Name (Synonyms) | Correlation | |
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drug1950 | Observational Wiki | 0.50 |
drug79 | ADYNOVI Wiki | 0.50 |
drug78 | ADVATE Wiki | 0.50 |
drug1071 | FEIBA Wiki | 0.50 |
drug328 | BAX 888 Wiki | 0.50 |
There are 4 clinical trials
The purpose of the study is to document the natural history of hemophilia A disease and long-term outcomes in terms of effectiveness, safety and quality of life in participants receiving Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) in routine clinical practice
Description: The World Federation of Hemophilia developed a musculoskeletal evaluation system, commonly referred to as the Gilbert test, to measure hemophilia joint health status.The Gilbert test needs to be performed in the absence of acute bleed, acute pain, and acute inflammation into the evaluated joint. Four parameters are used in each Gilbert test: pain (score: 0-3), bleeding (score: 0-3), physical exam (score: 0-12), and X-ray evaluation (score: 0-13) Scores of 0, represent no pain, no bleeding, no physical exam issues, and/or no x-ray issues. Higher scores for each of these categories represents worsening conditions.
Measure: Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale Time: Up to approximately 12 yearsDescription: The annualized bleed rate for all joints will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.
Measure: Annualized Bleed Rate, All Joints Time: Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: The annualized bleed rate for all bleeds will be calculated per participant and summarized over the set of available participants.with a minimum observation period of 90 days per treatment regimen.
Measure: Annualized Bleed Rate, All Bleeds Time: Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: The annualized bleed rate for pre-existing target joints at baseline will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.
Measure: Annualized bleed rate, pre-existing target joints at baseline Time: Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: The incidence of new target joints will be calculated as the total number of new target joints in all participants divided by the total number of observation days.
Measure: Incidence of New Target Joints Time: Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: The status of joint health by X-ray by Pettersson score will be summarized for each observational year.
Measure: Status of joint health by X-ray by Pettersson scale Time: Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit.Description: LSS score format= A(e:s:h). Sum of values for Subchondral Cyst (score: 1-6), irregularity/erosion of Subchondral Cortex (score: 1-4), and Chondral Destruction (score: 1-6) gives value for the A component of score. e, s, h components represent effusion/hemarthrosis, hypertrophic synovial, & hemosiderin deposition (score: 0-4 for each). Max. score is 16(4:4:4). Subchondral Cyst: ≥1 bone ≥2 bones >3 cysts in ≥1 bone >3 cysts ≥2 bones Largest size >4 mm: ≥1 bone Largest size >4 mm: ≥2 bones Subchondral Cortex ≥1 bone ≥2 bones Involve > half joint surface: ≥1 bone Involve > half of joint surface: ≥2 bones Chondral Destruction ≥1 bone ≥2 bones Full thickness defect (FTD): ≥1 bone FTD: ≥2 bones FTD involves >1/3 of joint surface: ≥1 bone FTD involves >1/3 of joint surface: ≥2 bones Effusion/hemarthrosis (e): Hypertrophic synovial (s): Hemosiderin (h): (0-4 for each): 0 absent 1 equivocal 2 small 3 moderate 4 large
Measure: Status of Joint Health by Magnetic Resonance Imaging (MRI) Scoring System- Using The Lund Scoring System (LSS) Time: Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: The International Prophylaxis Study Group (IPSG) developed a scoring system for musculoskeletal evaluation, the HJHS, optimized for use in children with no or minimal joint disease. The HJHS includes the following parameters: swelling, duration of swelling, muscle atrophy, joint pain, crepitus on motion, flexion loss, extension loss, strength and global gait.
