Investigator Initiated Research Areas of Interest

Maribavir and Cytomegalovirus (CMV) Infections - US Only

• Studies exploring maribavir effectiveness and safety in the real-world setting including:
  
  • Immunocompromised patients with high-risk of CMV infection
  • Recurrent CMV
  • Longer term impact, early treatment (e.g., prophylaxis, preemptive, 1L (1st line therapy/treatment)
  • Diverse demographic populations (e.g., racial, ethnic)
  • Patterns of use
• Studies focusing on effectiveness of maribavir following letermovir use or combination therapy
• Studies examining the impact of maribavir on Health Related Quality of Life Measures (HRQoL) and Patient Reported Outcomes (PROs) among thoracic (heart/lung) transplant patients with CMV infection

Lanadelumab

• Real-world clinical effectiveness of lanadelumab, for patients newly initiated on lanadelumab or for patients who have previous experience with alternative therapies for HAE
• Novel efficacy outcomes (e.g. remission) or composite efficacy end-points (e.g. patient-reported outcomes and biomarkers) in patients with HAE treated with lanadelumab
• Investigate potential for lanadelumab to modify the contact system (e.g. changes in trigger sensitivity, sustained control or suppression)
• Efficacy of lanadelumab in other conditions where bradykinin or the kallikrein–kinin system may form the pathological basis of disease

HAE

• Approaches to facilitating diagnosis and decreasing diagnostic delay
• Characterization of non-histaminergic angioedema, including pathophysiology, prodrome, diagnosis and biomarkers
• Explore the burden of illness and the burden of treatment, including paediatrics

Ulcerative Colitis (UC), Crohn’s Disease (CD) and Inflammatory Bowel Diseases (IBD)

• Understanding the biological mechanisms that drive clinical response to Vedolizumab in TNF naïve vs exposed UC/CD patients
• Transmural healing with Vedolizumab
• Efficacy of Vedolizumab in early/non-complicated CD
• Efficacy of Vedolizumab in combination with advanced therapeutic agents in UC and CD
• “Treat-to-Target”, “Tight Monitoring” approach with Vedolizumab in CD
• Disease modification by Vedolizumab in CD and UC
• Real World SC Vedolizumab Efficacy and Safety
  
  

Gastrointenstinal Fistulizing Conditions

Clinical outcomes:

• Effect of long-term seton vs short-term seton on subsequent gastrointestinal fistula treatment success
• Impact of radiological healing of gastrointestinal fistula on disease recurrence
• Patient care pathways and effect of multidisciplinary teams and/or patient involvement in shared decision making on gastrointestinal fistula outcomes
• Gastrointestinal fistula surgeries: post-surgical complications and/or short-term Health related quality of life (HRQoL) and work productivity
• Identification of optimal treatment targets in gastrointestinal fistulizing conditions leading to long-term remission
• Novel treatment approaches and clinical outcomes in Crohn's-related Rectovaginal Fistula (RVF)

Translational research:

• Identification, development and characterization of pre-clinical in vivo proof of concept and in vitro cellular models predictive of fistulizing pathobiology
• Microbiome composition in fistulizing conditions such as Complex Perianal Fistula (CPF), Complex Cryptoglandular Fistula (CCF), and Rectovaginal Fistula (RVF) (fistula swabs, stool samples)
• Cellular composition and characterization of inflammation in Crohn’s disease complex perianal fistula and/or complex cryptoglandular fistula
  
  

Celiac Disease (CeD)

• Adoption of new methods and technologies to diagnose and manage patients with celiac disease:
  
  • use of video capsule endoscopy (VCE) tools for diagnosing and monitoring CeD and its role in clinical guidelines
  • tools and studies objectively assessing real world gluten exposure
• Gluten exposure/challenge design in clinical trials
• Biomarkers of the future when monitoring CeD
• Disease burden in special populations with highest unmet need (pediatric, adolescents, elderly)
• Correlation of histology/serology/symptoms/other risk factors with disease progression/complications
• Epidemiology studies
• Humanistic burden and patient / healthcare professional (HCP) preferences
• Disease pathophysiology (especially advances in immunology)
• Optimization of gluten-free diet (GFD) when managing CeD
• New modalities and development targets in CeD
• New indications with relevance to gluten sensitivities/wheat allergy
  
