Clinical trials: Hematologic Disorders, open to volunteers
Hemophilia A, Prophylaxis of Bleeding
A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A
In this study, researchers will observe and study the data from participants with hemophilia A who receive damoctocog alfa pegol as prescribed by their doctors.
- Conditions
- Hemophilia A, Prophylaxis of Bleeding, Treatment of Bleeding
- Trial name
- NCT06222697
- Testing
- Damoctocog-alfa-pegol (Jivi, BAY94-9027)
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Bayer
Sickle Cell Disease
Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening
Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-long debilitation, evidence-based strategies to control SCD are not…
- Trial name
- SIMCS SCD (NCT06563440)
- Testing
- Digital app and information system
- Where
- 2 sites in United States, Uganda
- Volunteers
- 24,000 people, healthy volunteers welcome
- Sponsor
- Makerere University
Acute Lymphoblastic Leukemia ALL
Tracjectories and Predictors of Chemotherapy Induced Peripheral Neuropathy in Children With Acute Lymphoblastic Leukemia
The goal of this study is to explore the trajectory patterns of chemotherapy induced peripheral neuropathy over the course of chemotherapy and identify predictors of distinct trajectories in children with acute lymphoblastic…
- Trial name
- NCT07326930
- Where
- 3 sites in China
- Volunteers
- 173 people
- Sponsor
- The Hong Kong Polytechnic University
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Eosinophilic Esophagitis
Pediatric Eosinophilic Esophagitis (pedEoE): Effect of Allergen Heat Denaturation on EoE Remission: a Pilot Trial
The objective of the study is to study whether the introduction of heated food products (more specifically heated hen's egg and/or cow's milk) in children with EoE would be possible without re-occurrence of the eosinophilic…
- Trial name
- NCT06381219
- Testing
- Implementation of cow's milk in decreasingly heated forms, Implementation of hen's egg in decreasingly heated forms
- Where
- Belgium
- Volunteers
- 36 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Solid Tumors, Hematological Tumor
Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients
The goal of this clinical trial is to evaluate the efficacy and safety of romiplostim N01 in treating chemotherapy-induced thrombocytopenia (CIT) in children and adolescents/young adults (CAYA; aged 6-24 years) with hematologic…
- Conditions
- Solid Tumors, Hematological Tumor, Children, Adolescent
- Trial name
- NCT07043894
- Testing
- Romiplostim N01
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Diet Intervention, Physical Activity Intervention
Obesity Prevention in Children and Young People Treated for Acute Lymphoblastic Leukaemia with ALLTogether
Background Leukaemia is the most common cancer in children with 800 diagnoses per year in England. To survive, children need strong treatments like chemotherapy and steroids given usually through clinical trials.
- Conditions
- Diet Intervention, Physical Activity Intervention, Standard Care Control
- Trial name
- BREVARY (NCT06846294)
- Testing
- Diet, Diet and physical activity
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 90 people
- Sponsor
- University of Exeter
Leukaemia (Acute Lymphoblastic), Leukaemia (Acute Myeloid)
Phase I Clinical Trial of ThINKK Adoptive Immunotherapy After Allogeneic Hematopoietic Transplantation in Children With Leukemia or Neuroblastoma
A first-in-class adoptive immunotherapy we called ThINKK, for Therapeutic Inducers of Natural Killer (NK) cell Killing, have been designed for use after hematopoietic stem cell transplantation (HSCT), where the proper stimulation…
- Conditions
- Leukaemia (Acute Lymphoblastic), Leukaemia (Acute Myeloid), Neuroblastoma, Neuroblastoma, Metastatic
- Trial name
- ThINKK-01 (NCT07518654)
- Testing
- Therapeutic Inducers of Natural Killer Killing (ThINKK)
- Where
- 2 sites in Canada
- Volunteers
- 12 people
- Sponsor
- Michel Duval
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Severe Asthma, Asthma in Children
Severe Eosinophilic Asthma Phenotypes During Childhood Have Various Origins
Study of the clinical evolution at 10 years of children from the SAMP cohort (severe asthma, eosinophilic or not, allergic or not) in order to understand the different possible evolutions of these phenotypes at different ages.
- Trial name
- SAMP 2 (NCT05762627)
- Where
- France
- Volunteers
- 360 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Iron Deficiency Anemia
Short-term Intravenous Iron Isomaltose Anhydride for IDA
This prospective, randomized, controlled study aims to evaluate the impact of short-term intravenous iron isomaltose anhydride on postoperative recovery and the requirement for allogeneic red blood cells (RBC) transfusion in…
- Trial name
- NCT03915327
- Testing
- Intravenous iron isomaltose anhydride
- Volunteers
- 1,600 people
- Sponsor
- West China Hospital
Severe Aplastic Anemia, Hypo-Plastic MDS
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.
- Conditions
- Severe Aplastic Anemia, Hypo-Plastic MDS, Myelodysplastic Syndrome (MDS)
- Trial name
- NCT03173937
- Testing
- Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent System
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Blood Coagulation Disorders, Inherited, Thrombotic Disorder
Evaluation of Hemostasis in Bleeding and Thrombotic Disorders
The objective of this study will be to assess the coagulation system in-vitro in a variety of bleeding and clotting disorders using the ROTEG analyzer and the thrombin generation assay.
- Trial name
- NCT00178594
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- The University of Texas Health Science Center, Houston
Hematologic Malignancy, AML
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).
- Conditions
- Hematologic Malignancy, AML, ALL, BPDCN
- Trial name
- NCT05476770
- Testing
- Tagraxofusp, Fludarabine, Cytarabine
- Where
- 31 sites in United States, Australia
- Volunteers
- 54 people
- Sponsor
- Therapeutic Advances in Childhood Leukemia Consortium
Invasive Pulmonary Fungal Disease
Blood mNGS for Diagnosing Invasive Pulmonary Fungal Disease in Hematologic Patients
The goal of this clinical trial is to learn whether a blood test called metagenomic next-generation sequencing (mNGS) can help diagnose invasive pulmonary fungal disease in patients with blood disorders.
- Trial name
- NCT07511595
- Testing
- Blood microbial metagenomic next-generation sequencing (mNGS)
- Volunteers
- 1,000 people
- Sponsor
- Peking University People's Hospital
ITP - Immune Thrombocytopenia
"Cytotoxic T Lymphocyte-Associated Antigen-4 (CTLA-4) Gene Single-nucleotide Polymorphism in Primary Immune Thrombocytopenic Purpura in Children"
the investigators aim in this study to investigate the potential association of single gene polymorphisms of CTLA-4 (SNPs; rs: 3087243) using real-time PCR in children with primary ITP.
- Trial name
- ITP (NCT07014904)
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Sohag University
Hemophilia A
Lentiviral FVIII Gene Therapy
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene…
- Trial name
- NCT03217032
- Testing
- YUVA-GT-F801
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Acute Leukemia, Myelodysplastic Syndrome
Study of QUAIL-100 in Pediatric and Young Adult Participants With High-Risk Hematologic Malignancies Who Have Received a Hematopoietic Stem Cell Transplantation
Study of QUAIL-100 in Patients With High Risk Acute Leukemia and Myelodysplastic Syndrome Who Have Received Hematopoietic Stem Cell Transplant
- Trial name
- NCT07573111
- Testing
- QUAIL-100
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Laguna Biotherapeutics, Inc.
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