Measure: Status of joint health using the Hemophilia Joint Health Score (HJHS) Time: Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: Excellent: Same or lower breakthrough bleed rate (BBR) within last 12 months (M) compared with prior prophylaxis; if participant did not receive prior prophylaxis with rAHF-PFM, rAHF-PEG or other Factor VIII (FVIII), same or better than expected outcome according to investigator's expectation Good: Minor increase in BBR within last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, slightly less than expected outcome according to investigator's expectation Fair: Moderate increase in BBR in last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, somewhat less than expected outcome according to investigator's expectation Poor: Significant increase in BBR in the 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, little to no benefit according to investigator's expectation
Measure: Overall effectiveness assessment for prophylaxis therapy Time: Annual/Interval visits:- Up to 8 years if rAHF-PFM alone;- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years;- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: Evaluation of patients´ compliance to prescribed prophylactic treatment will be performed by the treating physician. Compliance will be categorized according to a 4-point table (Highly compliant, Fairly compliant, Moderately compliant, Poorly compliant)
Measure: Compliance with the dosing prescribed and its relationship with effectiveness Time: Annual/Interval visits:- Up to 8 years if rAHF-PFM alone;- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years;- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: Excellent: Bleed episodes typically respond to same or fewer number of infusion and same or lower dose as compared with previous on-demand treatment or investigator's expectation Good: Most bleed episodes typically respond to same number of infusion and dose but some require more infusions or higher dose as compared with previous on-demand treatment or investigator's expectation Fair: Bleed episodes typically require more infusions and/or higher dose than expected as compared with previous on-demand treatment or investigator's expectation Poor: Bleed episodes routinely fail to respond to same number of infusion and dose and require additional or different factor concentrate for hemostatic control as compared with previous on-demand treatment or investigator's expectation
Measure: Overall effectiveness assessment for on-demand treatment Time: Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: Excellent: Full relief of pain and cessation of bleeding as evidenced by objective signs (e.g., swelling, tenderness, irritability, inconsolability, and decreased range of motion in the case of musculoskeletal hemorrhage) within approximately 8 hours of a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after the infusion. Possibly requires more than 1 infusion for complete resolution. Fair: Probable or slight relief of pain and slight improvement in signs of bleeding within approximately 8 hours after the infusion. Requires more than 1 infusion for complete resolution. Poor: No improvement or condition worsens.
Measure: Global effectiveness assessment for on-demand treatment Time: Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)
Measure: Number of rAHF-PFM or rAHF-PEG units required for bleed cessation Time: Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)
Measure: Number of rAHF-PFM or rAHF-PEG infusions required for bleed cessation Time: Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visitDescription: The the lHAL measures activities involving the upper extremities, basic activities involving ower extremities and complex activities involving the lower extremities as well as an overall physical activity score for adults.
Measure: Quality of Life: HAL questionnaire - for adult patients Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: The SF-12v2 measures generic health-related quality of life for adults.
Measure: Quality of Life: SF-12v2 questionnaire - for adult patients Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: The EQ-5D measures health utility in adult participants.
Measure: Quality of Life: EQ-5D questionnaire - for adult patients Time: Enrollment visit;Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: The PedHAL measures activities involving the upper extremities, basic activities involving the lower extremities and complex activities involving the lower extremities as well as an overall physical activity score for children. For participants 4-13 years of age: - PedHAL (parent version) For participants 14-17 years of age: - PedHAL (child version)
Measure: Quality of Life: PedHAL questionnaire - for pediatric patients Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: The SF-10 measures generic health-related quality of life for children and is parent-completed.
Measure: Quality of Life: SF-10 questionnaire - for pediatric patients Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: The EQ-5D measures health utility in subjects aged 14 and up.
Measure: Quality of Life: EQ-5D (14 and up) questionnaire - for pediatric patients Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain). During screening visit and on an annual basis, the investigators shall ask participants to rate the average level of chronic pain associated with hemophilia over the period of 4 weeks prior to visit date using the VAS.
Measure: Chronic pain associated with hemophilia, as measured over a period of 4 weeks on an annual basis, using the visual analog scale (VAS) Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain). Participants will be asked to provide ratings on level of acute pain associated with each bleeding episode using the VAS. The VAS scores will be recorded in the participant diary.