  

Prucalopride - US Only

• Pilot studies in Functional Gastrointestinal Diseases with rationale for prokinetic use
• Safety and efficacy of prucalopride in secondary causes of constipation
• Real world, retrospective studies of prucalopride in adults with chronic idiopathic constipation
  
  

Alpha-1 Antitrypsin Deficiency (AATD) Liver Disease

• Studies focusing on identification and validation of non-invasive methods for assessment of liver fibrosis in AATD
• Studies exploring predictors of disease progression in AATD liver disease or for the development and or progression of liver disease in patients with or without AATD lung disease
• Studies exploring novel markers for the early identification of patients with AATD-LD and diagnostic characterization of disease stage
• Studies evaluating correlations between intrahepatic globule accumulation, patient-centered outcomes and/or clinical outcomes

Dravet syndrome or Lennox-Gastaut syndrome

• Disease management, treatment patterns, polypharmacy, predictors of treatment response or discontinuation, Health Resources Utilization (HRU), or long-term treatment outcomes
• Novel diagnostic approaches or algorithms
• Linkage of genotype to clinical phenotype and/or treatment response
• Burden of illness, caregiver burden and/or treatment burden
• Natural history, seizure patterns or disease progression
• Novel tools or methodologies evaluating comorbidities or non-seizure symptoms, e.g. cognitive functioning, behavior, daily functioning
• Epidemiology [Prevalence, incidence and diagnostic rates]

Narcolepsy

• Explore the role of orexin in the pathophysiology of narcolepsy and idiopathic hypersomnia
• Functional outcomes research related to treatment patterns for narcolepsy
• Characterize the disease burden and unmet needs of patients with narcolepsy

Attention Deficit Hyperactivity Disorder

• C&A and Adult ADHD disease management – recognition, & treatment
• Functional outcomes research related to optimal and suboptimal treatment of ADHD symptoms
• Lisdexamfetamine dimesylate in C&A and Adult ADHD and other comorbid conditions
• Effectiveness of Lisdexamfetamine dimesylate RWE
  
  • Guanfacine use in complex ADHD patients
  • Guanfacine use in combination with stimulants RWE
• Guanfacine effect on symptoms and functioning in ADHD patients across ages RWE
• Adherence & persistence to treatment options in ADHD
• Role of adjunctive psychotherapy

Binge Eating Disorder

• Observational Studies on patient characteristics, comorbidities, treatment patterns, outcomes
• Role of adjunctive psychotherapy

Hemophilia A

• Studies examining the cost efficiency of recombinant Antihemophilic Factor (ADVATE) and PEGylated –recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI) with or without myPKFiT
• Studies examining the relationship between FVIII levels and the occurrence of bleeds at varying physical activity levels with or without the use of the myPKFiT mobile app
• Studies to investigate changes in adherence and quality of life (QoL) in patients using recombinant Antihemophilic Factor (ADVATE) and the myPKFiT patient app
• Real World Evidence on use of PEGylated -recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI), an extended half-life rFVIII (EHL rFVIII), in clinical practice with or without myPKFiT (including safety, efficacy, utilization, QoL, adherence, patient satisfaction etc.)
• Studies on other non-coagulation effects of Factor VIII
• Studies looking at the GOAL-HEM (Goal Attainment Scaling for Life – Hemophilia) as a patient-centered reported outcome measure to monitor clinical progress
• Non-clinical studies on Polyethylenglycol (PEG) safety
• Role of PEGylated -recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI) for tolerization or in previously tolerized/partially tolerized patients
• Studies to investigate appropriate assessment of joint health and prevention/management of all bleeds including subclinical
• Studies to investigate benefit of FVIII replacement therapy vs. non-factor therapy in optimising bleed outcomes
  
