Clinical trials: Sickle Cell Disorder
Infections, Streptococcal
Immunization of Children Between 8 Weeks and 2 Years of Age With GSK Pneumococcal Vaccine GSK1024850A
The aim of the study is to evaluate the immunogenicity, safety and reactogenicity of GSK Biologicals' pneumococcal conjugate vaccine GSK1024850A.
- Trial name
- NCT01175083
- Testing
- GSK1024850A (Synflorix), Tritanrix-HepB/Hib, Polio Sabin
- Where
- Burkina Faso
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
- Trial name
- IMPACTS-2 (NCT01522196)
- Testing
- Varespladib
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Anthera Pharmaceuticals
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease
Managed Access Programs for SEG101, Crizanlizumab
The purpose of this registration is to list Managed Access Programs (MAPs) related to SEG101, Crizanlizumab
- Trial name
- NCT03720626
- Testing
- crizanlizumab
- Sponsor
- Novartis Pharmaceuticals
Healthy Adult Subjects, Sickle Cell Disease
Safety, Tolerability and Pharmacokinetics of FTX-6058
This is a study to evaluate the safety, tolerability and pharmacokinetics of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease (SCD).
- Trial name
- NCT04586985
- Testing
- FTX-6058 - Two Dosing Periods, FTX-6058 / Midazolam Syrup
- Where
- 2 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Fulcrum Therapeutics
Sickle Cell Disease
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
Sickle Cell Disease
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with…
- Trial name
- NCT00445978
- Testing
- Sapropterin Dihydrochloride
- Where
- 12 sites in United States
- Volunteers
- 32 people
- Sponsor
- BioMarin Pharmaceutical
Sickle Cell Disease
Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)
The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.
- Trial name
- NCT01783691
- Testing
- NKTT120
- Where
- 6 sites in United States
- Volunteers
- 21 people
- Sponsor
- NKT Therapeutics
Sickle Cell Disease, Iron Overload
Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
- Conditions
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- Trial name
- NCT00110617
- Testing
- Deferasirox (ICL670), Deferoxamine (DFO)
- Where
- 59 sites in United States, Canada
- Volunteers
- 212 people
- Sponsor
- Novartis Pharmaceuticals
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 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
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
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.
Sickle Cell Disease
A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
- Trial name
- NCT03401112
- Testing
- IMR-687
- Where
- 13 sites in United States, United Kingdom
- Volunteers
- 100 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Sickle Cell Disease
Crizanlizumab Pregnancy Outcomes Intensive Monitoring
This was a non-interventional study to evaluate pre-specified pregnancy and infant outcomes in women treated with crizanlizumab during pregnancy or within 105 days before the last menstrual period (LMP).
- Trial name
- PRIM (NCT07554547)
- Where
- Switzerland
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Sickle Cell Anemia
A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.
ICA-17043 is being developed for the chronic treatment of patients with sickle cell disease (SCD) in both adults and children. ICA-17043 is a potent and specific inhibitor of a channel in human red blood cells (RBCs) that blocks…
- Trial name
- NCT00040677
- Testing
- Low Dose ICA-17043, High dose ICA-17043
- Where
- 19 sites in United States
- Volunteers
- 90 people
- Sponsor
- Icagen
Sickle Cell Disease
A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease
The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of Aes-103 (active ingredient 5-hydroxymethyl-2-furfural [5-HMF]) compared with placebo in subjects with stable sickle…
- Trial name
- NCT01597401
- Testing
- Aes-103
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Disease
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
- Trial name
- GBT_HOPE (NCT03036813)
- Testing
- voxelotor
- Where
- 62 sites in United States, Canada and 10 more
- Volunteers
- 449 people
- Sponsor
- Pfizer