Clinical trials: Severe Anaemia
Aplastic Anemia
A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia
The aim of this study was to investigate the safety, tolerability, and effectiveness of eltrombopag in combination with immunosuppressive therapy (IST) in real world practice in pediatric patients with severe aplastic anemia…
- Trial name
- NCT07415083
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
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
Beta Thalassemia Major
Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation
A pilot study to explore safety and efficacy of NBMI treatment in patients with Beta Thalassemia Major requiring iron chelation Investigational product: NBMI (N1,N3-bis(2-mercaptoethyl) isophthalamide), INN: Emeramide Indication…
- Trial name
- EMERA007 (NCT04092205)
- Testing
- Emeramide
- Where
- Albania
- Volunteers
- 13 people
- Sponsor
- EmeraMed
Sickle Cell Disease
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease.
- Trial name
- Restore (NCT04819841)
- Testing
- nula-cel Drug Product
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Kamau Therapeutics
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
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Fallopian Tube, Primary Peritoneal Cancer
Study of ABT-767 in Subjects With Breast Cancer 1 and Breast Cancer 2 (BRCA 1 and BRCA 2) Mutations and Solid Tumors or High Grade Serous Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
This is a Phase 1, dose escalation trial evaluating the tolerability, pharmacokinetics, and pharmacodynamics of ABT-767 in subjects with advanced Breast Cancer 1 or 2 gene (BRCA1 or BRCA2)-mutated solid tumors and high grade…
- Conditions
- Fallopian Tube, Primary Peritoneal Cancer, Solid Tumors (e.g. Breast, Ovarian, Prostate, or Pancreatic) and Ovarian
- Trial name
- NCT01339650
- Testing
- ABT-767
- Where
- 3 sites in Netherlands
- Volunteers
- 93 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Aplastic Anemia
Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA).
- Trial name
- NCT03988608
- Testing
- Eltrombopag
- Where
- 5 sites in China
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Beta-Thalassemia, Hemosiderosis
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions.
- Trial name
- NCT00171171
- Testing
- deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 252 people
- Sponsor
- Novartis
Cancer, Breakthrough Pain
Efficacy/Safety Study of ACTIQ® for Opioid-Tolerant Children and Adolescents With Breakthrough Pain (BTP)
The primary objective of the study is to evaluate the efficacy of ACTIQ treatment for the management of breakthrough pain (BTP) compared to placebo treatment in children with cancer and non-cancer pain who are receiving…
- Trial name
- NCT00236041
- Testing
- ACTIQ®
- Where
- 31 sites in United States, Puerto Rico
- Volunteers
- 38 people
- Sponsor
- Cephalon
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
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.
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
Severe Aplastic Anemia (SAA)
REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the…
- Trial name
- NCT04409080
- Testing
- REGN7257
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 17 people
- Sponsor
- Regeneron Pharmaceuticals
Treatment-naive Severe Aplastic Anemia
Extension Study of Hetrombopag in Severe Aplastic Anemia
This is a multicenter, placebo-control, phase 3 study of hetrombopag in patients with treatment-naive severe aplastic anemia. All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate…
- Trial name
- NCT04961710
- Testing
- Hetrombopag Olamine
- Where
- China
- Volunteers
- 157 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Acute Anemia
A Phase 1 Safety Study of SANGUINATE™ In Patients With Acute Severe Anemia.
In this open-label study, eligible and consenting adult patients admitted to the hospital for critical care that have acute severe anemia, defined as a blood hemoglobin level ≤ 5 g/dL, but who are unwilling or unable to receive…
- Trial name
- NCT02754999
- Testing
- SANGUINATE™
- Where
- 32 sites in United States
- Volunteers
- 103 people
- Sponsor
- Prolong Pharmaceuticals
Anemia, Blood Transfusion
A Study to Determine Whether Epoetin Alfa Can Reduce the Need for Blood Transfusions in Patients During the Period of Time Around Major Orthopedic Surgery.
The purpose of this study is to evaluate the safety of epoetin alfa and to determine whether epoetin alfa will reduce the need for blood transfusions during the period of time around major orthopedic surgery.
- Conditions
- Anemia, Blood Transfusion, Orthopedic Procedures
- Trial name
- NCT00270088
- Testing
- epoetin alfa
- Volunteers
- 316 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.