Clinical trials: Blood Coagulation Disorders
Haemophilia A
Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A
Primary objective: To assess post-marketing immunogenicity of Optivate® by monitoring plasma inhibitor levels for at least 100 Exposure Days (EDs) for each subject.
- Trial name
- NCT01811875
- Testing
- Optivate 500IU
- Where
- 4 sites in Colombia, Germany and 1 more
- Volunteers
- 7 people
- Sponsor
- Bio Products Laboratory
Amorphous, Eosinophilic, and Acellular Deposit, Constipation
S1702 Isatuximab in Treating Patients With Relapsed or Refractory Primary Amyloidosis
This phase II trial studies how well isatuximab works in treating patients with primary amyloidosis that has come back or does not respond to treatment.
- Conditions
- Amorphous, Eosinophilic, and Acellular Deposit, Constipation, Diarrhea, Early Satiety
- Trial name
- NCT03499808
- Testing
- Isatuximab, Laboratory Biomarker Analysis
- Where
- 345 sites in United States
- Volunteers
- 43 people
- Sponsor
- SWOG Cancer Research Network
Severe Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A…
- Trial name
- HAVEN 7 (NCT04431726)
- Testing
- Emicizumab
- Where
- 32 sites in United States, Australia and 12 more
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Hemophilia
LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures
The purpose of this study, PerSept 3, is to evaluate LR769 for the prevention of excessive bleeding and achievement of hemostasis in congenital hemophilia A or B patients who have inhibitors to Factor VIII or Factor IX , are aged…
- Trial name
- PerSept3 (NCT02548143)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 9 sites in United States, Mexico and 4 more
- Volunteers
- 12 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Primary Thrombocytopenia
Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.
- Trial name
- NCT01349790
- Testing
- NewGam
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Octapharma
Immune Thrombocytopenic Purpura
Higher Dose of Rituxan Versus Standard Doses of Rituxan With Cyclophosphamide, Vincristine, and Prednisone in Subjects With Chronic ITP
This study is designed to compare the efficacy and safety of higher doses of Rituxan with a regimen combining standard doses of Rituxan + CVP in patients with chronic ITP who did not respond to or relapsed after standard doses of…
- Trial name
- NCT00774202
- Testing
- Rituxan, Cyclophosphamide, Vincristine, Prednisone, Higher Dose of Rituximab
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Weill Medical College of Cornell University
Thrombocytopenia, Idiopathic Thrombocytopenic Purpura
Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)
The purpose of this study is to evaluate changes in bone marrow morphology (structure) after long-term exposure to romiplostim.
- Trial name
- NCT00907478
- Testing
- romiplostim
- Volunteers
- 169 people
- Sponsor
- Amgen
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP)…
- Trial name
- BMN 270-301 (NCT03370913)
- Testing
- valoctocogene roxaparvovec
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 144 people
- Sponsor
- BioMarin Pharmaceutical
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer
This clinical trial studies the side effects and best dose of giving fludarabine and total-body irradiation (TBI) together followed by a donor stem cell transplant and cyclosporine and mycophenolate mofetil in treating human…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00112593
- Testing
- fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Fred Hutchinson Cancer Center
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche
Hemophilia B
Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including: 1) Unknown…
- Trial name
- NCT00484185
- Testing
- BeneFIX (coagulation factor IX (recombinant))
- Where
- South Korea
- Volunteers
- 183 people
- Sponsor
- Pfizer
Ischemic Stroke, Hyperglycemia
Procoagulant Effects of Hyperglycemia After Acute Stroke: A Pilot Study
Between twenty and fifty percent of people who have acute stroke have hyperglycemia (high blood sugar) with it. The purpose of this study is to examine the relationships between diabetes mellitus, hyperglycemia,whole blood tissue…
- Trial name
- NCT00373269
- Volunteers
- 21 people
- Sponsor
- Temple University
Treatment of Bleeding, Prophylaxis of Bleeding
A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Researchers are looking for a better way to treat hemophilia A. Hemophilia A is a genetic disorder where the body does not create enough of a protein called clotting factor 8 (FVIII) present in the blood.
- Conditions
- Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A, Children
- Trial name
- Alfa-PROTECT (NCT05147662)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- 18 sites in United States, Argentina and 5 more
- Volunteers
- 36 people
- Sponsor
- Bayer
Immune Thrombocytopenia
A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)
This was a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count <30,000/μL on two…
- Trial name
- NCT03395210
- Testing
- Rilzabrutinib
- Where
- 31 sites in United States, Australia and 6 more
- Volunteers
- 86 people
- Sponsor
- Principia Biopharma, a Sanofi Company
Hemophilia A
Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq
Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe…
- Trial name
- PREVAIL (NCT03344003)
- Testing
- Wilate or Nuwiq
- Where
- 3 sites in Canada
- Volunteers
- 14 people
- Sponsor
- Octapharma
Primary Immune Thrombocytopenia
A Study of TAK-079 in Adults With Persistent/Chronic Primary Immune Thrombocytopenia
Primary immune thrombocytopenia (ITP) is a rare disease that results in low levels of platelets - the cells that help blood clot.
- Trial name
- NCT04278924
- Testing
- TAK-079
- Where
- 55 sites in United States, Bulgaria and 9 more
- Volunteers
- 41 people
- Sponsor
- Takeda
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Wiskott-Aldrich Syndrome (WAS)
Gene Therapy for Wiskott-Aldrich Syndrome
This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.
- Trial name
- TIGET-WAS (NCT01515462)
- Testing
- TLT003
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Fondazione Telethon
Hemophilia
Patient Reported Outcomes Burdens and Experiences - Phase 2
The Patient Reported Outcomes, Burdens, and Experiences (PROBE) Study aims to develop a new global tool to enhance the direct patient-voice in health care decision-making.
- Trial name
- PROBE (NCT02439710)
- Testing
- Quality of life measure (survey)
- Where
- Canada
- Volunteers
- 656 people
- Sponsor
- McMaster University
Hemophilia B
Registry For Patients Treated With BeneFix In Usual Care Setting In Germany
The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.
- Trial name
- NCT00714415
- Testing
- BeneFIX
- Where
- 21 sites in Austria, Germany
- Volunteers
- 80 people
- Sponsor
- Pfizer