Clinical trials: Sickle Cell Anemia
Sickle Cell Disease, Sickle Cell Anemia
A Study of HQK-1001 in Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease
- Trial name
- NCT01322269
- Testing
- HQK-1001
- Where
- 16 sites in United States, Canada and 3 more
- Volunteers
- 52 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Sickle Cell Disease
Voxelotor CYP and Transporter Cocktail Interaction Study
This research study is examining multiple doses of voxelotor (a study drug intended for treatment of sickle cell disease) and how it interacts with additional substrates (substrates are drugs or other substances that are…
- Trial name
- NCT05981365
- Testing
- Voxelotor, Bupropion, Repaglinide
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Pfizer
Vaso-occlusive Crisis, Sickle Cell Disease
Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease.
- Trial name
- EPIC (NCT01737814)
- Testing
- Saline, MST-188
- Where
- 72 sites in United States, Belgium and 9 more
- Volunteers
- 388 people
- Sponsor
- Mast Therapeutics, Inc.
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
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
- Trial name
- NCT05036512
- Testing
- GBT021601
- Where
- 4 sites in United States, Australia
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- Pfizer
Healthy, Sickle Cell Anemia
Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease
This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in…
- Trial name
- NCT04255875
- Testing
- PF-07209326
- Where
- 16 sites in United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Pfizer
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease
ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study
As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed.
- Trial name
- NCT04707235
- Testing
- Hydroxycarbamide
- Where
- 87 sites in France, French Guiana and 6 more
- Volunteers
- 2,093 people
- Sponsor
- Theravia
Sickle Cell Disease
Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization
- Trial name
- NCT03943615
- Testing
- Voxelotor
- Where
- 18 sites in United States
- Sponsor
- Pfizer
Sickle Cell Disease
Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)
This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety…
- Trial name
- NCT05789355
- Testing
- NUV001
- Where
- France
- Volunteers
- 12 people
- Sponsor
- LGD
Sickle Cell Disease
European Sickle Cell Disease Cohort - Hydroxyurea
In the context of the Risk Management Plan (RMP), as requested from Addmedica by the EMEA, to collect information about long-term safety of Siklos® (hydroxycarbamide) when used in patients with Sickle Cell Disease.
- Trial name
- ESCORT-HU (NCT02516579)
- Testing
- Siklos
- Where
- 60 sites in France, French Guiana and 5 more
- Volunteers
- 1,906 people
- Sponsor
- Theravia
Sickle Cell Disease
A 3-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) Patients
A long term observational study in sickle cell disease will enhance the understanding of the disease patterns, current transfusion practices, treatments and outcomes in sickle cell disease.
- Trial name
- FISCO (NCT01220115)
- Where
- 42 sites in United States
- Volunteers
- 498 people
- Sponsor
- Novartis
Sickle Cell Disease
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs…
- Trial name
- NCT04247594
- Testing
- Voxelotor
- Where
- 6 sites in United Kingdom
- Volunteers
- 6 people
- Sponsor
- Pfizer
Sickle Cell Disease
Open-Label Extension of Voxelotor
Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.
- Trial name
- NCT04188509
- Testing
- Voxelotor
- Where
- 20 sites in United States, Egypt and 3 more
- Volunteers
- 162 people
- Sponsor
- Pfizer
Sickle Cell Anemia
Evaluation of the AMICUS RBCx System in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the AMICUS Red Blood Cell Exchange (RBCx) System (Exchange and Depletion/Exchange procedures) in patients with sickle cell disease.
- Trial name
- NCT02372877
- Testing
- Amicus Red Cell Exchange in SCD patients
- Where
- 6 sites in United States
- Volunteers
- 83 people
- Sponsor
- Fenwal, Inc.
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
Healthy, Sickle Cell Disease
Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease
The purpose of the study is to determine whether imaging techniques, such as magnetic resonance imaging (MRI), near infrared spectroscopy (NIRS), laser speckle contrast imaging (LSCI), and optical imaging (OI), can detect…
- Trial name
- NCT02447627
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Bioverativ Therapeutics Inc.