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Why We Started Power

We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

Bask
Bask GillCEO at Power
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      Why We Started Power

      We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      29 Hemophilia B Trials Near You

      Power is an online platform that helps thousands of Hemophilia 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 Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      Gene Therapy for Hemophilia B

      Columbus, Ohio
      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 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:Active Not Recruiting
      Trial Phase:Phase 3
      Age:12+

      Key Eligibility Criteria

      Disqualifiers:Thromboembolic Disease, Coagulation Disorders, Others
      Must Be Taking:Coagulation Factors

      153 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

      91 Participants Needed

      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 for Hemophilia B

      Ann Arbor, Michigan
      This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:138+
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Thrombocytopenia, Uncontrolled Infection, Others
      Must Be Taking:FIX Prophylaxis

      18 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
      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

      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, or Factor VII (FVII) deficiency, with or without inhibitors (Part B).

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:18 - 60

      Key Eligibility Criteria

      Disqualifiers:Thrombosis, Cardiac Disease, Hypertension, HIV, Others

      36 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

      Factor IX Gene Therapy for Hemophilia B

      Indianapolis, Indiana
      This trial tests a new gene therapy treatment for adult men with severe hemophilia B. The treatment aims to help their bodies produce a crucial blood-clotting protein. Participants will receive one dose and be monitored over several years to see if it reduces their bleeding episodes. Gene therapy for hemophilia B has shown promising results in recent studies, suggesting potential long-term therapeutic production of the coagulant protein.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:18 - 65
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Chronic Infection, Liver Disease, HIV, Others
      Must Be Taking:Factor IX

      51 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

      Why Other Patients Applied

      "I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

      AG
      Paralysis PatientAge: 50

      "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

      "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 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

      "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
      Match to a Hemophilia B Trial

      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

      Gene Therapy for Hemophilia B

      Chicago, Illinois
      This trial tests a gene therapy for adults with Hemophilia B. The therapy aims to help their bodies produce a missing protein needed for blood clotting by delivering a healthy gene to their liver cells. Gene therapy for hemophilia B has shown promise in previous studies.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Hepatitis C, Cirrhosis, Others
      Must Be Taking:Factor IX

      30 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

      77 Participants Needed

      PF-06741086 for Hemophilia

      Hamilton, Ontario
      Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:12 - 74
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Coronary Artery Disease, Thrombosis, Others
      Must Be Taking:FVIII/FIX Replacement

      186 Participants Needed

      Pediatric Palliative Care for Rare Diseases

      Washington D.C., District of Columbia
      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. This trial will test the FACE-Rare intervention to see if investigators can identify and meet those needs; and if FACE-Rare effects family caregivers' quality of life and child healthcare utilization. Finally, investigators will determine if the intersectionality of child-sex, family-race, Federal poverty level, and social connection influences family quality of life and child health care utilization longitudinally.
      No Placebo Group

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Unphased
      Age:12 - 99

      Key Eligibility Criteria

      Disqualifiers:Homicidal, Suicidal, Psychotic

      480 Participants Needed

      SerpinPC for Hemophilia B

      Greenville, North Carolina
      The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Age:12 - 65
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      3 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

      Recombinant Factor VIIa for Hemophilia

      Atlanta, Georgia
      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) or factor IX (FIX) undergoing elective major surgical procedures.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:12 - 65
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Coagulation Disorders, Immunosuppression, Others
      Must Not Be Taking:Immunosuppressants, NSAIDs, Others

      19 Participants Needed

      Tranexamic Acid for Facelift Surgery

      Minneapolis, Minnesota
      Tranexamic acid (TXA) is a fibrinolytic inhibitor which prevents prolonged bleeding by interfering with fibrin clot breakdown by competitively binding to lysine receptors on plasminogen; this prevents the conversion of plasminogen to plasmin. TXA will be applied to a randomly assigned side of the face during facelift surgery. The intervention groups will include 1% TXA mixed with standard local consisting 1/4% lidocaine with 1:100,000 epinephrine, 3% TXA on TXA-soaked pledgets applied for 10 minutes, and 1% TXA with local plus 3% TXA-soaked pledgets. Each treatment arm will be compared to saline in place of TXA on the contralateral side of the face. Although TXA has been widely used in surgical fields for decades and is officially recommended by agencies such as ACOG for use during maternal hemorrhage, its current FDA approval only pertains to oral TXA for heavy menstrual bleeding and IV use for patients with hemophilia to prevent or reduce hemorrhage (cite). The main concern with intravenous TXA is the increased risk for the potential formation of blood clots, mainly in patients with clotting disorders, such as Facor V Leiden, and patients on estrogen containing medication. A recent systemic review with metanalysis by Wang et.al contained a total of 2150 patients receiving IV TXA while undergoing plastic surgery concluded that use of IV TXA does not lead to increased adverse events.\[12\] Given the low rate of adverse events while using TXA systemically, this protocol's application of TXA topically and/or locally negates the risk for any potential systemic adverse effects. No systemic adverse effects have been reported in studies examining local TXA in facial plastic surgery to date.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1

      Key Eligibility Criteria

      Disqualifiers:Under 18, Adverse TXA Reaction, Others

      50 Participants Needed

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      Frequently Asked Questions

      How much do Hemophilia 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 Hemophilia 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 Hemophilia 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 Hemophilia 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 Hemophilia 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 Hemophilia B clinical trials?

      Most recently, we added Pediatric Palliative Care for Rare Diseases, aPCC-Emicizumab Safety for Hemophilia A and CSL222 for Hemophilia B to the Power online platform.