Measure: Acute pain associated with hemophilia, as measured with individual bleeding episodes, using the visual analog scale (VAS) Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: Difference in number of weekly prophylactic infusions between previous regimen and rAHF-PEG
Measure: Modalities of switching from a standard FVIII product to rAHF-PEG - 1 Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: Difference in number of weekly doses between previous regimen and rAHFPEG
Measure: Modalities of switching from a standard FVIII product to rAHF-PEG - 2 Time: Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visitDescription: Incidence of inhibitors after switching to rAHF-PEG in the same subgroups of patients
Measure: Incidence of inhibitors after switching to rAHF-PEG Time: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)The purpose of this study is to: - 1. To evaluate the tolerability and safety of infusing reduced volume Factor Eight Inhibitor Bypassing Activity (FEIBA) at the standard infusion rate of 2 U/kg/min - 2. To evaluate the tolerability and safety of infusing reduced volume FEIBA at increased rates of 4 and 10 U/kg/min, in comparison to the standard rate of 2 U/kg/min at the regular volume
Description: Number of participants with serious adverse events (SAEs) and non-SAEs will be assessed.
Measure: Number of Participants With Serious Adverse Events (SAEs) and Non-Serious Adverse Events Time: Throughout the study period of approximately 13 monthsDescription: Number of participants with AEs particular to allergic-type hypersensitivity reactions will be assessed.
Measure: Number of Participants With Adverse Events (AEs) Related to Hypersensitivity Reactions Time: Throughout the study period of approximately 13 monthsDescription: Number of participants with AEs particular to thromboembolic events will be assessed.
Measure: Number of Participants With Adverse Events (AEs) Related to Thromboembolic Events Time: Throughout the study period of approximately 13 monthsDescription: Number of participants with AEs related to infusion site reactions will be assessed.
Measure: Number of Participants With Adverse Events (AEs) Related to Infusion Site Reactions Time: Throughout the study period of approximately 13 monthsDescription: Number of participants with adverse events (AEs) leading to discontinuation will be assessed.
Measure: Number of Participants With Adverse Events (AEs) Leading to Discontinuation Time: Throughout the study period of approximately 13 monthsDescription: Number of participants with AEs related to change in vital signs will be assessed. Vital signs will include body temperature (degree Celsius or degrees Fahrenheit [°C or °F]), respiratory rate (breaths/min), pulse rate (beats/min), and systolic and diastolic blood pressure (millimeter of mercury [mmHg]).
Measure: Number of Participants With Adverse Events (AEs) Related to Change in Vital Signs Time: Throughout the study period of approximately 13 monthsDescription: Number of participants with AEs related to change in laboratory assessments will be assessed. Laboratory assessments include hematology, clinical chemistry, coagulation testing, serological testing, pregnancy testing, cluster differentiation 4 (CD4).
Measure: Number of Participants With Adverse Events (AEs) Related to Change in Laboratory Assessments Time: Throughout the study period of approximately 13 monthsThe purpose of this study is to evaluate the safety and determine the dose of BAX 888.
Description: An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.
Measure: Number of Participants With BAX 888-Related Adverse Events (AEs) Time: From study drug administration to 3 YearsDescription: Change from baseline in circulating plasma FVIII activity level, based on one-stage clotting assay will be assessed.
Measure: Change from Baseline in Circulating Plasma FVIII Activity Level Time: Baseline, up to approximately 3 years per participantDescription: Change from baseline in circulating plasma FVIII antigen (protein) levels will be assessed.
Measure: Change from Baseline in Circulating Plasma FVIII Antigen Level Time: Baseline, up to approximately 3 years per participantDescription: Annualized bleed rate (ABR) in comparison to before gene transfer will be assessed. A bleed is defined as subjective or objective evidence of bleeding which may or may not require treatment with FVIII. Annualized bleed rate (ABR) was calculated as (number of bleeding episodes/observed treatment period in days)*365.25.
Measure: Annualized bleed rate (ABR) Time: Throughout the study period of approximately 3 years per participantDescription: The percentage of participants with a reduction in exogenous FVIII consumption 12 months post-infusion and 3 years post-infusion compared to the historical consumption (consumption of exogenous FVIII during the 12 month period prior to BAX 888 infusion).
Measure: Percentage of Participants with a Redaction Consumption of Exogenous Factor VIII (FVIII) Time: Historical data from 12 months prior to study enrollment; and 12 months post-infusion and 3 years post-infusionDescription: Number of participants develop inhibitory antibodies to FVIII will be assessed.
