Clinical trials: Blood Coagulation Disorders, Inherited
Hemophilia B
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
- Trial name
- NCT01335061
- Testing
- Nonacog alfa
- Where
- 17 sites in Bulgaria, Canada and 7 more
- Volunteers
- 25 people
- Sponsor
- Pfizer
Haemophilia
Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa
Occurrence of inhibitors to coagulation factor VIII is diagnosed in ~30% patients with haemophilia A. Presence of inhibitor with a titre >5 BU/ml requires the use of by-passing agents: recombinant activated Factor VIIa…
- Trial name
- Thrombus (NCT01856751)
- Where
- 6 sites in Poland
- Volunteers
- 80 people
- Sponsor
- Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
RATIONALE: Infection prophylaxis and management may help prevent cytomegalovirus (CMV) infection caused by a stem cell transplant.
- Conditions
- Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
- Trial name
- NCT01199562
- Testing
- infection prophylaxis and management, laboratory biomarker analysis, flow cytometry
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- City of Hope Medical Center
Hemophilia A, Hemophilia B
Validation and Reliability of the CHO-KLAT in Turkish
The aim of this study is to investigate the Turkish validity and reliability of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool version2.0.
- Trial name
- NCT05439642
- Testing
- Evaluation of health related quality of life
- Where
- 4 sites in Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- Yuzuncu Yil University
Hemophilia A
Individualizing Hemophilia Prophylaxis Using Thromboelastography
Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day.
- Trial name
- NCT02582060
- Testing
- Thromboelastography/Thromboelastometry-Guided Treatment Regimen
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Children's Hospital Los Angeles
Chediak-Higashi Syndrome
Study of Chediak-Higashi Syndrome
Chediak-Higashi syndrome (CHS) is a rare autosomal recessive disorder characterized in its classical form by oculocutaneous albinism, a bleeding diathesis, recurrent infection due to abnormal neutrophil and natural killer cell…
- Trial name
- NCT00005917
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Wiskott-Aldrich Syndrome
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE…
- Trial name
- WASFUP (NCT02333760)
- Testing
- Autologous CD34+ cells transduced with WASP lentiviral vector
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Genethon
Hemophilia
The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median…
- Trial name
- NCT04108260
- Testing
- Albutrepenonacog Alfa 1 UNT [IDELVION]
- Where
- Taiwan
- Volunteers
- 5 people
- Sponsor
- National Taiwan University Hospital
Hemophilia
Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery
This is an interventional, prospective, international, multicenter, single-arm, Phase 3, and sequential efficacy and safety study in adolescents and adults with congenital hemophilia A or B with inhibitors to factor VIII (FVIII)…
- Trial name
- SCOPE HIM (NCT05695391)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 15 sites in United States, Malaysia and 4 more
- Volunteers
- 2 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hemophilia A
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855.
- Trial name
- NCT02615691
- Testing
- PEGylated Recombinant Factor VIII, ITI
- Where
- 91 sites in United States, Austria and 21 more
- Volunteers
- 120 people
- Sponsor
- Baxalta now part of Shire
Chronic Myeloproliferative Disorders, Leukemia
Peripheral Stem Cell Transplant in Treating Patients With High-Risk Leukemia
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00066417
- Testing
- cyclophosphamide, cyclosporine, fludarabine phosphate
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Hemophilia
Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia
The primary goal of this observational study is to determine if: health-related quality of life of boys with moderate/severe hemophilia A who are followed in Canadian pediatric hemophilia treatment centres is significantly…
- Trial name
- NCT06191068
- Where
- Canada
- Volunteers
- 190 people
- Sponsor
- The Hospital for Sick Children
Hypoprothrombinemia
Randomized Controlled Feasibility Trial of the Fearon Algorithm to Improve Management of Unstable Warfarin
Patients on warfarin but with unstable international normalized ratio (INR) will be recruited to a randomized trial comparing dosing based on an algorithm (Fearon algorithm, named after the mathematician Michael Fearon), which…
- Trial name
- NCT02267408
- Testing
- Warfarin adjustment using the Fearon algorithm, Warfarin adjustment using standard dosing
- Where
- Canada
- Volunteers
- 42 people
- Sponsor
- McMaster University
Von Willebrand Disease
IL-11 in Adults With Von Willebrand Disease Undergoing Surgery
The purpose of this study is to determine the efficacy and safety of rhIL-11 when given subcutaneously in adults with type 1 von Willebrand disease undergoing elective surgery or major dental procedure.
- Trial name
- NCT00524225
- Testing
- Neumega (Oprelvekin, Interleukin 11, IL-11)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Margaret Ragni
Hemophilia A, Hemophilia B
Ultrasonography in Hemophilic Joint Disease and Serum Markers
Hemophilia is a bleeding disorder (deficiency of a blood clotting factor/ protein) resulting in bleeding in joints and muscles. As patients continue to bleed into their joints they develop progressive joint damage leading to…
- Conditions
- Hemophilia A, Hemophilia B, Hemarthroses
- Trial name
- NCT02634918
- Testing
- ultrasound
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Northwell Health
Wiskott-Aldrich Syndrome (WAS), X-linked Thrombocytopenia
Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
Funding Source--FDA OOPD. Orphan Product Grant Number--1R01FD004091-01A1 Context: Wiskott-Aldrich syndrome (WAS) is a fatal, devastating disease with ill-defined treatment modalities, which affects young boys.
- Trial name
- WAS (NCT00774358)
- Testing
- Interleukin-2
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Soma Jyonouchi
Haemophilia
Combining Registry Data in Haemophilia: TARGET H
This is an investigator-initiated, multinational, retrospective, non-interventional pilot study conducted in five haemophilia treatment centres from different geographical regions that maintain a local (Algeria, Malaysia) or…
- Trial name
- TARGET-H (NCT03264014)
- Volunteers
- 100 people
- Sponsor
- University of Pretoria
Congenital Bleeding Disorder, Haemophilia A
Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds
This trial is conducted in Africa, Asia, Europe, Japan, and North and South America. The aim of this trial is to evaluate the safety and efficacy of activated recombinant human factor VII analogue (vatreptocog alfa (activated))…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT00486278
- Testing
- eptacog alfa (activated), vatreptacog alfa (activated)
- Where
- 45 sites in United States, Argentina and 16 more
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Covalent Tolerance Induction to Factor VIII-Prediction of Inhibitors in Hemophilia
To correlate the Human Leukocyte Antigen type and genetic defect with hemophilia A.
- Trial name
- NCT00178607
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- The University of Texas Health Science Center, Houston