Clinical trials: Congenital Bleeding Disorder, 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
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
Dengue Disease, Dengue Haemorrhagic Fever
International Registry of Dengue Infection in Congenital Bleeding Disorders (DengueCBDR)
Dengue fever, a viral infection transmitted by Aedes mosquitoes, is a major health issue in tropical and subtropical regions. Around 20-30% of symptomatic patients developed Dengue Hemorrhagic Fever (DHF), which leads to impaired…
- Conditions
- Dengue Disease, Dengue Haemorrhagic Fever, Dengue Fever, Dengue Fever With Warning Signs
- Trial name
- DengueCBDR (NCT07409857)
- Where
- Thailand
- Volunteers
- 100 people
- Sponsor
- Mahidol University
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
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
Cerebral Arteriovenous Malformation, Intracranial Arterial Diseases
Cerebral Arteriovenous Malformation With Aneurysm: Epidemiology, Clinical Features, and Prognosis
The study is a multicenter, prospective cohort study designed to assess the natural history, clinical safety and efficacy of various treatment strategies in patients with cerebral arteriovenous malformations (AVMs) and…
- Conditions
- Cerebral Arteriovenous Malformation, Intracranial Arterial Diseases, Intra Cerebral Hemorrhage, Intracranial Aneurysm
- Trial name
- NCT07353736
- Where
- China
- Volunteers
- 630 people
- Sponsor
- Duan Chuanzhi
Hemophilia
Feasibility and Safety of Blood-Flow-Restriction Training in Patients With Hemophilia
In the course of a cross-over study design, 12 patients with hemophilic arthropathy will perform a training load on the knee extensor muscles.
- Trial name
- NCT07259356
- Testing
- Control, BFR Exercise (50% of the LOP) while Leg Press Exercise (4 Sets, All out)
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- University Hospital, Bonn
Hemophilia A
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients.
- Trial name
- NCT06238908
- Testing
- NGGT003
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
MRI, Hemophilia
MRI Role in Knee Hemophilic Arthopathy
The aim of this study is to assess role of MRI in detecting synovial, cartilaginous , osseous abnormalities ، bleeding inside knee joint and to use a system for assessing HA as support for therapeutic regimes and for monitoring…
- Conditions
- MRI, Hemophilia, Arthropathy Hemophilic, Arthropathy of Knee
- Trial name
- NCT07233122
- Volunteers
- 95 people
- Sponsor
- Assiut University
Hemophilia B
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Trial name
- IX-TEND 3003 (NCT05962398)
- Testing
- AAV5-hFIXco-Padua
- Where
- 28 sites in United States, Belgium and 6 more
- Volunteers
- 56 people
- Sponsor
- CSL Behring
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
The World Federation of Hemophilia Gene Therapy Registry
The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
- Trial name
- WFH GTR (NCT04883710)
- Testing
- Gene therapy
- Volunteers
- 5,000 people
- Sponsor
- World Federation of Hemophilia
Thrombotic Thrombocytopenic Purpura (TTP)
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce…
- Trial name
- NCT07353099
- Testing
- No Intervention
- Where
- 7 sites in Austria, Germany
- Volunteers
- 50 people
- Sponsor
- Takeda
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Systemic Lupus Erythematosus, Inflammatory Myopathy
BCMA-CD19 CAR-T Therapy for Refractory Autoimmune Diseases
The objective of this study is to evaluate the efficacy and safety of BCMA/CD19 chimeric antigen receptor (CAR)-modified T cells in the treatment of autoimmune diseases.
- Conditions
- Systemic Lupus Erythematosus, Inflammatory Myopathy, Systemic Sclerosis (SSc), ANCA-associated Vasculitis
- Trial name
- NCT06794008
- Testing
- BCMA-CD19 CAR-T therapy
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Peking University People's Hospital
Chronic Myeloproliferative Disorders, Cognitive/Functional Effects
Registry of Older Patients With Cancer
RATIONALE: Gathering information about older patients with cancer may help the study of cancer in the future. PURPOSE: This research study is gathering information from older patients with cancer into a registry.
- Conditions
- Chronic Myeloproliferative Disorders, Cognitive/Functional Effects, Leukemia, Lymphoma
- Trial name
- NCT01137825
- Where
- 7 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Hemophilia A
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
This is an observational study of children with mild, moderate, or severe hemophilia A who are receiving damoctocog alfa pegol, and are between 7 to <12 years of age at the time of enrolment.
- Trial name
- HEM-POWR Kids (NCT07088458)
- Testing
- Damoctocog alfa pegol (Jivi, Bay94-9027)
- Where
- 12 sites in United States, Brazil and 9 more
- Volunteers
- 40 people
- Sponsor
- Bayer
Factor X Deficiency
A Database For The Collection Of Clinical, Laboratory, And Treatment Data From Patients With Factor X Deficiency To Define The Natural History Of The Disease And Improve Patient Management
Factor X deficiency is one of the most severe rare bleeding disorders and may lead to serious spontaneous hemorrhages, particularly in patients with severe congenital forms.
- Trial name
- PRO-RBDD-FX (NCT07875478)
- Where
- 2 sites in Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Fondazione Luigi Villa
Hemophilia A Patient
Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A
Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly…
- Trial name
- E-PROTECT (NCT07830693)
- Testing
- Study questionnaires, Clinical/pharmacological data collection
- Where
- 8 sites in France
- Volunteers
- 553 people
- Sponsor
- Nantes University Hospital