Clinical trials: Severe Anemia
Beta-Thalassemia, Thalassemia
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD).
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn
- Trial name
- NCT05477563
- Testing
- CTX001
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pulmonary Hypertension, Lung Disease
Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide
The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients.
- Conditions
- Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant
- Trial name
- NCT00041574
- Testing
- Inhaled Nitric Oxide
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Mallinckrodt
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
Thalassemia Major
Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation
This study is a multicenter, open-label, two-period crossover design that evaluates the safety, tolerability, pharmacokinetics and preliminary evidence of iron chelating activity of DST-0509 as compared to Jadenu and Exjade in…
- Trial name
- NCT03637556
- Testing
- DST-0509, Jadenu, Exjade
- Where
- 4 sites in United States, Thailand
- Volunteers
- 25 people
- Sponsor
- DisperSol Technologies, LLC
Advanced Solid Tumors
A Phase 1/2 Trial SRA737 in Combination With Gemcitabine and Cisplatin or Gemcitabine Alone in Advanced Cancer Subjects
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose (RP2D) and schedule of SRA737 in combination with low dose gemcitabine; and to…
- Trial name
- NCT02797977
- Testing
- SRA737, gemcitabine, cisplatin, SRA737, gemcitabine
- Where
- 20 sites in Spain, United Kingdom
- Volunteers
- 153 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Sickle Cell Disease, Hemoglobinopathies
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
- Trial name
- NCT04853576
- Testing
- EDIT-301
- Where
- 24 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Editas Medicine, Inc.
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Severe Aplastic Anemia
A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient
This is a Multicentre, Open-label, single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia. 55 adult patients with SAA will be enrolled in the study.
- Trial name
- NCT03557099
- Testing
- Hetrombopag Olamine
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Thalassemia Major, Hemosiderosis
Efficacy Study in Removing Excess Iron From the Heart
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
- Trial name
- NCT00105495
- Testing
- Ferriprox (deferiprone), Desferal (deferoxamine)
- Where
- 4 sites in Greece, Italy
- Volunteers
- 60 people
- Sponsor
- ApoPharma
Transfusional Iron Overload, β-thalassemia Major
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed.
- Conditions
- Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
- Trial name
- NCT00390858
- Testing
- Deferasirox
- Where
- 4 sites in France, Italy
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Beta-Thalassemia Major
A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia.
- Trial name
- NCT06291961
- Testing
- CS-101 injection
- Where
- 3 sites in China
- Volunteers
- 9 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Sickle Cell Disease
Clinical Study of BRL-101 in Severe SCD
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06300723
- Testing
- BRL-101
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Bioray Laboratories
Severe Aplastic Anemia
Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response
- Trial name
- SOAR (NCT02998645)
- Testing
- eltrombopag, Cyclosporine
- Where
- 20 sites in Brazil, Hong Kong and 7 more
- Volunteers
- 54 people
- Sponsor
- Novartis Pharmaceuticals
Transfusion Dependent Beta Thalassemia, Hemoglobinopathies
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
- Conditions
- Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
- Trial name
- NCT05444894
- Testing
- EDIT-301
- Where
- 8 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Editas Medicine, Inc.
Thalassemia Majors (Beta-Thalassemia Major), Osteoporosis
Denosumab Versus Zoledronic Acid in Thalassemia-Induced Osteoporosis
This study is to compare the two medications Denosumab and Zoledronic Acid For Patients With Beta Thalassemia Major Induced Osteoporosis.
- Trial name
- DOHA (NCT03040765)
- Testing
- Denosumab 60 MG/ML Prefilled Syringe, Zoledronic Acid 5Mg/Bag 100Ml Inj
- Where
- Qatar
- Volunteers
- 17 people
- Sponsor
- Hamad Medical Corporation
Anemia, Venous Thrombosis
A Study to Confirm the Safety and Efficacy of Epoetin Alfa (PROCRIT) Administered Perioperatively vs. the Standard of Care in Blood Conservation in Patients Undergoing Major Elective Spinal Surgery (SPINE Study)
This study is designed to investigate the incidence of deep vein thrombosis in patients receiving a perisurgical regimen of epoetin alfa (PROCRIT®) as compared to patients receiving standard of care blood conservation management.
- Trial name
- NCT00211146
- Testing
- epoetin alfa
- Volunteers
- 680 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Leukemia, Lymphoma
Safety Study of Human Myeloid Progenitor Cells (CLT-008) After Cord Blood Transplant for Hematologic Malignancy
Ex vivo expanded human myeloid progenitor cells (hMPCs; CLT-008) have the potential to accelerate neutrophil recovery in patients receiving myeloablative conditioning as part of an umbilical cord blood transplant for hematologic…
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Plasma Cell Neoplasm
- Trial name
- NCT00891137
- Testing
- human myeloid progenitor cells
- Where
- 8 sites in United States
- Volunteers
- 30 people
- Sponsor
- Cellerant Therapeutics
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Severe Postpartum Haemorrhage
Treatment of Women After Severe Postpartum Haemorrhage
The primary purpose of this study is to get explorative information about IV high single dose infusion of iron isomaltoside 1000 compared to RBC transfusion in the treatment of severe PP-IDA evaluated as physical fatigue
- Trial name
- PP-02 (NCT01895205)
- Testing
- Iron isomaltoside 1000, Red blood cell transfusion
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Pharmacosmos A/S
Thalassemia Major
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic…
- Trial name
- NCT05442346
- Testing
- γ-globin reactivated autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Bioray Laboratories