  

Recombinant-porcine Antihemophilic Factor and Acquired Hemophilia (AHA)

• Prospective or retrospective studies that provide insights on first-line use, loading dose, dosing over time, FVIII:c and anti-drug antibodies
• Explore efficacy and safety of recombinant-porcine Antihemophilic Factor in patient subpopulations (i.e. post-partum or patients with specific comorbidities) with AHA
• Relationship between treatment effectiveness, FVIII level and anti-pFVIII inhibitor titer in subjects with AHA receiving recombinant-porcine Antihemophilic factor
• Development/validation of dosing algorithms for recombinant-porcine Antihemophilic factor, initial and follow-on, when the anti-porcine FVIII titers are unknown.
• Relationship between treatment effectiveness and recombinant-porcine Antihemophilic factor dosing in subjects with AHA
• Explore the potential use of recombinant-porcine Antihemophilic factor as treatment for breakthrough bleeds in patients treated with non-factor therapies [i.e. Emicizumab]
• Studies intended to develop flexible and tailored dosing regimens for recombinant-porcine Antihemophilic Factor
• Investigate effectiveness, safety and treatment outcomes of the continuous infusion of recombinant-porcine Antihemophilic Factor
• Collect long term data on treatment for patients with AHA
• Clinical outcomes of Anti-Inhibitor Coagulant Complex for treatment of AHA patients
  
  

Hemophilia Gene Therapy

• Measures to evaluate outcomes with hemophilia gene therapy (clinical, humanistic, quality of life, economic, biomarkers, etc.)
  
  

Thrombotic Thrombocytopenic Purpura (TTP)

• Long-term outcomes and disease progression in TTP (congenital and or/acquired TTP) with current standard of care
• Studies of the epidemiology, disease burden, and/or health care utilization of TTP patients
• Genotype/phenotype correlations among TTP patients
• Correlation of ADAMTS13 levels with outcomes
• Investigate effects and adverse events associated with chronic plamma use
• Mechanistic investigations into rADAMTS13 and TTP
• Interactions of ADAMTS13 with other proteins that might affect clinical outcomes
• Understanding the relationship between subclinical events and development of disease-related complications in TTP
• Using clinical biomarkers as proxy for on-going development of disease-related complications in TTP
  
  

von Willebrand Disease (VWD)

• Projects aiming to improve knowledge in the management of prophylaxis, heavy menstrual bleeding (HMB), post-partum bleeding, gastrointestinal (GI) bleeding, severe epistaxis, major surgeries
• Comparison of plasma-derived von Willebrand factor (pdVWF) and recombinant von Willebrand factor (rVWF) in the treatment of GI bleeds or HMB
• Effectiveness and safety of von Willebrand factor (VWF) in on demand/surgery and prophylaxis in real world setting (including elderly patient or those having a cancer or thrombotic risk)
• Personalization of VWD therapy (genetic, bleed prediction, bleeding assessment tools, multidisciplinary approach, …)
• Studies to examine the relationship(s) between the rVWF characteristics, its half-life, its multimeric profile and its clinical efficacy
• Role of VWF multimeric forms in the control of angiogenesis including biomarkers of angiogenesis (rVWF in angiodysplasia)
• Impact of long-term management of joint bleeds on quality of life (QoL) and/or healthcare resource utilization (HRU); impact of the management of HMB on QoL and HRU
• Assessment of the efficacy of VWF (pdVWF or rVWF) in acquired VWS – left ventricular assist device (LVAD), extracorporeal membrane oxygenation (ECMO)

Rare autoimmune diseases (RAID), including chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), multifocal motor neuropathy (MMN) and other peripheral neuropathies

• Studies intended to improve diagnosis and care
• Research that intends to identify biomarkers to improve diagnosis or to measure treatment outcome
• Studies that assess and evaluate QoL, personalized health, and perceived health

Immunodeficiencies

Secondary Immunodeficiency (SID):

