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Hemophilia Medical Research Studies

Up-to-date List of Hemophilia Medical Research Studies

What Research is Being Done?

A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies. Following list includes both interventional and observational studies.

Latest Hemophilia Medical Research Studies

Rank Status Study
1 Not yet recruiting Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
Condition: Hemophilia
Intervention:
Outcome Measures: Assess the range of motion;   Assess the muscular strength;   Assess the joint pain;   Assess the variables of strength;   Assess the peak force;   Assess the contractile activity;   Assess the state joint;   Assess the list of activities;   Assess the physical performance;   Assess the health Profile;   Assess the quality of life;   Age, sex, weight of the patients with Hemophilia;   Diagnosis, severity of Hemophilia, type of treatment (prophylactic or on-demand) and inhibitors;   Frequency of joint bleeds in the previous month and bleeding during the assessment
2 Recruiting Inhibitor Development in Patients With Hemophilia A Undergoing Surgery
Condition: Hemophilia A
Intervention:
Outcome Measure: Inhibitor development (inhibitor titer > 0.4 BU/ml)
3 Not yet recruiting Study About of Proprioceptive in Patients With Haemophilia
Condition: Hemophilia
Intervention: Other: Home Training
Outcome Measures: Change of assessment of muscle balance;   Change of isokinetic assessment and analytical force-time;   Change of functional force-time assessment;   Change of functional assessment of peak force;   Change of assessment of contractile activity myotendinous;   Change of joint condition based on clinical assessment;   Change of degree of activities of daily living that are able to perform the patients with haemophilia.;   Change of perceived quality of life.;   Age, sex, height and weight of the patients with haemophilia;   Diagnosis, severity of Hemophilia, treatment (prophylactic or on demand) and inhibitors;   Frequency of bleeding prior to treatment Frequency of bleeding during treatment
4 Unknown  Socialization of Adult Men With Congenital Hemophilia A or B
Conditions: Hemophilia A;   Hemophilia B
Intervention:
Outcome Measures: Health related quality of life, description of support and networks.;   Quality of life and description of role of SSO in health care of their PWCBD partner
5 Recruiting Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: Gene Transfer
Outcome Measure: To assess the safety of systemic administration of a novel self complementary AAV vector in adults with severe Hemophilia B at up to four different dosage levels.
6 Recruiting Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease
Conditions: Hemophilia A;   Hemophilia B;   Von Willebrand's Disease
Intervention:
Outcome Measure: Number of microparticles
7 Unknown  Efficacy and Safety of MK-0518 in Treatment-Experienced HIV-1 Infected Adult Patients With Hemophilia
Conditions: HIV Infection;   Hemophilia A
Intervention:
Outcome Measures: The safety and tolerability of MK-0518 400 mg b.i.d. compared to placebo, both in combination with OBT, assessed by review of the accumulated safety data in HIV-infected patients with Hemophilia.;   Antiretroviral activity of MK-0518 400 mg b.i.d. compared to placebo, both in combination with OBT.
8 Not yet recruiting Allogenic Bone Marrow Derived Mesenchymal Stem Cell Therapy in Cases of Hemophilia
Condition: Hemophilia
Interventions: Biological: Cellular therapy;   Biological: cellular therapy
Outcome Measures: Assessment of Safety / Efficacy;   Assessment of Coagulation Profile
9 Recruiting Hemophilia Inhibitor Previously Untreated Patient Study
Condition: Hemophilia A
Intervention: Drug: FVIII concentrate
Outcome Measures: Total number of FOXP3-positive T regulatory cells in the circulation;   FVIII-specific T-cells
10 Recruiting Hemophilia B Gene Therapy - Spark
Condition: Hemophilia B
Intervention: Biological: AAV8-hFIX19
Outcome Measures: Number of subjects with adverse events related to investigational product;   Circulating plasma factor IX levels
11 Recruiting Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
Condition: Hemophilia
Intervention: Other: Recombinant Factor VIII (Kogenate, BAY14-2222)
Outcome Measures: Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group;   Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group;   Number of overall bleeding episodes;   Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score);   Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score);   Cost-effectiveness (cost of additional joint bleed);   Cost-utility;   Comparison of patient compliance between prophylaxis and on-demand therapy groups;   Number of spontaneous bleeds;   Quality of life as measured with the SF-36;   Quality of life as measured with Hemo-QoL
12 Recruiting A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX
Conditions: Hemophilia A With Inhibitors;   Hemophilia B With Inhibitors
Intervention: Biological: Coagulation Factor VIIa (Recombinant)
Outcome Measures: Bleeding episode treatment success;   Time to bleeding success
13 Recruiting Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
Condition: Hemophilia B
Intervention: Biological: AskBio009
Outcome Measures: Number of patients experiencing treatment-related adverse events by dose group;   Change from baseline in clinical laboratory evaluations;   Changes from Baseline in FIX activity levels, FIX protein levels, and Bleeding Episode Severity & Frequency;   Immune Response to AskBio009;   Detection of AskBio009 genomes in blood, saliva, urine, stool, and semen
14 Recruiting Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa
Condition: Haemophilia
Intervention:
Outcome Measure: Assessment of patient's individual response to therapy with by-passing agents by simultaneous use of TGA and TEM methods.
15 Not yet recruiting Individualized Prophylaxis for Severe Hemophilia A Children
Condition: Hemophilia
Intervention: Other: according to the efficacy of AJBRs
Outcome Measure: AJBRs (annualized Joint bleeding rates)
16 Recruiting A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients
