Clinical trials: Sickle Cell Anaemia
Iron Overload, Sickle Cell Disease
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron…
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- NCT02443545
- Testing
- Deferiprone
- Where
- 13 sites in United States, Canada and 3 more
- Volunteers
- 134 people
- Sponsor
- ApoPharma
Sickle Cell Disease
Biotin-Acridine Red Cell Exchange Kinetics
The primary objective of the study is to describe the recovery and survival of pathogen-reduced (PR) red blood cells (RBCs) prepared and stored with the INTERCEPT Blood System for RBCs® (hereafter referred to as INTERCEPT RBCs)…
- Trial name
- BARK (NCT07155031)
- Testing
- Pathogen reduced red blood cells
- Sponsor
- Cerus Corporation
Sickle Cell Disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD).
- Trial name
- HIBISCUS KIDS (NCT06198712)
- Testing
- Etavopivat
- Where
- 18 sites in Canada, France and 5 more
- Volunteers
- 95 people
- Sponsor
- Forma Therapeutics, Inc.
Sickle Cell Anemia
Sickle Cell Disease: A Retrospective Chart Review
This study is a retrospective chart review of sickle cell patients and will include patients whom have received blood transfusions and those whom have not.
- Trial name
- NCT01441375
- Where
- 2 sites in United States
- Volunteers
- 261 people
- Sponsor
- Novartis Pharmaceuticals
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Sickle Cell Disease
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
- Trial name
- BEACON (NCT05456880)
- Testing
- BEAM-101
- Where
- 19 sites in United States
- Volunteers
- 15 people
- Sponsor
- Beam Therapeutics Inc.
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
Renal Impairment
Study of GBT021601 in Participants With Renal Impairment
Renal Impairment study of GBT021601.
- Trial name
- NCT05878704
- Testing
- GBT021601
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Pfizer
Anemia, Sickle Cell
Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
- Trial name
- NCT00067080
- Testing
- ICL670, deferoxamine
- Where
- 34 sites in United States
- Volunteers
- 195 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.
- Trial name
- NCT06155500
- Testing
- OTQ923
- Where
- 3 sites in United States
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Characteristics and Treatment Patterns of Patients With Sickle Cell Disease in Globin Research Network for Data and Discovery Registry
This was a retrospective observational cohort study. This study was a secondary analysis of individuals with sickle cell disease (SCD) enrolled in the GRNDaD registry.
- Trial name
- NCT06662812
- Where
- United States
- Volunteers
- 1,665 people
- Sponsor
- Novartis
Pain, Cancer
Extension Study of ACTIQ Treatment for Children and Adolescents With Breakthrough Pain
The objective of the study is to monitor the safety (adverse event data) of longer-term use of ACTIQ (Oral Transmucosal Fentanyl Citrate [OTFC]) treatment in children with pain associated with cancer, sickle cell disease, or…
- Conditions
- Pain, Cancer, Sickle Cell Anemia, Severe Burns
- Trial name
- NCT00236093
- Testing
- ACTIQ (Oral Transmucosal Fentanyl Citrate [OTFC])
- Where
- 28 sites in United States, Canada and 1 more
- Sponsor
- Cephalon
Anemia, Sickle Cell
Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease
The purpose of the study is to determine the maximum tolerated dose, safety and effect on induction of fetal hemoglobin of pomalidomide in patients with Sickle Cell Disease.
- Trial name
- SCD-001 (NCT01522547)
- Testing
- pomalidomide
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Celgene
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
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine).
- Trial name
- ASCENT1 (NCT05405114)
- Testing
- NDec - oral decitabine-tetrahydrouridine, HU - Hydroxyurea
- Where
- 118 sites in United States, Canada and 12 more
- Volunteers
- 96 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease & Thalassemia
Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
- Trial name
- NCT01590628
- Testing
- NiCord
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Gamida Cell ltd
Phase 1 Sickle Cell
Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell Disease
This study is being conducted to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of an investigational drug, PF-04447943, in subjects with stable sickle cell disease with and without co-administration with…
- Trial name
- NCT02114203
- Testing
- PDE9i
- Where
- 23 sites in United States, Belgium and 3 more
- Volunteers
- 30 people
- Sponsor
- Pfizer