• Studies that intend to understand the course of infections in patients with cancer-and treatment-related SID (especially chimeric antigen receptor (CAR) T-cell therapy, posthematopoietic stem cell transplant (HSCT), and solid organ transplantation (SOT)), for example: timing of infections, frequency, types, and impact on patient outcomes and treatment
• Studies that aim to establish or validate existing predictive model or to identify single biomarker that could help to select patients at high risk of developing infections in cancer related and treatment related SID
• Studies with a focus on understanding who would benefit the most from prophylactic or on demand immunoglobin (IG) therapy
• Analyses that quantify and qualify the impact of SIDs and/or impact of IG therapy (prophylactic and on-demand) vs standard of care (SOC) on clinical and patients’ related outcomes (e.g. efficacy, long term safety, quality of life, personalized health, and perceived health)

Primary Immunodeficiency (PID):

• Real World Evidence studies that provide insights into usage and administration parameters of subcutaneous immunoglobulin (SCIG) products, particularly in special populations (pediatric, elderly, obese patients)
• Studies that assess and evaluate QoL, personalized health, and perceived health for IG replacement therapy
• Studies that assess and evaluate the application of new device or digital health concepts in the management of PID
• Studies that explore novel or validate existing approaches in support of earlier PID diagnosis – especially those that could have global impact
• Studies with a focus on understanding the full presentation of PID including the impact of comorbidities to PID and/or vice versa

Albumin

• Clinical and analytical studies related to the concept of functional albumin
• Clinical experience with Flexbumin in patients with liver disease, and patients managed in intensive care units (ICUs)
• Central line-associated bloodstream infections (CLABSIs): Burden of illness,
  pharmacoeconomic impact and evaluations of prevention strategies
• Clinical outcomes of hyperoncotic (25% albumin) Flexbumin in patients with liver cirrhosis and critical care
• Impact of closed infusion system in albumin therapy on albumin storage, preparation, and handling
• Identification of biomarkers for albumin treatment in liver disease and critically ill patients 
  
  

Alpha1-antitrypsin (AAT) deficiency (AATD) and AAT Augmentation Therapy:

• AATD pathogenesis, mechanisms, and biomarkers
• AATD clinical, humanistic, and economic burden of illness and co-morbid conditions
• Characterization of MZ patients
• Impact of early diagnosis and screening
• AAT Augmentation Therapy: for example, but not limited to:
  
  • Adherence in AATD patients with emphysema/chronic obstructive pulmonary disease (COPD)
  • AAT deficient patients in specific populations, (i.e. young
    adults/adolescents/older/post lung-transplant/forced expiratory volume (FEV) 1 35% Predicted)
  • Exploratory research on anti-inflammatory, immunomodulatory, and tissue protective effects of AAT therapy in non-AATD individuals (i.e. graft vs host disease, autoimmune, rheumatoid arthritis, cardiac)

C1-esterase inhibitor

• Real-world data on effectiveness and safety of on demand use, as well as pre-procedural treatment, short-, and long-term prophylaxis with C1-esterase inhibitor in HAE patients
• Understand dosing of C1-esterase inhibitor in the real-world setting
• Switching from oral HAE medications to C1-esterase inhibitor (including, but not limited to, reasons and patients` perception/preferences/reported outcomes)

For disease related, non-product specific areas of interest please visit Hereditary Angioedema (HAE) section of the website (see above).

