Clinical trials: Hemolytic Anemia
Atypical Hemolytic Uremic Syndrome(aHUS)
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
This is a China, non-interventional, observational study and will follow the Good Phar-macoepidemiology Practices guidelines. This study will enrol paediatric and adult patients diagnosed with aHUS who will be treated according…
- Trial name
- NCT06099236
- Where
- 26 sites in China
- Volunteers
- 367 people
- Sponsor
- AstraZeneca
Paroxysmal Nocturnal Hemoglobinuria
rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study
Long term management of patients with complement related diseases including Paroxysmal Nocturnal Haemoglobinuria and Atypical Haemolytic Uraemic Syndrome
- Trial name
- CONSERVE (NCT03829449)
- Testing
- rVA576
- Where
- Poland
- Volunteers
- 15 people
- Sponsor
- AKARI Therapeutics
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Thalassemia
Development of THALEA Kit and Its Impact on Knowledge, Attitude, and Intention for Premarital Thalassemia Screening
Thalassemia, a common genetic disease, is often overlooked compared to infectious diseases like HIV, despite its significant effects for families planning to have children.
- Trial name
- THALEA (NCT06800495)
- Testing
- THALEA (Thalassemia Education and Awareness) Kit, Basic thalassemia and family health education
- Where
- Malaysia
- Volunteers
- 148 people, healthy volunteers welcome
- Sponsor
- Universiti Teknologi Mara
Multiple Myeloma, Acute Myeloid Leukemia
Effects of Prebiotics on Gut Microbiome in Patients Undergoing HSCT
The purpose of this study is to see whether hematopoietic stem cell transplant (HSCT) patients can consistently eat a diet rich in prebiotics.
- Conditions
- Multiple Myeloma, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Non Hodgkin Lymphoma
- Trial name
- HCTDiet (NCT04629430)
- Testing
- Pre-biotic foods/drinks
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Virginia
Sickle Cell Disease, Acute Chest Syndrome
Tocilizumab for Acute Chest Syndrome
The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease.
- Trial name
- NCT05640271
- Testing
- Tocilizumab
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- University of Chicago
Paroxysmal Nocturnal Hemoglobinuria
To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH.
- Trial name
- NCT05476887
- Testing
- KP104
- Where
- 4 sites in China
- Volunteers
- 35 people
- Sponsor
- Kira Pharmacenticals (US), LLC.
Immune Thrombocytopenia
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
This study will evaluate the safety and efficacy of tafasitamab in adult participants with primary autoimmune blood cell disorders.
- Trial name
- NCT07104565
- Testing
- INCA000585
- Where
- 39 sites in United States, Australia and 5 more
- Volunteers
- 56 people
- Sponsor
- Incyte Corporation
Hemolytic-Uremic Syndrome
Shiga Toxin Producing Escherichia Coli (STEC) Volume Expansion
This study will provide feasibility data regarding the conduct of a clinical trail evaluating the use of early aggressive inpatient intravenous rehydration in children with Shiga Toxin producing E. coli infection.
- Trial name
- NCT03275792
- Testing
- D5-0.9%NS, Routine home oral rehydration
- Where
- Canada
- Sponsor
- University of Calgary
Thalassemia Major, Thalassemia Intermedia
Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia
This is a multicenter observational case-control analysis lasting 12 months aimed at determining the prevalence of pulmonary hypertension (PAH) in patients with Thalassemia Major and Intermedia.
- Conditions
- Thalassemia Major, Thalassemia Intermedia, Pulmonary Arterial Hypertension
- Trial name
- PAH2010 (NCT01496963)
- Testing
- Physician standard-of-care according to ESC/ERS Guidelines
- Where
- 8 sites in Italy
- Volunteers
- 1,500 people
- Sponsor
- Ente Ospedaliero Ospedali Galliera
Beta Thalassemia Major Anemia
Thrombin Generation in Beta-thalassemia Major
This study aims to evaluate the persistence of a hypercoagulable state in chronically-transfused patients with beta thalassemia major, by using the thrombin generation test (performed in whole blood and plasma).
- Trial name
- TG-THAL (NCT04962984)
- Testing
- blood sampling
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Central Hospital, Nancy, France
Sickle Cell Disease
SMYLS Multi-site Trial
The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms.
- Trial name
- NCT06035939
- Testing
- Voice Crisis Alert V2, Electronic educational materials
- Where
- United States
- Volunteers
- 272 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Pain
The Effect of Acupressure on Pain in Children With Thalassemia
This randomized controlled trial evaluates the effect of acupressure on reducing the pain of blood collection in children with Thalassemia. The hypothesis of this study is that acupressure application reduces acute pain.
- Trial name
- acupressure (NCT06163196)
- Testing
- acupressure
- Where
- Turkey (Türkiye)
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Mersin University
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the…
- Trial name
- NCT05741346
- Testing
- BCX9930
- Where
- 11 sites in France, Hungary and 5 more
- Volunteers
- 28 people
- Sponsor
- BioCryst Pharmaceuticals
Sickle Cell Disease
Utilizing A Single Session Problem-Solving Intervention With Caregivers of Pediatric Patients Receiving Chronic Transfusion to Treat Sickle Cell Disease
This study seeks to utilize an innovative approach of a single session problem-solving intervention to address psychosocial factors affecting patient outcomes within the pediatric sickle cell population.
- Trial name
- NCT04606160
- Testing
- Single Session Problem-Solving Intervention
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Sickle Cell Disease
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD).
- Trial name
- NCT03263559
- Testing
- Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG
- Where
- 32 sites in United States
- Volunteers
- 95 people
- Sponsor
- Medical College of Wisconsin
Hemolytic Anemia, Thalassemia
Native T1 Mapping and ECV in Chronic Hemolytic Anemia
This cross-sectional study aims to evaluate the diagnostic performance of native T1 mapping and extracellular volume fraction for the detection of myocardial iron overload and fibrosis in patients with chronic hemolytic anemia…
- Conditions
- Hemolytic Anemia, Thalassemia, Iron Overload
- Trial name
- NCT07882303
- Volunteers
- 54 people
- Sponsor
- Assiut University
Sickle Cell Crisis, Sickle Cell Disease
Mindfulness and Yoga Therapy for Acute Pain in Sickle Cell Disease
Patients with sickle cell disease suffer from acute and chronic pain that diminishes their quality of life. The purpose of this study is to assess the feasibility and acceptability of mindfulness meditation, breathing exercises…
- Trial name
- NCT05572294
- Testing
- Mindfulness meditation, breathing exercises (including incentive spirometry), and yoga therapy
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- University of Texas Southwestern Medical Center
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Autoimmune Hemolytic Anemia, CD19/BCMA CAR T-cells
CAR T-cell Therapy Targeting CD19 and BCMA in Patients with AIHA Who Have Failed ≥3 Lines of Therapy.
This is an investigator-initiated trial to evaluate the safety and efficacy of universal allogeneic anti-CD19/BCMA CAR T-cells in AIHA who have failed ≥ 3 lines of therapy.
- Conditions
- Autoimmune Hemolytic Anemia, CD19/BCMA CAR T-cells, Universal Allogeneic CAR T-cells
- Trial name
- NCT06733610
- Testing
- universal allogeneic anti-CD19/BCMA CAR T-cells
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China