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45 Hemophilia Trials

Power is an online platform that helps thousands of Hemophilia 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.

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Efanesoctocog 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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
261 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18+
Sex:Male
22 Participants Needed
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Depemokimab for Hypereosinophilic Syndrome

Columbus, Ohio
This trial is testing a new medication called depemokimab for adults with Hypereosinophilic Syndrome (HES) who are not responding well to their current treatments. The medication aims to lower the number of certain white blood cells that cause symptoms to worsen. Participants will either receive depemokimab or continue their usual treatment.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
120 Participants Needed
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Benralizumab for Hypereosinophilic Syndrome

Columbus, Ohio
This is a multicentre, randomised, double-blind (DB), parallel-group, placebo-controlled, 24-week Phase III study to compare the efficacy and safety of benralizumab versus placebo administered by SC injection Q4W in patients with hypereosinophilic syndrome (HES). This study comprises 2 distinct periods (together defined as the 'main study'): A 24-week, DB treatment period, during which patients will be randomised to receive either benralizumab or placebo, in addition to their prior stable HES background therapy, and an open-label extension (OLE) period, during which all patients will receive benralizumab. The primary database lock (DBL) will occur when approximately 38 patients have had their first HES worsening/flare event during the DB treatment period and all randomised patients have had the opportunity to be followed up for the 24-week DB treatment period. A patient must complete the 24-week DB treatment period on investigational product (IP) to be eligible to enter the OLE treatment period. The final DBL will occur after the last patient completes the OLE.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
117 Participants Needed
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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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
90 Participants Needed
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Mim8 for Hemophilia A

Dayton, Ohio
This trial is testing Mim8, a new medicine to prevent bleeding in people with haemophilia A. It works by replacing the missing part in their blood that helps it clot. The study will last several years and involves frequent injections under the skin.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:1+
Sex:All
451 Participants Needed
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BAX 888 for Hemophilia A

Cincinnati, Ohio
The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults. Participants will receive one infusion with BAX 888 at the hemophilia treatment center. During the study, participants will visit their study clinic multiple times.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18 - 75
Sex:Male
21 Participants Needed
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Mepolizumab for Hypereosinophilic Syndrome

Cincinnati, Ohio
The purpose of this study is to investigate the efficacy and safety of mepolizumab in children and adolescents with hypereosinophilic syndrome (HES) who are receiving standard of care (SoC) therapy.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:6 - 17
Sex:All
16 Participants Needed
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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
21 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
281 Participants Needed
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Emicizumab for Hemophilia A

Cleveland, Ohio
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 (intrinsic factor VIII \[FVIII\] level \<1%) without FVIII inhibitors. The study is designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at 3 milligrams per kilogram of body weight (mg/kg) once every 2 weeks (Q2W) for 52 weeks. After 1 year of treatment, participants will continue to receive emicizumab (1.5 mg/kg once every week \[QW\], 3 mg/kg Q2W or 6 mg/kg once every 4 weeks \[Q4W\]) over a 7-year long-term follow-up period under this study frame.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:0 - 12
Sex:All
55 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:7 - 11
Sex:Male
33 Participants Needed
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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
Sex:All
55 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
79 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:1 - 11
Sex:Male
32 Participants Needed
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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
Age:18+
Sex:Male
14 Participants Needed
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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
Age:18+
Sex:Male
14 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
73 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
136 Participants Needed
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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
Age:18+
Sex:Male
11 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18+
Sex:Male
67 Participants Needed
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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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:12 - 65
Sex:Male
150 Participants Needed
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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
Trial Phase:Phase 2
Age:12 - 65
Sex:Male
60 Participants Needed
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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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
35 Participants Needed
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Emicizumab for Hemophilia A

Pittsburgh, Pennsylvania
This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
51 Participants Needed
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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
Age:18+
Sex:Male
24 Participants Needed
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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
Age:18+
Sex:Male
130 Participants Needed
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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
36 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:Male
158 Participants Needed
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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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18 - 65
Sex:Male
51 Participants Needed
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Frequently Asked Questions

How much do Hemophilia 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 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 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 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 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 clinical trials ?

Most recently, we added aPCC-Emicizumab Safety for Hemophilia A, Dupilumab for Hypereosinophilic Syndrome and BE-101 for Hemophilia B to the Power online platform.

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