Hemophilia A and B Inhibitor prevention and management

• Pre-clinical and clinical studies examining the care and treatment of persons with inhibitors and the use of Anti-Inhibitor Coagulant Complex to control and prevent bleeding episodes and perioperative management in adults and children as well as its impact on quality of life (QoL).
• Studies exploring the potential immunotolerance effect of Anti-Inhibitor Coagulant Complex prophylaxis during Immunotolerance induction (ITI) in hemophilia patients with inhibitors.
• Studies examining clinical effectiveness and pharmacoeconomic aspects of using Anti-Inhibitor Coagulant Complex in prophylaxis as well as in ITI.
• Studies examining the use of Anti-Inhibitor Coagulant Complex in prophy-laxis with low dose regimens: regimens, clinical outcomes, pharmacoeco-nomic implications, QoL
• Studies examining potential correlations between thrombin generation assay (TGA) parameters and clinical outcomes.
• Investigations of differences in patient profiles for hemophilia patients with and without inhibitors, and possible correlations with prediction, pre-vention, eradication and prophylaxis of inhibitors.
• Explore whether knowledge from other immunological disease states can be used to develop better management of inhibitor patients.
• Investigate safety profile of Anti-Inhibitor Coagulant Complex to identify possible predictors of outcomes.
• Investigate the use and cost effectiveness of low dose FVIII ITI with Anti-Inhibitor Coagulant Complex as compared to high dose FVIII ITI alone. Investigate the use and cost effectiveness of low dose FIX ITI with Anti-Inhibitor Coagulant Complex as compared to high dose FIX ITI alone or with other components
• Preclinical and clinical studies examining the interaction between the co-administration of Anti-Inhibitor Coagulant Complex and non-factor replacement therapies: regimens and doses, clinical outcomes, safety (TMAs & TEEs), pharmacoeconomic implications, global assays.
• Investigate the use of Anti-Inhibitor Coagulant Complex in other diseases where it is required to reestablish the coagulation cascade (i.e. vitamin K deficiency, reversal of NOACs, hepatopathy, etc.)

Brentuximab vedotin - Outside the US and Canada

• Combination studies in Hodgkin’s Lymphoma with programmed cell death protein 1 (PD-1) / programmed cell death ligand 1 (PD-L1) inhibitors
• Studies which evaluate CD30 expression level, binding and resistance, and reversibility of peripheral neuropathy
• Studies with CD30 expressing Peripheral T-cell lymphoma (PTCL) in front-line therapy and combination studies in relapsed/refractory PTCL

Contact Seagen for US or Canadian proposals

Brigatinib - Global

• Studies investigating brigatinib in NSCLC, evaluation of CNS activity, rational combinations with other agents/modalities, and sequencing of ALK TKIs
  
  

Ixazomib - Global

Currently not accepting proposals

Mobocertinib - Global

• Studies exploring optimal sequencing of epidermal growth factor receptor (EGFR) inhibitors in Exon 20 insertion mutated NSCLC
• Combination studies with mobocertinib in patients with EGFR exon 20 insertion mutated NSCLC, utilizing agents with a clear scientific rationale
• Studies focusing on characterizing and managing Adverse Events (AEs) associated with mobocertinib (mainly GI toxicity, associated mechanisms of cardiac toxicity) including studies that investigate alternate dosing schedules in patients treated with mobocertinib
• Real-World Data (RWD) to evaluate disease state, efficacy and safety of current treatment options and treatment patterns of NSCLC patients with EGFR exon 20 insertion mutations
• Studies that address CNS metastases by exploring different modalities to target CNS disease in patients treated with mobocertinib

Out of Scope

• Studies of mobocertinib in pediatric populations
  
  

Modakafusp alfa (TAK-573) – Global

• Single agent proposals in hematological malignancies

Dengue Disease

• Impact of co-circulation of dengue and COVID-19 on the healthcare system and resources, reporting of dengue, on patient outcomes and quality of care.
• Impact of long-term persistence of dengue symptoms on quality of life and cost to dengue patients and their families; out of pocket financial impact related to dengue borne by families/households
• Studies examining predicted relative morbidity gain (QALY/DALY) in dengue prevention vs other infectious disease preventions
• Impact of socioeconomic factors and urban/rural location on probability of dengue patients to present for medical care
• Epidemiology studies to determine Dengue seroprevalence (national, rural, urban, by age); age groups most at risk of severe dengue outcomes
• Studies to determine extent of dengue under-reporting in Dengue endemic setting
• Burden of Dengue on tourism and migration (Travelers)