Conditions: Hemophilia A;   Hemophilia B
Interventions: Drug: ALN-AT3SC;   Drug: Sterile Normal Saline (0.9% NaCl)
Outcome Measures: The safety of ALN-AT3SC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicities (DLTs), and AEs leading to study drug discontinuation.;   The pharmacokinetics (PK) of ALN-AT3SC as characterized by plasma PK profiles and urine samples.;   The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma AT levels.;   The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma TG.
17 Recruiting A Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of PF-05280602, A Recombinant Factor VIIa Variant (813d), In Adult Subjects With Hemophilia A Or B
Condition: Hemophilia A
Intervention: Biological: PF-05280602
Outcome Measures: Incdence of subjects wtih treatment emergent adverse events;   Incidence of of subjects with treatment emergent Hemophilia adverse events;   Incidence of subjects with treatment emergent serious adverse events;   Changes from baseline in patient's vital signs- blood pressure;   Changes from baseline in patient's ECG;   Changes from baseline in patient's physical examination;   Incidence of treatment emergent clinical laboratory abnormalities for Tropin T levels;   Magnitude of treatment emergent clinical laboratory abnormalities for Troponin T levels;   Incidence of an immune response;   Incidence of treatment emergent clinical laboratory abnormalities for Anti-Thrombin III;   Incidence of treatment emergent clinical laboratory abnormalities for Tissue Factor Pathway Inhibitor;   Number of subjects with clinically significant changes from baseline in their fibrinogen;   Changes from baseline in patient's vital signs- weight;   Changes from baseline in patient's vital signs- temperature;   Changes from baseline in patient's vital signs- respiration rate;   Changes from baseline in patient's vital signs- pulse rate;   Severity of subjects wtih treatment emergent adverse events;   Severity of of subjects with treatment emergent Hemophilia adverse events;   Severity of subjects with treatment emergent serious adverse events;   Withdrawals due to treatment emergent adverse events;   Withdrawals due to treatment emergent Hemophilia events;   Incidence of treatment emergent clinical laboratory abnormalities for hematology;   Magnitude of treatment emergent clinical laboratory abnormalities for hematology;   Incidence of treatment emergent clinical laboratory abnormalities for chemistry;   Magnitude of treatment emergent clinical laboratory abnormalities for chemistry;   Incidence of treatment emergent clinical laboratory abnormalities for urinalysis;   Magnitude of treatment emergent clinical laboratory abnormalities for urinalysis;   Incidence of treatment emergent clinical laboratory abnormalities for platelet count;   Magnitude of treatment emergent clinical laboratory abnormalities for platelet count;   Magnitude of treatment emergent clinical laboratory abnormalities for Anti-Thrombin III;   Magnitude of treatment emergent clinical laboratory abnormalities for Tissue Factor Pathway Inhibitor;   Incidence of treatment emergent clinical laboratory abnormalities for C-Reactive Protein;   Magnitude of treatment emergent clinical laboratory abnormalities for C-Reactive Protein;   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, Cmax;   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, area under the curve (AUC last);   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, terminal half-life;   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, recovery;   Pharmacodynamic (Hematologic) activity as measured by the Prothrombin Time;   Pharmacodynamic (Hematologic) activity as measured by the activated partial thrombinplastin time;   Pharmacodynamic (Hematologic) activity as measured by the thrombin antithrombin complexes;   Pharmacodynamic (Hematologic) activity as measured by the prothrombin fragments 1+2;   Pharmacodynamic (Hematologic) activity as measured by D-Dimers;   Pharmacodynamic (Hematologic) activity as measured by thrombin generation;   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, area under the curve (AUC inf);   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, mean residence time;   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, Vss;   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, clearance;   Factor VIIa concentration in subject plasma as measured by FVIIa PK assay, Tmax
18 Recruiting A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
Condition: Hemophilia A
Intervention: Biological: BAY94-9027
Outcome Measures: Annualized number of total bleeds;   Safety variables will be summarized using descriptive statistics based on adverse events collection;   Quantification of blood loss in major surgery;   Pharmacokinetic parameters will be measured by Tmax, Cmax, t1/2, AUC, and incremental recovery
19 Not yet recruiting Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
Condition: Hemophilia B
Intervention: Biological: IB1001
Outcome Measures: Number of study subjects with adverse events;   Number of bleeding episodes divided by number of months of observation
20 Recruiting Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery
Condition: Hemophilia A
Intervention: Drug: Recombinant Protein-Free Factor VIII (rAHF-PFM)
Outcome Measures: Comparison of the cumulative packed red blood cell (PRBC) volume in the drainage fluid during 24 hours following surgery in subjects receiving rAHF-PFM by bolus infusion or continuous infusion;   Total amount of hemoglobin in the cumulative drainage fluid;   Actual postoperative blood loss;   Number of bleeding episodes during treatment with continuous or bolus infusion

These studies may lead to new treatments and are adding insight into Hemophilia etiology and treatment.

A major focus of Hemophilia research is the development of new drugs and other treatment options. Studies seek to identify new drugs to treat various related disorders and to find safer, more effective doses for medications already being used. Other research is aimed at identifying receptors or drug targets.


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