IR) oral liquid as an analgesic in pediatric subjects having severe to moderate chronic pain.
- Trial name
- NCT01206907
- Testing
- oxymorphone IR
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Anemia, Sickle Cell
An Assessment of Prasugrel on Healthy Adults and Sickle Cell Adults
The purpose of this study is to measure the exposure to prasugrel's active metabolite and the pharmacodynamic effects of prasugrel treatment in people with Sickle Cell Disease (SCD).
- Trial name
- NCT01178099
- Testing
- Prasugrel
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Brain Development Abnormality, Sickle Cell Disease
Brain Structure and Neurocognitive Development in Sickle Cell Disease; a Longitudinal Cohort Study (BRICK Study)
Sickle cell disease (SCD) is an autosomal recessive red blood cell blood disorder. One especially vital organ affected in SCD is the brain.
- Trial name
- BRICK (NCT05564845)
- Testing
- MR scan of the brain, Blood work, Neurological examination
- Where
- 2 sites in Netherlands
- Volunteers
- 84 people
- Sponsor
- Dr. Marjon H. Cnossen MD PhD
Sickle Cell Disease, Sickle Cell Crisis
A Comprehensive Care Plan for Pediatric Patients With Vaso-Occlusive Crises
Sickle cell disease (SCD) is the most common inherited blood disorder affecting 80,000 to 90,000 individuals in the United States.[10] There are 13,000 hospital admissions for a sickle cell crises, costing $448 million dollars…
- Conditions
- Sickle Cell Disease, Sickle Cell Crisis, Vaso-Occlusive Crises
- Trial name
- NCT02698761
- Testing
- Comprehensive Care Plan
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Valley Anesthesiology Consultants
Anemia, Sickle Cell
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor…
- Trial name
- NCT04091737
- Testing
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- CSL Behring
Sickle Cell Anemia
Serial Assessment of Fertility Experiences
The SAFE study is a long-term research project that watches people with sickle cell anemia (SCA) over time. The main goal is to see how a medicine called hydroxyurea affects their growth, puberty, and ability to have children.
- Trial name
- SAFE (NCT07116772)
- Where
- 3 sites in Dominican Republic, Jamaica and 1 more
- Volunteers
- 250 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Cognitive Impairment
Minocycline In Neurocognitive Outcomes - Sickle Cell Disease
Sickle cell disease (SCD) is a common, inherited blood disorder that primarily affects people of African Ancestry. It has a lot of complications including neurological complications.
- Conditions
- Sickle Cell Disease, Cognitive Impairment, Cognitive Decline, Cognitive Change
- Trial name
- MINO-SCD (NCT05605366)
- Testing
- Minocycline
- Volunteers
- 30 people
- Sponsor
- University of Cincinnati
Sickle Cell Disease
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare…
- Trial name
- NCT05810181
- Testing
- Interview
- Where
- United States
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Hemoglobinopathy, Sickle Cell Disease
A Long-term Follow-up Study in Patients Who Received BEAM-101
This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing…
- Trial name
- NCT07373639
- Testing
- Long-Term Follow-up Study of patients who received BEAM-101
- Where
- 17 sites in United States
- Volunteers
- 50 people
- Sponsor
- Beam Therapeutics Inc.
Hematologic Malignancy, Solid Tumor
Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular…
- Conditions
- Hematologic Malignancy, Solid Tumor, Sickle Cell Disease, Aplastic Anemia
- Trial name
- NCT06689800
- Testing
- Let's Get REAL
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease
Intranasal Ketamine for Pain Control in Patients with Sickle Cell Disease and Vaso-occlusive Episode (VOE) in the PED
This will be a descriptive cohort study of intranasal ketamine as the initial analgesic for children with sickle cell disease who present to the pediatric emergency department with vaso-occlusive crisis and are awaiting…
- Conditions
- Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis, Ketamine
- Trial name
- NCT04150757
- Testing
- Intranasal ketamine
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Abigail Nixon
Sickle Cell Disease, Asthma
Molecular Phenotyping of Asthma in Sickle Cell Disease
Asthma and sickle cell disease each are serious medical problems. People with asthma have difficulty breathing, wheeze (a whistling noise when breathing), cough, produce sputum or phlegm, and have inflammation (swelling…
- Trial name
- MoP-ASC (NCT01879592)
- Where
- 3 sites in United States
- Volunteers
- 99 people
- Sponsor
- Nemours Children's Clinic
Anemia, Sickle Cell, Hemoglobin SC Disease
Effectiveness of a Computerized Tool (PAINRelieveIt) to Help Manage Pain Related to Sickle Cell Disease
Sickle cell disease (SCD) is a blood disorder that is characterized by intense, painful episodes known as sickle cell crises. This study will evaluate the effectiveness of PAINRelieveIt, a three-part computer-based pain…
- Trial name
- NCT00600665
- Testing
- PAINReportIt (Phase 1 + 2), PAINUCope (Phase 1), PAINConsultN (Phase 2)
- Where
- United States
- Volunteers
- 279 people
- Sponsor
- University of Illinois Chicago
Sickle Cell Crisis
Sickle Cell Improvement: Enhancing Care in the Emergency Department
Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 36,000 children in the United States, approximately 90% of whom are Black.
- Trial name
- SCIENCE (NCT05373771)
- Testing
- Care pathway
- Where
- United States
- Volunteers
- 5,328 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease
Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)
This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD).
- Trial name
- EMPOWER-AYA (NCT05383911)
- Testing
- SCD Shared Decision-Making Toolkit for AYAs - including VR, SCD Shared Decision-Making Toolkit for AYAs - without VR
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Nemours Children's Clinic