Clinical trials: Hemoglobinopathies
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
Liver Iron Overload
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Trial name
- NCT01033747
- Testing
- Deferasirox
- Where
- 4 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals
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
Iron Overload, Beta-Thalassemia
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Trial name
- NCT02274233
- Testing
- SP-420
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 24 people
- Sponsor
- Sideris Pharmaceuticals
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
Thalassemia, Iron Overload
Evaluation of the Quality of Life in Patients With Chronic Iron Overload Due to Hemoglobinopathies in Greece.
This observational clinical study aims to evaluate the HRQoL of thalassemia patients with iron overload in Greece, who are under treatment with deferasirox based on standard clinical practice.
- Trial name
- FEROUSA (NCT07352878)
- Volunteers
- 150 people
- Sponsor
- Elpen Pharmaceutical Co. Inc.
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Post Hematopoietic Stem Cell Transplantation
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant…
- Trial name
- NCT01610297
- Testing
- ICL670
- Where
- 6 sites in Turkey (Türkiye)
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
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
Transfusion-dependent Thalassemia, Non-transfusion-dependent Thalassemia
Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet
Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox dispersible tablet (DT) in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by…
- Trial name
- Jupiter (NCT02993224)
- Testing
- Deferasirox dispersable tablet (DT), Deferasirox film coated tablet (FCT)
- Where
- 17 sites in Egypt, Lebanon and 5 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals