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34 Haemophilia B Trials Near You
Power is an online platform that helps thousands of Haemophilia B patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
Learn More About PowerEfanesoctocog Alfa for Hemophilia A
Columbus, Ohio
Primary Objective:
- To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A
Secondary Objectives:
* To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
* To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes.
* To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes.
* To evaluate the effect of BIVV001 prophylaxis on joint health outcomes.
* To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes.
* To evaluate the safety and tolerability of BIVV001 treatment.
* To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B).
* To evaluate the efficacy of BIVV001 for perioperative management
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Liver Disease, Infections, Coagulation Disorders, Others
Must Not Be Taking:Aspirin, Antiplatelets, NSAIDs, Emicizumab
261 Participants Needed
Gene Therapy for Hemophilia A
Columbus, Ohio
This trial tests a new gene therapy combined with anti-inflammatory medication for patients with severe hemophilia A. The treatment aims to help their bodies produce a necessary clotting protein and reduce inflammation.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Sex:Male
Key Eligibility Criteria
Disqualifiers:Liver Disease, Hepatitis B, Hepatitis C, Others
Must Be Taking:Prophylactic Corticosteroids
22 Participants Needed
Concizumab for Hemophilia A and B
Columbus, Ohio
This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.
Participants will have to inject the study medicine every day under the skin with a pen-injector.
The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12+
Key Eligibility Criteria
Disqualifiers:Thromboembolic Disease, Coagulation Disorders, Others
Must Be Taking:Coagulation Factors
153 Participants Needed
Gene Therapy for Hemophilia A
Lexington, Kentucky
This trial tests a gene therapy that uses a harmless virus to deliver a healthy gene to adults with severe haemophilia A. The goal is to help their bodies produce a missing blood-clotting protein, potentially curing their condition. Gene therapy for hemophilia has been explored for many years, with several programs in advanced stages.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Sex:Male
Key Eligibility Criteria
Disqualifiers:HIV, Liver Dysfunction, Hypertension, Others
Must Be Taking:HFVIII Concentrates
14 Participants Needed
Emicizumab for Hemophilia A
Detroit, Michigan
This trial tests emicizumab, a medication that helps blood clot, in people with mild or moderate hemophilia A. It works by mimicking a missing protein to improve blood clotting. Emicizumab has been approved for treatment of hemophilia A patients.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Other Bleeding Disorders, HIV, Others
Must Not Be Taking:Thromboembolic Treatments
73 Participants Needed
Gene Therapy for Hemophilia A
Ann Arbor, Michigan
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Sex:Male
Key Eligibility Criteria
Disqualifiers:Obesity, Hepatitis, HIV, Others
Must Not Be Taking:Antivirals, FVIII Inhibitors
11 Participants Needed
SR604 for Hemophilia
Ann Arbor, Michigan
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors (Part B).
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 60
Sex:Male
Key Eligibility Criteria
Disqualifiers:Thrombosis, Cardiac Disease, Hypertension, HIV, Others
36 Participants Needed
Concizumab for Hemophilia
Detroit, Michigan
This trial tests concizumab, a new daily injectable medicine, in people with haemophilia A or B without inhibitors. The goal is to see if it can safely prevent bleeding episodes. Participants will either start the new medicine immediately or after a period of time, depending on their current treatment plan. Concizumab has shown significant reduction in bleeding rates in previous trials.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Thromboembolic Disease, Coagulation Disorder, Others
156 Participants Needed
NXT007 for Hemophilia A
Indianapolis, Indiana
WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts:
* Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without factor VIII (FVIII) inhibitors.
* Part 2 is a multiple-dose study in pediatric male participants with severe or moderate hemophilia A with or without FVIII inhibitors.
The overall aim of the study is to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:2 - 59
Sex:Male
Key Eligibility Criteria
Disqualifiers:Bleeding Disorders, Thromboembolic Disease, Diabetes, Malignancies, Others
Must Be Taking:Recombinant Activated Factor VII
60 Participants Needed
Gene Therapy for Hemophilia A
Hamilton, Ontario
This trial tests a single treatment for adult males with severe hemophilia A. The treatment aims to help their bodies produce a necessary protein, reducing the need for regular preventive treatments. Research has been ongoing for a long time to enable the body to produce this protein and reduce bleeding episodes.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18 - 64
Sex:Male
Key Eligibility Criteria
Disqualifiers:Liver Disease, Thrombophilia, Hepatitis, HIV, Others
Must Be Taking:Factor VIII
76 Participants Needed
Marstacimab for Hemophilia
Hamilton, Ontario/canada
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.
This study will enroll participants who:
* have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)
* have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study
* if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry
* if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry
All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.
To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.
Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:1 - 17
Sex:Male
Key Eligibility Criteria
Disqualifiers:Coronary Artery, Thrombotic, Ischemic, Others
Must Be Taking:Factor VIII, Factor IX
100 Participants Needed
Marstacimab for Hemophilia
Hamilton, Ontario
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005 and from the Phase 3 Study B7841008.
Study B7841005: approximately 145 adolescent and adult participants 12 to \<75 years of age with severe hemophilia A or moderately severe to severe hemophilia B (defined as FVIII activity \<1% or FIX activity ≤2%, respectively) with or without inhibitors are expected to be enrolled in Study B7841005 during which they will receive prophylaxis (defined as treatment by SC injection of marstacimab).
Study B7841008: this is an ongoing Phase 3, open-label study in pediatric participants \<18 years of age with severe hemophilia A (FVIII Coagulation Factor Activity \<1%) or moderately severe to severe hemophilia B (FIX Coagulation Factor Activity ≤2%). A sequential approach will be used in enrolling at least 100 pediatric participants, at least 20 of which will be aged ≥12 to \<18 years and at least 80 participants will be aged ≥1 to \<12 years. At the start of study B7841008, the dosing and data available in adolescent and adult participants in Study B7841005 supported the initiation of B7841008 study in participants aged ≥12 to \<18 years. Subsequently, additional safety and efficacy data from adolescent participants in Study B7841005 became available for benefit/risk assessment in support of dosing participants aged ≥6 to \<12 years. Based on the positive benefit/risk assessment conducted by both internal Pfizer review and eDMC review, dosing of the ≥6 to \<12 years age group was initiated in June 2023 in B7841008 Study. Data from participants ≥6 years from B7841008 Study and Study B7841005 will support the dosing of participants aged ≥1 to \<6 years.
All participants will be provided the prefilled pen (PFP) for administration of marstacimab in the study. Use of the prefilled syringe (PFS) will be permitted at the investigator's discretion for those participants who have difficulty with administration of the PFP. Additionally, participants will be provided the PFS for use in this study in countries where the PFS is anticipated to be the only presentation available commercially. An optional, open-label, single arm, substudy using the PFP was completed in the first 23 participants rolled over from Study B7841005 who agreed to participate in the substudy.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:1 - 74
Sex:Male
Key Eligibility Criteria
Disqualifiers:Coronary Artery Disease, Thrombosis, Ischemic Disease, Abnormal Renal Function, Unstable Hepatic Function, Others
Must Be Taking:Marstacimab
245 Participants Needed
aPCC-Emicizumab Safety for Hemophilia A
Atlanta, Georgia
This trial uses a special blood-clotting medicine along with emicizumab to help control bleeding in children and adults with hemophilia A who have inhibitors. The treatment works by helping the blood to clot better. Emicizumab is approved for regular use in patients with severe hemophilia A with and without inhibitors.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:6+
Key Eligibility Criteria
Disqualifiers:Thromboembolic Disease, HIV, Others
Must Be Taking:Emicizumab
5 Participants Needed
Gene Therapy for Hemophilia A
Little Rock, Arkansas
This trial is testing a new gene therapy for hemophilia A patients. It uses a harmless virus to help liver cells produce a missing protein needed for blood clotting. This could reduce the need for frequent injections. The therapy aims to provide a long-term solution by enabling the body to produce the necessary protein on its own.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Sex:Male
Key Eligibility Criteria
Disqualifiers:Liver Fibrosis, Renal Disease, Infections, Others
Must Be Taking:FVIII Replacement
12 Participants Needed
Novel Replacement Therapies for Hemophilia A
Dallas, Texas
This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both standard of care treatment for persons with Hemophilia A.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:< 17
Key Eligibility Criteria
Disqualifiers:FVIII Inhibitor, Target Joints, Synovectomy, Others
Must Not Be Taking:Bisphosphonates, Corticosteroids, Estrogen, Others
28 Participants Needed
Nuwiq for Hemophilia A
San Antonio, Texas
Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:12+
Sex:Female
Key Eligibility Criteria
Disqualifiers:Coagulation Disorder, FVIII Inhibitor, Severe Liver/kidney Disease, Pregnancy, Others
Must Be Taking:Factor VIII
28 Participants Needed
PF-06838435 for Hemophilia B
Highland Heights, Kentucky
Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation sub-study evaluating safety, tolerability, and kinetics of a higher dose with long-term safety and efficacy follow-up. Participants in the substudy do not need to have participated in C0371005.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18 - 65
Sex:Male
Key Eligibility Criteria
Disqualifiers:Hepatitis, HIV, Liver Disease, Others
Must Be Taking:Factor IX Products
21 Participants Needed
Fitusiran for Hemophilia
Akron, Ohio
Primary Objective:
To characterize the long-term safety and tolerability of fitusiran
Secondary Objectives:
* To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of:
* Bleeding episodes
* Spontaneous bleeding episodes
* Joint bleeding episodes
* To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Surgery, Liver Disease, Thromboembolism, Others
Must Not Be Taking:Factor Concentrates, BPAs
281 Participants Needed
SEVENFACT® for Hemophilia
Cleveland, Ohio
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk of experiencing a breakthrough bleeding event (BE), or who are not on prophylactic treatment who may need to control a BE.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:12 - 100
Key Eligibility Criteria
Disqualifiers:Other Hemostasis Disorders, Rabbit Allergy, Others
Must Not Be Taking:Unapproved Prophylactic Drugs
55 Participants Needed
Fitusiran for Hemophilia
Cleveland, Ohio
This trial tests fitusiran, a medication that helps reduce bleeding in male adults and adolescents with hemophilia A or B. It works by lowering antithrombin levels to improve blood clotting.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Thromboembolism, Liver Disease, HIV, Others
79 Participants Needed
Why Other Patients Applied
"My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."
HZ
Arthritis PatientAge: 78
"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."
WR
Obesity PatientAge: 58
"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."
ZS
Depression PatientAge: 51
"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."
FF
ADHD PatientAge: 31
"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."
ID
Pancreatic Cancer PatientAge: 40
Fitusiran for Pediatric Hemophilia
Cleveland, Ohio
Primary Objective:
- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to \<12 years of age) with severe hemophilia A or B
Secondary Objectives:
* To characterize the safety and tolerability
* To determine fitusiran plasma concentrations at selected time points
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:1 - 11
Sex:Male
Key Eligibility Criteria
Disqualifiers:Bleeding Disorders, Liver Disease, HIV, Others
Must Not Be Taking:Emicizumab
32 Participants Needed
Jivi for Hemophilia A in Children
Cleveland, Ohio
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. People with hemophilia A may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding. In severe hemophilia A (clotting factor 8 levels less than 1%) bleedings are more likely to happen.
In this study researchers want to learn more about the treatment called BAY94-9027. BAY94-9027 is an injectable medicine used to replace missing clotting factor 8. In BAY94-9027 the clotting factor 8 has been pegylated (combined with a substance called polyethylene glycol (PEG)). This is to make the treatment last longer in the body so that less injections are required. BAY94-9027 is already available for the prevention and treatment of bleeding in adults and children who are 12 years and older. BAY 94-9027 is also called Jivi.
BAY94-9027 is not yet available for children aged 7 to less than 12 years. One potential specific risk of pegylated drugs is that proteins in the blood called antibodies are built. These may attach to the pegylation part of the drug and this in turn may lead to allergic reactions and the drug not working as well as it should during first 4 infusions. In studies that have been done so far, this has been seen in some children younger than six years, but not in 29 children aged 6 to less than 12 years treated with BAY94-9027. Further safety information related to how the body reacts to BAY94-9027 is however still needed for this age group.
The main purpose of this study is to learn how safe BAY94-9027 is (safety) and how it affects the body (tolerability) in previously treated children with severe hemophilia A who are between 7 to less than 12 years. To answer this question, the researchers will study information about two medical problems of special interest, if allergic reactions occur (also called hypersensitivity) and if the drug is not working as well as it should (also called loss of efficacy) during the first 4 infusions.
Allergic reactions may range from mild local reactions to widespread effects such as shortness of breath, skin rashes and low blood pressure. Only allergic reactions related to the study treatment will be considered.
The assessment if loss of efficacy occurred will be based on the occurrence of bleeding, the clotting factor 8 level in blood after injection called recovery, clotting factor 8 inhibitor tests and measurement of antibodies against the PEG.
The study has two parts, A and B. Part A takes 6 months and part B takes 18 months. In part A the participants will receive two injections of BAY94-9027 per week. In part B, the number of injections may be decreased, with up to five days between the injections. The participants in this study will visit the study site around 14 times and will have 15 phone visits. In part A, visit 1 is for screening. Visits 2 to 5 take place twice a week for two weeks. Visit 6 two weeks after visit 5, visits 7 to 10 take place monthly with visit 11 six weeks after visit 10. In part B, site visits will occur on month 9, 12, 18 and 24 and phone calls every month between the site visits. The participants' and their caregivers will record in an electronic patient diary information about when the study treatment was given and bleeding episodes that have happened.
During the study, the study doctors and their team will
* take blood samples,
* do physical examinations,
* review the participants' electronic diary
* ask questions about the participants' quality of life,
* ask the participants questions about how they are feeling and what adverse events they are having An adverse event is a medical problem that happens during the study. Doctors keep track of all adverse events that happen in study, even if they do not think the adverse events might be related to the study treatments.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:7 - 11
Sex:Male
Key Eligibility Criteria
Disqualifiers:FVIII Inhibitors, Other Bleeding Disorders, Others
Must Not Be Taking:Antihistamines, Chemotherapy, Corticosteroids, Others
35 Participants Needed
Gene Therapy for Hemophilia B
Lexington, Kentucky
The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene. Recruitment will be limited to adults (≥ 18 years) with a confirmed diagnosis of hemophilia B (HB), resulting from a missense mutation in the coagulation factor IX (FIX) gene or a nonsense mutation that has not been associated with an inhibitor. Only subjects who have no evidence of active hepatitis or anti-hFIX antibodies, and who have been treated/exposed to Factor IX concentrates for at least ten years and have had an average of 3 bleeding episodes per year requiring FIX administration will be enrolled. Patients will be recruited within the United States for treatment at St. Jude Children's Research Hospital, and patients will be recruited in England and other countries for treatment in London by our British collaborators.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Sex:Male
Key Eligibility Criteria
Disqualifiers:Hepatitis, HIV, Liver Dysfunction, Others
Must Be Taking:Factor IX Concentrates
14 Participants Needed
CSL222 Gene Therapy for Hemophilia B
Ann Arbor, Michigan
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Key Eligibility Criteria
Disqualifiers:Severe Hepatic Impairment, Thrombocytopenia, Others
Must Be Taking:FIX Prophylaxis
35 Participants Needed
BE-101 for Hemophilia B
Ann Arbor, Michigan
The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately severe or severe Hemophilia B. Once infused, BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX. BE-101 is an autologous (person's own cells) B Cell Medicine (BCM) which uses CRISPR/Cas9 gene editing to precisely insert human FIX gene into those cells.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Sex:Male
Key Eligibility Criteria
Disqualifiers:B-cell Malignancy, Immunodeficiency, Thromboembolic, Others
Must Be Taking:Factor IX Products
24 Participants Needed
Gene Therapy for Hemophilia B
Ann Arbor, Michigan
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of a gene into your DNA) called REGV131-LNP1265, also called the "study drug". Gene insertion therapy aims to teach the body how to produce clotting factor long-term, without the need for factor replacement therapy.
The main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it.
The study is looking at several other research questions including:
* How much study drug is in the blood at different times
* Whether the body makes antibodies against parts of the study drug, which could make the drug less effective or could lead to side effects. Antibodies are proteins produced by the body's immune system in response to a foreign substance
* Whether the body makes antibodies against the clotting factor replacement therapy
* How quality of life is affected by hemophilia B and if it changes after taking study drug
* How joint health is affected by hemophilia B and if it changes after taking study drug
* How often visits are required for the emergency room, urgent care center, physician's office, hospital, telephone or online are required as a result of bleeding events, and if the frequency changes after taking study drug
* How often factor replacement therapy is needed, both on a regular basis for prevention of bleeding, and as needed to treat bleeding events (and it if changes after taking study drug)
* Whether there is a difference in 2 different methods for measuring Factor 9 activity in the blood
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Sex:Male
Key Eligibility Criteria
Disqualifiers:Liver Disease, Thrombo-embolic Events, Others
Must Be Taking:FIX Prophylaxis
130 Participants Needed
Gene Therapy for Hemophilia B
Ann Arbor, Michigan
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile.
The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Sex:Male
Key Eligibility Criteria
Disqualifiers:Not Listed
67 Participants Needed
SerpinPC for Hemophilia
Pittsburgh, Pennsylvania
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with hemophilia
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Recruiting
Age:12 - 65
Sex:Male
Key Eligibility Criteria
Disqualifiers:Not Listed
150 Participants Needed
SerpinPC for Severe Hemophilia
Indianapolis, Indiana
This trial tests a new injection called SerpinPC to prevent bleeding in people with severe hemophilia A or B. It aims to see if SerpinPC is safe and effective, especially for those who don't respond well to other treatments. SerpinPC works by helping the blood clot better. Serpins are being explored as potential treatments for hemophilia by rebalancing coagulation.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:12 - 65
Sex:Male
Key Eligibility Criteria
Disqualifiers:Not Listed
60 Participants Needed
Concizumab for Hemophilia
Indianapolis, Indiana
This trial tests concizumab, a new medicine, in people with hemophilia A or B who have inhibitors. It aims to see if concizumab can prevent bleeding by helping the blood clot more effectively. Concizumab has shown good results in earlier studies for hemophilia A and B.
No Placebo Group
Pivotal Trial (Near Approval)
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
Key Eligibility Criteria
Disqualifiers:Hypersensitivity, Coagulation Disorder, Thromboembolic Disease, Others
Must Be Taking:Bypassing Agents
134 Participants Needed
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Frequently Asked Questions
How much do Haemophilia B clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Haemophilia B clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Haemophilia B trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Haemophilia B is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Haemophilia B medical study?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Haemophilia B clinical trials?
Most recently, we added Pediatric Palliative Care for Rare Diseases, Nuwiq for Hemophilia A and aPCC-Emicizumab Safety for Hemophilia A to the Power online platform.
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