Measure: Number of Participants Develop Inhibitory Antibodies to FVIII Time: Throughout the study period of approximately 3 years per participantDescription: Number of participants develop total binding antibodies to FVIII (Immunoglobulin G [IgG] and Immunoglobulin M [IgM]) will be assessed.
Measure: Number of Participants Develop Total Binding Antibodies to FVIII Time: Throughout the study period of approximately 3 years per participantDescription: Number of participants with humoral (antibody-mediated) and cell-mediated immune response to adeno-associated virus (AAV8) (the vector) and FVIII proteins will be assessed.
Measure: Number of Participants With Humoral and Cell-Mediated Immune Response to AAV8 and Factor VIII (FVIII) Proteins Time: Throughout the study period of approximately 3 years per participantDescription: Surveillance of adeno-associated virus (AAV8) genome shedding in blood, saliva, semen, urine and stool will be assessed.
Measure: Surveillance of AAV8 Genome Shedding Time: Throughout the study period of approximately 3 years per participant,or until 2 consecutive measurements are negative, which ever is soonerBackground. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative and intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and elbows. Pain is one of the main clinical manifestations of hemophilic arthropathy. As a consequence of the COVID-19 pandemic, the Government of Spain, among others, established a total confinement for two months, in order to avoid contagion of the population Objective. To assess the effect of COVID-19 confinement on bleeding frequency, pain perception, and range of motion in patients with hemophilic arthropathy. Study design. Prospective observational study. Method. 27 patients with hemophilia A and B will be included in this study. Patients will be recruited from the Spanish Hemophilia Federation (Fishemo) specialized center for hemophilia patients. The dependent variables will be: the frequency of bleeding (through a self-report), the perception of pain (measured with the visual analog scale and a pressure algometer), the joint state (with the Hemophilia Joint Health Score), and the range of joint movement (measured with a goniometer). Two evaluations will be carried out: pre-treatment (carried out in the month of February, as a periodic evaluation) and post-treatment (at the end of the period of confinement in Spain). Expected results. The aim is to observe the sequelae caused by confinement and a sedentary lifestyle in patients with hemophilic arthropathy, through changes in joint status, pain and range of motion.
Description: The frequency of joint bleeding was assessed using a self-assessment log provided to patients at the beginning of confinement electronically. This registry included a monthly calendar where the patients included the date, symptoms and location of the hemarthrosis.
Measure: Change from baseline frequency of hemarthrosis after confinement period Time: Screening visit, within the first seven days after confinementDescription: Joint status was assessed with the Hemophilia Joint Health Score (HJHS) version 2.1. This tool, specific for hemophilia patients, evaluates joint deterioration in the ankle, knee and elbow joints. This scale measures 8 items: swelling, duration of swelling, atrophy and muscle strength, crepitus, mobility and joint pain. The scoring range for this additive scale is 0-20 points at each joint.
Measure: Change from baseline joint health after confinement period Time: Screening visit, within the first seven days after confinementDescription: Joint pain perception was measured with the visual analog scale. This scale has shown moderate reliability. The scoring range is from 0 to 10 points (from no pain to maximum perceived pain).
Measure: Change from baseline joint pain after confinement period Time: Screening visit, within the first seven days after confinementDescription: Range of motion was measured with a goniometer to assess flexion and extension of the elbow, knee, and ankle joints. This measuring instrument has shown good intra and inter-observer reliability in the joints evaluated. The measurement protocols described by Cleffken, Green and Gerhardt were used to measure mobility in the elbow, knee and ankle joints, respectively.
Measure: Change from baseline range of motion after confinement period Time: Screening visit, within the first seven days after confinementDescription: The pressure algometer measures the pressure, bilaterally, at which the patient perceives pressure pain on the evaluation point, as the pressure increases at a speed of approximately 50kPa / s. The unit of measurement is in Newton / cm2. The perception of joint pain in the elbow was measured on the lateral epicondyle, in the knee at a distance of 3 cm from the midpoint of the internal edge of the patella, and in the ankle it was measured in the ventral region of the lateral malleolus.
Measure: Change from baseline joint pain after confinement period Time: Screening visit, within the first seven days after confinement